From 27f56b4586f6046769cb00139b49af53602ef2e7 Mon Sep 17 00:00:00 2001 From: James Stevenson Date: Mon, 16 Dec 2024 09:08:11 -0500 Subject: [PATCH 1/2] fix: resolve all remaining parsing issues --- src/regbot/fetch/clinical_trials.py | 53 +- ...linical_trial_zolgensma_parsing_error.json | 30734 ++++++++++++++++ tests/test_fetch_clinical_trials.py | 13 + 3 files changed, 30793 insertions(+), 7 deletions(-) create mode 100644 tests/fixtures/fetch_clinical_trial_zolgensma_parsing_error.json diff --git a/src/regbot/fetch/clinical_trials.py b/src/regbot/fetch/clinical_trials.py index 6ba9bf0..bf0114e 100644 --- a/src/regbot/fetch/clinical_trials.py +++ b/src/regbot/fetch/clinical_trials.py @@ -565,7 +565,10 @@ class StandardAge(StrEnum): # these are obviously imprecise, to varying degrees, but it's what we have to work with -_SECONDS_IN_DAY = 24 * 60 * 60 +_SECONDS_IN_MINUTE = 60 +_SECONDS_IN_HOUR = 60 * _SECONDS_IN_MINUTE +_SECONDS_IN_DAY = 24 * _SECONDS_IN_HOUR +_SECONDS_IN_WEEK = 7 * _SECONDS_IN_DAY _SECONDS_IN_MONTH = 31 * _SECONDS_IN_DAY _SECONDS_IN_YEAR = 365 * _SECONDS_IN_DAY @@ -581,8 +584,14 @@ def _age_to_timedelta(raw_age: str) -> datetime.timedelta: factor = _SECONDS_IN_YEAR elif "Month" in raw_age: factor = _SECONDS_IN_MONTH + elif "Week" in raw_age: + factor = _SECONDS_IN_WEEK elif "Day" in raw_age: factor = _SECONDS_IN_DAY + elif "Hour" in raw_age: + factor = _SECONDS_IN_HOUR + elif "Minute" in raw_age: + factor = _SECONDS_IN_MINUTE else: msg = f"Unable to parse '{raw_age}' as a duration" raise ValueError(msg) @@ -879,11 +888,31 @@ def _format_study(study_input: dict) -> Study: ) -def make_fda_clinical_trials_request(url: str) -> list[Study]: +def _get_id(study_response: dict, url: str, i: int) -> str | None: + study_id = ( + study_response.get("protocolSection", {}) + .get("identificationModule", {}) + .get("nctId") + ) + if not study_id: + _logger.error( + "Unable to fetch study ID for the %s 'th entry in %s. Is the JSON object malformed?", + i, + url, + ) + study_id = "" + return study_id + + +def make_fda_clinical_trials_request( + url: str, skip_parsing_failures: bool +) -> list[Study]: """Issue a request against provided URL for FDA Clinical Trials API :param url: URL to request. This method doesn't add any additional parameters except for pagination. + :param skip_parsing_failures: if ``True``, catch and suppress failures to parse + study metadata :return: studies contained in API response """ results = [] @@ -899,9 +928,16 @@ def make_fda_clinical_trials_request(url: str) -> list[Study]: ) raise e raw_data = r.json() - results.extend( - _format_study(study) for study in raw_data.get("studies", []) - ) + for i, study in enumerate(raw_data.get("studies", [])): + try: + parsed_data = _format_study(study) + except ValueError as e: + if skip_parsing_failures: + nct_id = _get_id(study, formatted_url, i) + _logger.warning("Failed to parse study %s: %s", nct_id, e) + continue + raise e + results.append(parsed_data) next_page_token = raw_data.get("nextPageToken") if not next_page_token: @@ -909,7 +945,9 @@ def make_fda_clinical_trials_request(url: str) -> list[Study]: return results -def get_clinical_trials(drug_name: str | None = None) -> list[Study]: +def get_clinical_trials( + drug_name: str | None = None, skip_parsing_failures: bool = False +) -> list[Study]: """Get data from the FDA Clinical Trials API. >>> results = get_clinical_trials("imatinib") @@ -919,6 +957,7 @@ def get_clinical_trials(drug_name: str | None = None) -> list[Study]: :param drug_name: name of drug used for trial intervention. This is passed to the API intervention parameter, which appears to search for inclusion as a substring rather than a full-span match + :param skip_parsing_failures: if ``True`` :return: list of matching trial descriptions """ if not drug_name: @@ -928,4 +967,4 @@ def get_clinical_trials(drug_name: str | None = None) -> list[Study]: if drug_name: params.append(f"query.intr={drug_name}") url = f"https://clinicaltrials.gov/api/v2/studies?{'&'.join(params)}" - return make_fda_clinical_trials_request(url) + return make_fda_clinical_trials_request(url, skip_parsing_failures) diff --git a/tests/fixtures/fetch_clinical_trial_zolgensma_parsing_error.json b/tests/fixtures/fetch_clinical_trial_zolgensma_parsing_error.json new file mode 100644 index 0000000..46fa926 --- /dev/null +++ b/tests/fixtures/fetch_clinical_trial_zolgensma_parsing_error.json @@ -0,0 +1,30734 @@ +{ + "studies": [ + { + "protocolSection": { + "identificationModule": { + "nctId": "NCT04851873", + "orgStudyIdInfo": { "id": "COAV101A12306" }, + "secondaryIdInfos": [ + { "id": "2020-005995-37", "type": "EUDRACT_NUMBER" } + ], + "organization": { "fullName": "Novartis", "class": "INDUSTRY" }, + "briefTitle": "Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)", + "officialTitle": "A Phase IIIb, Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Gene Replacement Therapy With Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)", + "acronym": "SMART" + }, + "statusModule": { + "statusVerifiedDate": "2024-10", + "overallStatus": "INVALID_STATUS_VALUE", + "expandedAccessInfo": { "hasExpandedAccess": false }, + "startDateStruct": { "date": "2021-09-08", "type": "ACTUAL" }, + "primaryCompletionDateStruct": { + "date": "2023-06-13", + "type": "ACTUAL" + }, + "completionDateStruct": { "date": "2023-06-13", "type": "ACTUAL" }, + "studyFirstSubmitDate": "2021-04-06", + "studyFirstSubmitQcDate": "2021-04-19", + "studyFirstPostDateStruct": { + "date": "2021-04-21", + "type": "ACTUAL" + }, + "resultsFirstSubmitDate": "2023-11-27", + "resultsFirstSubmitQcDate": "2024-01-03", + "resultsFirstPostDateStruct": { + "date": "2024-01-05", + "type": "ACTUAL" + }, + "lastUpdateSubmitDate": "2024-10-07", + "lastUpdatePostDateStruct": { "date": "2024-10-09", "type": "ACTUAL" } + }, + "sponsorCollaboratorsModule": { + "responsibleParty": { "type": "SPONSOR" }, + "leadSponsor": { + "name": "Novartis Pharmaceuticals", + "class": "INDUSTRY" + } + }, + "oversightModule": { + "oversightHasDmc": true, + "isFdaRegulatedDrug": true, + "isFdaRegulatedDevice": false + }, + "descriptionModule": { + "briefSummary": "To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing \u2265 8.5 kg and \u2264 21 kg, over a 12 month period.", + "detailedDescription": "This was an open-label, single arm, multi-center study designed to evaluate the safety, tolerability and efficacy of OAV101 in participants with SMA who weigh \u2265 8.5 kg and \u2264 21 kg. The study aimed to enroll approximately 24 to 30 participants, with approximately 6 to 10 participants across each of 3 weight brackets (8.5 to 13 kg, \\>13 to 17 kg, \\>17 to 21 kg).\n\nEligible participants received a single administration of OAV101 at the approved dose of 1.1e14 vg/kg on Day 1 (Treatment period), and were followed for a period of 12 months.\n\nParticipants were admitted to the hospital on Day -1 for pre-treatment baseline procedures. After receiving OAV101 on Day 1, participants underwent in-patient safety monitoring over the next 48 hours, after which the participant could be discharged, based on Investigator judgment.\n\nAfter study completion, eligible participants could enroll into a Long Term follow-up study to collect additional safety and efficacy data. (COAV101A12308 (NCT05335876) https://classic.clinicaltrials.gov/ct2/show/NCT05335876?term=COAV101A12308\\&draw=2\\&rank=1))" + }, + "conditionsModule": { + "conditions": ["Spinal Muscular Atrophy"], + "keywords": [ + "Zolgensma", + "OAV101", + "AVXS 101", + "gene therapy", + "Muscle atrophy", + "SBMA", + "spinal and bulbar muscular atrophy", + "spinal muscular atrophy", + "bulbar muscular atrophy", + "muscle function", + "myopathy", + "muscle wasting", + "atrophied muscle", + "loss of muscle strength" + ] + }, + "designModule": { + "studyType": "INTERVENTIONAL", + "phases": ["PHASE3"], + "designInfo": { + "allocation": "NA", + "interventionModel": "SINGLE_GROUP", + "interventionModelDescription": "Participants will receive a single administration of OAV101", + "primaryPurpose": "TREATMENT", + "maskingInfo": { "masking": "NONE" } + }, + "enrollmentInfo": { "count": 24, "type": "ACTUAL" } + }, + "armsInterventionsModule": { + "armGroups": [ + { + "label": "OAV101", + "type": "EXPERIMENTAL", + "description": "Participants received a single IV dose administration of OAV101", + "interventionNames": ["Genetic: OAV101"] + } + ], + "interventions": [ + { + "type": "GENETIC", + "name": "OAV101", + "description": "Gene Therapy - 1.1e14 vector genome (vg)/kg as a one-time IV infusion was administered over approximately 60 minutes.", + "armGroupLabels": ["OAV101"] + } + ] + }, + "outcomesModule": { + "primaryOutcomes": [ + { + "measure": "Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket", + "description": "An AE is any untoward medical occurrence (e.g. any unfavorable and unintended sign \\[including abnormal laboratory findings\\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study.", + "timeFrame": "Up to Month 12" + }, + { + "measure": "Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket", + "description": "Important identified and important potential risks included the following AESIs: Hepatotoxicity, Thrombocytopenia, Cardiac adverse events, Dorsal root ganglia toxicity and Thrombotic microangiopathy.\n\nThese were assessed by the investigator.", + "timeFrame": "Up to Month 12" + }, + { + "measure": "Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure", + "description": "Change from baseline in vital signs measurements - systolic and diastolic blood pressure (mmHg).\n\nSystolic Blood Pressure-Low:\\<=5th percentile of the age(Any Age), High:\\>=90th percentile of the age, gender, and height group (\\<18 yrs).\n\nDiastolic Blood Pressure-High:\\>=90th percentile of the age, gender, and height group(\\<18 yrs).", + "timeFrame": "12 months" + }, + { + "measure": "Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg)", + "timeFrame": "Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52" + }, + { + "measure": "Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg)", + "timeFrame": "Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52" + }, + { + "measure": "Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min)", + "description": "Change from baseline in vital signs measurements - Respiratory Rate (breaths/min)", + "timeFrame": "Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52" + }, + { + "measure": "Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min)", + "description": "Change from baseline in vital signs measurements - Pulse Rate (beats/min", + "timeFrame": "Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52" + }, + { + "measure": "Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Temperature", + "description": "Change from baseline in vital signs measurements - temperature (degrees Celsius)\n\nTemperature-Low:\\<=35\u00baC(Any Age),High:\\>=38.4\u00baC(\\<18 yrs).", + "timeFrame": "12 months" + }, + { + "measure": "Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius)", + "timeFrame": "Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52" + }, + { + "measure": "Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level", + "description": "Change from baseline in vital signs measurements - oxygen saturation level (%).\n\nOxygen saturation is the fraction of oxygen-saturated hemoglobin relative to total hemoglobin (unsaturated+saturated) in the blood and then multiplied by 100.", + "timeFrame": "Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52" + } + ], + "secondaryOutcomes": [ + { + "measure": "Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development.", + "description": "The World Health Organization-Multicentre Growth Reference Study (WHO-MGRS) and Bayley scale of Infant and Toddler Development was modified and combined into a single scale expressly for this study, to measure developmental motor milestones. These were assessed via the milestone checklist, formed of 10 yes/no questions with optional video documentation. The developmental milestones are: head control, sitting with support, sitting without support, sitting without support for 30 seconds, hands-and-knees crawling, pulls to stand, standing with assistance, walking with assistance, standing alone and walking alone. A yes response indicates that the patient reached a particular development milestone.", + "timeFrame": "Baseline, Week 26 and Week 52" + }, + { + "measure": "Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age", + "description": "The HFMSE was devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE is formed of 33 assessments. Each motor skill item is scored on a 3 point Likert scale from 0 (no response) to 2 (full response), with a total score range of 0 to 66. A higher score indicates a higher level of ability.", + "timeFrame": "Baseline, Week 4, Week 13, Week 26, Week 39 and Week 52" + }, + { + "measure": "Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age.", + "description": "The RULM assesses motor performance in the upper limbs from childhood through adulthood in ambulatory and non-ambulatory individuals with SMA. 'The scale consists of an entry item to establish functional levels and 19 items covering distal to proximal movements. The entry item is a modified version of the Brooke scale, including activities ranging from no functional use of hands (score 0) to full bilateral shoulder abduction (score 6). The entry item does not contribute to the total score but serves as a functional classification of overall upper limb functional ability. Of the remaining 19 items, 18 are scored on a 3 point scoring system and 1 item is scored on a 2 point scoring system. The test is performed unilaterally using the limb preferred by the participant. The total score ranges from 0, if all the items cannot be performed, to 37, if all the activities are achieved fully without any compensation. ' Higher scores indicate higher levels of motor ability.", + "timeFrame": "Baseline, Week 4, Week 13, Week 26, Week 39 and Week 52" + } + ] + }, + "eligibilityModule": { + "eligibilityCriteria": "Inclusion\n\n* Symptomatic SMA diagnosis based on gene mutation analysis with bi-allelic survival motor neuron 1 (SMN1) mutations (deletion or point mutations) and any copy of the survival motor neuron 2 (SMN2) gene.\n* Weight \u2265 8.5 kg and \u2264 21 kg at the time of Screening Visit 2\n* Naive to treatment or have discontinued an approved drug/therapy\n\nExclusion:\n\n* Previous OAV101 use or previous use of any adeno-associated virus serotype 9 (AAV9) gene therapy\n* BMI \\< 3rd percentile\n* Participant with history of aspiration pneumonia or signs of aspiration\n* Elevated anti-AAV9 antibody\n* History of gene therapy, hematopoietic transplantation, or solid organ transplantation\n* Inability to take corticosteroids\n* Concomitant use of immunosuppressive therapy\n* Requiring invasive ventilation, tracheostomy or awake non-invasive ventilation 9. Administration of vaccines 2 weeks prior to infusion of OAV101\n* Awake hypoxemia or awake oxygen saturation level decrease\n* Hepatic dysfunction\n* Presence of a confirmed or suspected infection\n* If previously treated with disease modifying therapy, specified washout times apply\n* Documented any parental consanguinity.", + "healthyVolunteers": false, + "sex": "ALL", + "maximumAge": "17 Years", + "stdAges": ["CHILD"] + }, + "contactsLocationsModule": { + "overallOfficials": [ + { + "name": "Novartis Pharmaceuticals", + "affiliation": "Novartis Pharmaceuticals", + "role": "STUDY_DIRECTOR" + } + ], + "locations": [ + { + "facility": "Novartis Investigative Site", + "city": "Boston", + "state": "Massachusetts", + "zip": "02115", + "country": "United States", + "geoPoint": { "lat": 42.35843, "lon": -71.05977 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Saint Louis", + "state": "Missouri", + "zip": "63110", + "country": "United States", + "geoPoint": { "lat": 38.62727, "lon": -90.19789 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Randwick", + "state": "New South Wales", + "zip": "2031", + "country": "Australia", + "geoPoint": { "lat": -33.91439, "lon": 151.24895 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Leuven", + "zip": "3000", + "country": "Belgium", + "geoPoint": { "lat": 50.87959, "lon": 4.70093 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Montreal", + "state": "Quebec", + "zip": "H4A 3J1", + "country": "Canada", + "geoPoint": { "lat": 45.50884, "lon": -73.58781 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Garches", + "zip": "92380", + "country": "France", + "geoPoint": { "lat": 48.84226, "lon": 2.18232 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Strasbourg", + "zip": "67000", + "country": "France", + "geoPoint": { "lat": 48.58392, "lon": 7.74553 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Roma", + "state": "RM", + "zip": "00168", + "country": "Italy", + "geoPoint": { "lat": 41.89193, "lon": 12.51133 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Lisboa", + "zip": "1600190", + "country": "Portugal", + "geoPoint": { "lat": 38.71667, "lon": -9.13333 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Kaohsiung", + "zip": "80756", + "country": "Taiwan", + "geoPoint": { "lat": 22.61626, "lon": 120.31333 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Taipei", + "zip": "10002", + "country": "Taiwan", + "geoPoint": { "lat": 25.04776, "lon": 121.53185 } + }, + { + "facility": "Novartis Investigative Site", + "city": "London", + "zip": "WC1N 3JH", + "country": "United Kingdom", + "geoPoint": { "lat": 51.50853, "lon": -0.12574 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Newcastle Upon Tyne", + "zip": "NE1 4LP", + "country": "United Kingdom", + "geoPoint": { "lat": 54.97328, "lon": -1.61396 } + } + ] + }, + "referencesModule": { + "seeAlsoLinks": [ + { + "label": "If you have inquiries about possible participation in this clinical study, please go to this website, and select your region or country, to see if there is an investigator site near you.", + "url": "https://medinfo.novartis.com/gene-therapies/" + }, + { + "label": "A Plain Language Trial Summary is available on www.novctrd.com", + "url": "https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=1937" + } + ] + }, + "ipdSharingStatementModule": { + "ipdSharing": "YES", + "description": "Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.\n\nThis trial data availability is according to the criteria and process described on https://www.clinicalstudydatarequest.com/.", + "url": "https://www.clinicalstudydatarequest.com/." + } + }, + "resultsSection": { + "participantFlowModule": { + "preAssignmentDetails": "The screening period began after signature of the study informed consent. The study included a screening period of up to 45 days. On Day -1, participants were admitted to the hospital for pre-treatment baseline procedures. On Day 1, participants received the study treatment.", + "recruitmentDetails": "Participants took part in 13 investigative sites across 9 countries.", + "groups": [ + { + "id": "FG000", + "title": "OAV101 1.1e14 vg/kg 8.5-13 kg", + "description": "8.5-13 kg" + }, + { + "id": "FG001", + "title": "OAV101 1.1e14 vg/kg >13-17 kg", + "description": "\\>13-17 kg" + }, + { + "id": "FG002", + "title": "OAV101 1.1e14 vg/kg >17-21 kg", + "description": "\\>17-21 kg" + } + ], + "periods": [ + { + "title": "Overall Study", + "milestones": [ + { + "type": "STARTED", + "achievements": [ + { "groupId": "FG000", "numSubjects": "7" }, + { "groupId": "FG001", "numSubjects": "8" }, + { "groupId": "FG002", "numSubjects": "9" } + ] + }, + { + "type": "COMPLETED", + "achievements": [ + { "groupId": "FG000", "numSubjects": "7" }, + { "groupId": "FG001", "numSubjects": "8" }, + { "groupId": "FG002", "numSubjects": "9" } + ] + }, + { + "type": "NOT COMPLETED", + "achievements": [ + { "groupId": "FG000", "numSubjects": "0" }, + { "groupId": "FG001", "numSubjects": "0" }, + { "groupId": "FG002", "numSubjects": "0" } + ] + } + ] + } + ] + }, + "baselineCharacteristicsModule": { + "groups": [ + { + "id": "BG000", + "title": "OAV101 1.1e14 vg/kg 8.5-13 kg", + "description": "8.5-13 kg" + }, + { + "id": "BG001", + "title": "OAV101 1.1e14 vg/kg >13-17 kg", + "description": "\\>13-17 kg" + }, + { + "id": "BG002", + "title": "OAV101 1.1e14 vg/kg >17-21 kg", + "description": "\\>17-21 kg" + }, + { + "id": "BG003", + "title": "Total", + "description": "Total of all reporting groups" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "BG000", "value": "7" }, + { "groupId": "BG001", "value": "8" }, + { "groupId": "BG002", "value": "9" }, + { "groupId": "BG003", "value": "24" } + ] + } + ], + "measures": [ + { + "title": "Age, Continuous", + "paramType": "MEAN", + "dispersionType": "STANDARD_DEVIATION", + "unitOfMeasure": "years", + "classes": [ + { + "categories": [ + { + "measurements": [ + { + "groupId": "BG000", + "value": "3.027", + "spread": "1.1458" + }, + { + "groupId": "BG001", + "value": "4.519", + "spread": "1.1769" + }, + { + "groupId": "BG002", + "value": "6.137", + "spread": "1.6018" + }, + { + "groupId": "BG003", + "value": "4.690", + "spread": "1.8240" + } + ] + } + ] + } + ] + }, + { + "title": "Age, Customized", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "title": "0 < 28 days", + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" }, + { "groupId": "BG003", "value": "0" } + ] + } + ] + }, + { + "title": "28 days - < 2 years", + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "1" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" }, + { "groupId": "BG003", "value": "1" } + ] + } + ] + }, + { + "title": "2 years - < 12 years", + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "6" }, + { "groupId": "BG001", "value": "8" }, + { "groupId": "BG002", "value": "9" }, + { "groupId": "BG003", "value": "23" } + ] + } + ] + }, + { + "title": "12 years - <18 years", + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" }, + { "groupId": "BG003", "value": "0" } + ] + } + ] + }, + { + "title": ">= 18 years", + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" }, + { "groupId": "BG003", "value": "0" } + ] + } + ] + } + ] + }, + { + "title": "Sex: Female, Male", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "title": "Female", + "measurements": [ + { "groupId": "BG000", "value": "3" }, + { "groupId": "BG001", "value": "5" }, + { "groupId": "BG002", "value": "4" }, + { "groupId": "BG003", "value": "12" } + ] + }, + { + "title": "Male", + "measurements": [ + { "groupId": "BG000", "value": "4" }, + { "groupId": "BG001", "value": "3" }, + { "groupId": "BG002", "value": "5" }, + { "groupId": "BG003", "value": "12" } + ] + } + ] + } + ] + }, + { + "title": "Race (NIH/OMB)", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "title": "American Indian or Alaska Native", + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" }, + { "groupId": "BG003", "value": "0" } + ] + }, + { + "title": "Asian", + "measurements": [ + { "groupId": "BG000", "value": "1" }, + { "groupId": "BG001", "value": "5" }, + { "groupId": "BG002", "value": "3" }, + { "groupId": "BG003", "value": "9" } + ] + }, + { + "title": "Native Hawaiian or Other Pacific Islander", + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" }, + { "groupId": "BG003", "value": "0" } + ] + }, + { + "title": "Black or African American", + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" }, + { "groupId": "BG003", "value": "0" } + ] + }, + { + "title": "White", + "measurements": [ + { "groupId": "BG000", "value": "6" }, + { "groupId": "BG001", "value": "1" }, + { "groupId": "BG002", "value": "6" }, + { "groupId": "BG003", "value": "13" } + ] + }, + { + "title": "More than one race", + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" }, + { "groupId": "BG003", "value": "0" } + ] + }, + { + "title": "Unknown or Not Reported", + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "2" }, + { "groupId": "BG002", "value": "0" }, + { "groupId": "BG003", "value": "2" } + ] + } + ] + } + ] + } + ] + }, + "outcomeMeasuresModule": { + "outcomeMeasures": [ + { + "type": "PRIMARY", + "title": "Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket", + "description": "An AE is any untoward medical occurrence (e.g. any unfavorable and unintended sign \\[including abnormal laboratory findings\\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study.", + "populationDescription": "Safety set (The Safety Set comprised all participants who were administered investigational drug.)", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "Up to Month 12", + "groups": [ + { + "id": "OG000", + "title": "OAV101 1.1e14 vg/kg 8.5-13 kg", + "description": "8.5-13 kg" + }, + { + "id": "OG001", + "title": "OAV101 1.1e14 vg/kg >13-17 kg", + "description": "\\>13-17 kg" + }, + { + "id": "OG002", + "title": "OAV101 1.1e14 vg/kg >17-21 kg", + "description": "\\>17-21 kg" + }, + { + "id": "OG003", + "title": "OAV101 1.1e14 vg/kg Overall", + "description": "Overall" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "classes": [ + { + "title": "Any treatment-emergent adverse events", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ] + }, + { + "title": "Any treatment-emergent adverse events related to OAV101", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ] + }, + { + "title": "Any severe treatment-emergent adverse events", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "1" }, + { "groupId": "OG001", "value": "4" }, + { "groupId": "OG002", "value": "3" }, + { "groupId": "OG003", "value": "8" } + ] + } + ] + }, + { + "title": "Any serious treatment-emergent adverse events", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "3" }, + { "groupId": "OG001", "value": "7" }, + { "groupId": "OG002", "value": "5" }, + { "groupId": "OG003", "value": "15" } + ] + } + ] + }, + { + "title": "Serious treatment-emergent adverse events related to OAV101", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "1" }, + { "groupId": "OG001", "value": "4" }, + { "groupId": "OG002", "value": "2" }, + { "groupId": "OG003", "value": "7" } + ] + } + ] + }, + { + "title": "Treatment-emergent adverse events leading to study discontinuation", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "0" }, + { "groupId": "OG002", "value": "0" }, + { "groupId": "OG003", "value": "0" } + ] + } + ] + }, + { + "title": "Treatment-emergent adverse events leading to death", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "0" }, + { "groupId": "OG002", "value": "0" }, + { "groupId": "OG003", "value": "0" } + ] + } + ] + }, + { + "title": "Treatment-emergent adverse events of special interest", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "7" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "23" } + ] + } + ] + } + ] + }, + { + "type": "PRIMARY", + "title": "Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket", + "description": "Important identified and important potential risks included the following AESIs: Hepatotoxicity, Thrombocytopenia, Cardiac adverse events, Dorsal root ganglia toxicity and Thrombotic microangiopathy.\n\nThese were assessed by the investigator.", + "populationDescription": "Safety set", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "Up to Month 12", + "groups": [ + { + "id": "OG000", + "title": "OAV101 1.1e14 vg/kg 8.5-13 kg", + "description": "8.5-13 kg" + }, + { + "id": "OG001", + "title": "OAV101 1.1e14 vg/kg >13-17 kg", + "description": "\\>13-17 kg" + }, + { + "id": "OG002", + "title": "OAV101 1.1e14 vg/kg >17-21 kg", + "description": "\\>17-21 kg" + }, + { + "id": "OG003", + "title": "OAV101 1.1e14 vg/kg Overall", + "description": "Overall" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "classes": [ + { + "title": "Hepatotoxicity", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "6" }, + { "groupId": "OG001", "value": "5" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "20" } + ] + } + ] + }, + { + "title": "Transient thrombocytopenia", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "4" }, + { "groupId": "OG001", "value": "6" }, + { "groupId": "OG002", "value": "7" }, + { "groupId": "OG003", "value": "17" } + ] + } + ] + }, + { + "title": "Cardiac adverse events", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "2" }, + { "groupId": "OG002", "value": "1" }, + { "groupId": "OG003", "value": "3" } + ] + } + ] + }, + { + "title": "Thrombotic microangiopathy", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "0" }, + { "groupId": "OG002", "value": "0" }, + { "groupId": "OG003", "value": "0" } + ] + } + ] + }, + { + "title": "Dorsal root ganglia cell inflammation", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "0" }, + { "groupId": "OG002", "value": "0" }, + { "groupId": "OG003", "value": "0" } + ] + } + ] + } + ] + }, + { + "type": "PRIMARY", + "title": "Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure", + "description": "Change from baseline in vital signs measurements - systolic and diastolic blood pressure (mmHg).\n\nSystolic Blood Pressure-Low:\\<=5th percentile of the age(Any Age), High:\\>=90th percentile of the age, gender, and height group (\\<18 yrs).\n\nDiastolic Blood Pressure-High:\\>=90th percentile of the age, gender, and height group(\\<18 yrs).", + "populationDescription": "Safety set", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "12 months", + "groups": [ + { + "id": "OG000", + "title": "OAV101 1.1e14 vg/kg 8.5-13 kg", + "description": "8.5-13 kg" + }, + { + "id": "OG001", + "title": "OAV101 1.1e14 vg/kg >13-17kg", + "description": "\\>13-17kg" + }, + { + "id": "OG002", + "title": "OAV101 1.1e14 vg/kg >17-21kg", + "description": "\\>17-21kg" + }, + { + "id": "OG003", + "title": "OAV101 1.1e14 vg/kg Overall", + "description": "Overall" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "classes": [ + { + "title": "Systolic Blood Pressure (mmHg) Low", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "1" }, + { "groupId": "OG001", "value": "0" }, + { "groupId": "OG002", "value": "1" }, + { "groupId": "OG003", "value": "2" } + ] + } + ] + }, + { + "title": "Systolic Blood Pressure (mmHg) High", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "8" }, + { "groupId": "OG003", "value": "23" } + ] + } + ] + }, + { + "title": "Diastolic Blood Pressure (mmHg) High", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "7" }, + { "groupId": "OG002", "value": "8" }, + { "groupId": "OG003", "value": "22" } + ] + } + ] + } + ] + }, + { + "type": "PRIMARY", + "title": "Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg)", + "populationDescription": "Safety set", + "reportingStatus": "POSTED", + "paramType": "MEAN", + "dispersionType": "Standard Deviation", + "unitOfMeasure": "mmHg", + "timeFrame": "Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52", + "groups": [ + { + "id": "OG000", + "title": "OAV101 1.1e14 vg/kg 8.5-13 kg", + "description": "8.5-13 kg" + }, + { + "id": "OG001", + "title": "OAV101 1.1e14 vg/kg >13-17kg", + "description": "\\>13-17kg" + }, + { + "id": "OG002", + "title": "OAV101 1.1e14 vg/kg >17-21kg", + "description": "\\>17-21kg" + }, + { + "id": "OG003", + "title": "OAV101 1.1e14 vg/kg Overall", + "description": "Overall" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "classes": [ + { + "title": "Change from baseline at Day 2", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "5.3", + "spread": "12.65" + }, + { + "groupId": "OG001", + "value": "-1.3", + "spread": "13.01" + }, + { + "groupId": "OG002", + "value": "0.4", + "spread": "10.36" + }, + { + "groupId": "OG003", + "value": "1.3", + "spread": "11.75" + } + ] + } + ] + }, + { + "title": "Change from baseline at Day 3", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "-2.3", + "spread": "15.01" + }, + { + "groupId": "OG001", + "value": "-5.6", + "spread": "11.16" + }, + { + "groupId": "OG002", + "value": "3.0", + "spread": "8.38" + }, + { + "groupId": "OG003", + "value": "-1.4", + "spread": "11.62" + } + ] + } + ] + }, + { + "title": "Change from baseline at Week 1", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "13.4", + "spread": "10.53" + }, + { + "groupId": "OG001", + "value": "-7.4", + "spread": "15.59" + }, + { + "groupId": "OG002", + "value": "2.0", + "spread": "12.84" + }, + { + "groupId": "OG003", + "value": "2.2", + "spread": "15.18" + } + ] + } + ] + }, + { + "title": "Change from baseline at Week 2", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "11.7", + "spread": "20.33" + }, + { + "groupId": "OG001", + "value": "-3.8", + "spread": "17.60" + }, + { + "groupId": "OG002", + "value": "1.8", + "spread": "9.15" + }, + { + "groupId": "OG003", + "value": "2.8", + "spread": "16.45" + } + ] + } + ] + }, + { + "title": "Change from baseline at Week 3 (n=6,8,9,23)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "6" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "23" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "15.8", + "spread": "8.04" + }, + { + "groupId": "OG001", + "value": "-3.5", + "spread": "12.87" + }, + { + "groupId": "OG002", + "value": "-0.7", + "spread": "7.25" + }, + { + "groupId": "OG003", + "value": "2.7", + "spread": "12.33" + } + ] + } + ] + }, + { + "title": "Change from baseline at Week 4 (n=6,8,8,22)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "6" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "8" }, + { "groupId": "OG003", "value": "22" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "12.3", + "spread": "16.50" + }, + { + "groupId": "OG001", + "value": "-3.5", + "spread": "15.89" + }, + { + "groupId": "OG002", + "value": "6.8", + "spread": "10.02" + }, + { + "groupId": "OG003", + "value": "4.5", + "spread": "15.05" + } + ] + } + ] + }, + { + "title": "Change from baseline at Week 6 (n=7,8,8,23)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "8" }, + { "groupId": "OG003", "value": "23" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "8.1", + "spread": "12.92" + }, + { + "groupId": "OG001", + "value": "-2.8", + "spread": "24.15" + }, + { + "groupId": "OG002", + "value": "5.4", + "spread": "15.39" + }, + { + "groupId": "OG003", + "value": "3.4", + "spread": "18.13" + } + ] + } + ] + }, + { + "title": "Change from baseline at Week 8 (n=7,7,9,23)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "7" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "23" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "3.9", + "spread": "15.49" + }, + { + "groupId": "OG001", + "value": "-0.7", + "spread": "14.57" + }, + { + "groupId": "OG002", + "value": "3.6", + "spread": "12.83" + }, + { + "groupId": "OG003", + "value": "2.3", + "spread": "13.69" + } + ] + } + ] + }, + { + "title": "Change from baseline at Week 10", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "10.0", + "spread": "16.99" + }, + { + "groupId": "OG001", + "value": "-5.6", + "spread": "13.19" + }, + { + "groupId": "OG002", + "value": "4.6", + "spread": "10.06" + }, + { + "groupId": "OG003", + "value": "2.8", + "spread": "14.32" + } + ] + } + ] + }, + { + "title": "Change from baseline at Week 13", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "6.1", + "spread": "10.78" + }, + { + "groupId": "OG001", + "value": "-4.5", + "spread": "17.50" + }, + { + "groupId": "OG002", + "value": "1.4", + "spread": "9.93" + }, + { + "groupId": "OG003", + "value": "0.8", + "spread": "13.28" + } + ] + } + ] + }, + { + "title": "Change from baseline at Week 26", 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"OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "0.1", + "spread": "1.07" + }, + { + "groupId": "OG001", + "value": "-0.4", + "spread": "2.00" + }, + { + "groupId": "OG002", + "value": "-0.8", + "spread": "1.20" + }, + { + "groupId": "OG003", + "value": "-0.4", + "spread": "1.47" + } + ] + } + ] + }, + { + "title": "Change from baseline at Week 2", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "-0.7", + "spread": "1.89" + }, + { + "groupId": "OG001", + "value": "-1.0", + "spread": "1.51" + }, + { + "groupId": "OG002", + "value": "-0.8", + "spread": "2.11" + }, + { + "groupId": 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"categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "0.0", + "spread": "1.91" + }, + { + "groupId": "OG001", + "value": "-0.5", + "spread": "1.31" + }, + { + "groupId": "OG002", + "value": "-0.8", + "spread": "0.89" + }, + { + "groupId": "OG003", + "value": "-0.4", + "spread": "1.38" + } + ] + } + ] + }, + { + "title": "Change from baseline at Week 6 (n=7,8,8,23)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "8" }, + { "groupId": "OG003", "value": "23" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "-0.1", + "spread": "1.21" + }, + { + "groupId": "OG001", + "value": "-1.0", + "spread": "1.51" + }, + { + "groupId": "OG002", + "value": "-0.6", + "spread": "1.19" + }, + { + "groupId": "OG003", + "value": "-0.6", + "spread": "1.31" + } + ] + } + ] + }, + { + "title": "Change from baseline at Week 8 (n=7,7,9,23)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "7" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "23" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "-0.7", + "spread": "1.89" + }, + { + "groupId": "OG001", + "value": "0.0", + "spread": "1.15" + }, + { + "groupId": "OG002", + "value": "-0.4", + "spread": "0.88" + }, + { + "groupId": "OG003", + "value": "-0.4", + "spread": "1.31" + } + ] + } + ] + }, + { + "title": "Change from baseline at Week 10", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "0.3", + "spread": "1.89" + }, + { + "groupId": "OG001", + "value": "-1.0", + "spread": "1.93" + }, + { + "groupId": "OG002", + "value": "-0.7", + "spread": "1.32" + }, + { + "groupId": "OG003", + "value": "-0.5", + "spread": "1.72" + } + ] + } + ] + }, + { + "title": "Change from baseline at Week 13", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "0.1", + "spread": "1.57" + }, + { + "groupId": "OG001", + "value": "-0.5", + "spread": "1.07" + }, + { + "groupId": "OG002", + "value": "-0.4", + "spread": "1.67" + }, + { + "groupId": "OG003", + "value": "-0.3", + "spread": "1.43" + } + ] + } + ] + }, + { + "title": "Change from baseline at Week 26", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "-0.6", + "spread": "1.51" + }, + { + "groupId": "OG001", + "value": "-0.1", + "spread": "1.36" + }, + { + "groupId": "OG002", + "value": "-0.2", + "spread": "1.99" + }, + { + "groupId": "OG003", + "value": "-0.3", + "spread": "1.60" + } + ] + } + ] + }, + { + "title": "Change from baseline at Week 39 (n=7,7,8,22)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "7" }, + { "groupId": "OG002", "value": "8" }, + { "groupId": "OG003", "value": "22" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "-1.0", + "spread": "1.41" + }, + { + "groupId": "OG001", + "value": "0.1", + "spread": "0.90" + }, + { + "groupId": "OG002", + "value": "-0.4", + "spread": "1.19" + }, + { + "groupId": "OG003", + "value": "-0.4", + "spread": "1.22" + } + ] + } + ] + }, + { + "title": "Change from baseline at Week 52", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "-1.0", + "spread": "2.00" + }, + { + "groupId": "OG001", + "value": "-0.6", + "spread": "1.51" + }, + { + "groupId": "OG002", + "value": "-0.1", + "spread": "1.54" + }, + { + "groupId": "OG003", + "value": "-0.5", + "spread": "1.64" + } + ] + } + ] + } + ] + }, + { + "type": "SECONDARY", + "title": "Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development.", + "description": "The World Health Organization-Multicentre Growth Reference Study (WHO-MGRS) and Bayley scale of Infant and Toddler Development was modified and combined into a single scale expressly for this study, to measure developmental motor milestones. These were assessed via the milestone checklist, formed of 10 yes/no questions with optional video documentation. The developmental milestones are: head control, sitting with support, sitting without support, sitting without support for 30 seconds, hands-and-knees crawling, pulls to stand, standing with assistance, walking with assistance, standing alone and walking alone. A yes response indicates that the patient reached a particular development milestone.", + "populationDescription": "Full analysis set", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "Baseline, Week 26 and Week 52", + "groups": [ + { + "id": "OG000", + "title": "OAV101 1.1e14 vg/kg 8.5-13 kg", + "description": "8.5-13 kg" + }, + { + "id": "OG001", + "title": "OAV101 1.1e14 vg/kg >13-17 kg", + "description": "\\>13-17 kg" + }, + { + "id": "OG002", + "title": "OAV101 1.1e14 vg/kg >17-21 kg", + "description": "\\>17-21 kg" + }, + { + "id": "OG003", + "title": "OAV101 1.1e14 vg/kg Overall", + "description": "Overall" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "classes": [ + { + "title": "Baseline Head control (Bayley GM #4)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ] + }, + { + "title": "Baseline Sits with support (Bayley GM #19)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ] + }, + { + "title": "Baseline Sitting without support (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "6" }, + { "groupId": "OG001", "value": "7" }, + { "groupId": "OG002", "value": "8" }, + { "groupId": "OG003", "value": "21" } + ] + } + ] + }, + { + "title": "Baseline Sits without support 30 s (Bayley GM #26)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "6" }, + { "groupId": "OG001", "value": "7" }, + { "groupId": "OG002", "value": "8" }, + { "groupId": "OG003", "value": "21" } + ] + } + ] + }, + { + "title": "Baseline Hands-and-knees crawling (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "1" }, + { "groupId": "OG001", "value": "3" }, + { "groupId": "OG002", "value": "6" }, + { "groupId": "OG003", "value": "10" } + ] + } + ] + }, + { + "title": "Baseline Pulls to stand (Bayley GM #35)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "1" }, + { "groupId": "OG001", "value": "3" }, + { "groupId": "OG002", "value": "5" }, + { "groupId": "OG003", "value": "9" } + ] + } + ] + }, + { + "title": "Baseline Standing with assistance (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "2" }, + { "groupId": "OG002", "value": "5" }, + { "groupId": "OG003", "value": "7" } + ] + } + ] + }, + { + "title": "Baseline Walking with assistance (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "2" }, + { "groupId": "OG002", "value": "5" }, + { "groupId": "OG003", "value": "7" } + ] + } + ] + }, + { + "title": "Baseline Standing alone (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "2" }, + { "groupId": "OG002", "value": "4" }, + { "groupId": "OG003", "value": "6" } + ] + } + ] + }, + { + "title": "Baseline Walking alone (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "2" }, + { "groupId": "OG002", "value": "4" }, + { "groupId": "OG003", "value": "6" } + ] + } + ] + }, + { + "title": "Week 26 Head control (Bayley GM #4)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ] + }, + { + "title": "Week 26 Sits with support (Bayley GM #19)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ] + }, + { + "title": "Week 26 Sitting without support (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "5" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "21" } + ] + } + ] + }, + { + "title": "Week 26 Sits without support 30 s (Bayley GM #26)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "5" }, + { "groupId": "OG002", "value": "8" }, + { "groupId": "OG003", "value": "20" } + ] + } + ] + }, + { + "title": "Week 26 Hands-and-knees crawling (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "2" }, + { "groupId": "OG001", "value": "3" }, + { "groupId": "OG002", "value": "6" }, + { "groupId": "OG003", "value": "11" } + ] + } + ] + }, + { + "title": "Week 26 Pulls to stand (Bayley GM #35)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "2" }, + { "groupId": "OG001", "value": "3" }, + { "groupId": "OG002", "value": "5" }, + { "groupId": "OG003", "value": "10" } + ] + } + ] + }, + { + "title": "Week 26 Standing with assistance (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "1" }, + { "groupId": "OG001", "value": "3" }, + { "groupId": "OG002", "value": "5" }, + { "groupId": "OG003", "value": "9" } + ] + } + ] + }, + { + "title": "Week 26 Walking with assistance (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "1" }, + { "groupId": "OG001", "value": "3" }, + { "groupId": "OG002", "value": "4" }, + { "groupId": "OG003", "value": "8" } + ] + } + ] + }, + { + "title": "Week 26 Standing alone (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "3" }, + { "groupId": "OG002", "value": "4" }, + { "groupId": "OG003", "value": "7" } + ] + } + ] + }, + { + "title": "Week 26 Walking alone (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "2" }, + { "groupId": "OG002", "value": "4" }, + { "groupId": "OG003", "value": "6" } + ] + } + ] + }, + { + "title": "Week 52 Head control (Bayley GM #4)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ] + }, + { + "title": "Week 52 Sits with support (Bayley GM #19)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "7" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "23" } + ] + } + ] + }, + { + "title": "Week 52 Sitting without support (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "7" }, + { "groupId": "OG002", "value": "8" }, + { "groupId": "OG003", "value": "22" } + ] + } + ] + }, + { + "title": "Week 52 Sits without support 30 s (Bayley GM #26)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "6" }, + { "groupId": "OG002", "value": "8" }, + { "groupId": "OG003", "value": "21" } + ] + } + ] + }, + { + "title": "Week 52 Hands-and-knees crawling (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "2" }, + { "groupId": "OG001", "value": "3" }, + { "groupId": "OG002", "value": "6" }, + { "groupId": "OG003", "value": "11" } + ] + } + ] + }, + { + "title": "Week 52 Pulls to stand (Bayley GM #35) (n=7,8,8,23)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "8" }, + { "groupId": "OG003", "value": "23" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "2" }, + { "groupId": "OG001", "value": "3" }, + { "groupId": "OG002", "value": "5" }, + { "groupId": "OG003", "value": "10" } + ] + } + ] + }, + { + "title": "Week 52 Standing with assistance (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "2" }, + { "groupId": "OG001", "value": "3" }, + { "groupId": "OG002", "value": "5" }, + { "groupId": "OG003", "value": "10" } + ] + } + ] + }, + { + "title": "Week 52 Walking with assistance (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "1" }, + { "groupId": "OG001", "value": "3" }, + { "groupId": "OG002", "value": "4" }, + { "groupId": "OG003", "value": "8" } + ] + } + ] + }, + { + "title": "Week 52 Standing alone (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "3" }, + { "groupId": "OG002", "value": "4" }, + { "groupId": "OG003", "value": "7" } + ] + } + ] + }, + { + "title": "Week 52 Walking alone (WHO MGRS)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "7" }, + { "groupId": "OG001", "value": "8" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "24" } + ] + } + ], + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "2" }, + { "groupId": "OG002", "value": "4" }, + { "groupId": "OG003", "value": "6" } + ] + } + ] + } + ] + }, + { + "type": "SECONDARY", + "title": "Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age", + "description": "The HFMSE was devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE is formed of 33 assessments. Each motor skill item is scored on a 3 point Likert scale from 0 (no response) to 2 (full response), with a total score range of 0 to 66. A higher score indicates a higher level of ability.", + "populationDescription": "Participants in the Full Analysis Set (FAS) who had an available value for the outcome measure at the specified timepoints. The FAS includes all participants who were administered investigational drug.", + "reportingStatus": "POSTED", + "paramType": "MEAN", + "dispersionType": "Standard Deviation", + "unitOfMeasure": "HFMSE total score on a scale", + "timeFrame": "Baseline, Week 4, Week 13, Week 26, Week 39 and Week 52", + "groups": [ + { + "id": "OG000", + "title": "OAV101 1.1e14 vg/kg 8.5-13 kg", + "description": "8.5-13 kg" + }, + { + "id": "OG001", + "title": "OAV101 1.1e14 vg/kg >13-17 kg", + "description": "\\>13-17 kg" + }, + { + "id": "OG002", + "title": "OAV101 1.1e14 vg/kg >17-21 kg", + "description": "\\>17-21 kg" + }, + { + "id": "OG003", + "title": "OAV101 1.1e14 vg/kg Overall", + "description": "Overall" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "5" }, + { "groupId": "OG001", "value": "6" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "20" } + ] + } + ], + "classes": [ + { + "title": "Change from baseline at Week 4 (n=4,6,9,19)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "4" }, + { "groupId": "OG001", "value": "6" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "19" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "3.8", + "spread": "2.99" + }, + { + "groupId": "OG001", + "value": "2.3", + "spread": "2.66" + }, + { + "groupId": "OG002", + "value": "3.1", + "spread": "3.10" + }, + { "groupId": "OG003", "value": "3.0", "spread": "2.83" } + ] + } + ] + }, + { + "title": "Change from baseline at Week 13 (n=4,6,9,19)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "4" }, + { "groupId": "OG001", "value": "6" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "19" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "1.3", + "spread": "7.89" + }, + { + "groupId": "OG001", + "value": "4.5", + "spread": "3.99" + }, + { + "groupId": "OG002", + "value": "3.9", + "spread": "3.98" + }, + { "groupId": "OG003", "value": "3.5", "spread": "4.83" } + ] + } + ] + }, + { + "title": "Change from baseline at Week 26 (n=5,6,9,20)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "5" }, + { "groupId": "OG001", "value": "6" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "20" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "3.4", + "spread": "5.18" + }, + { + "groupId": "OG001", + "value": "4.3", + "spread": "5.05" + }, + { + "groupId": "OG002", + "value": "3.1", + "spread": "4.14" + }, + { "groupId": "OG003", "value": "3.6", "spread": "4.45" } + ] + } + ] + }, + { + "title": "Change from baseline at Week 39 (n=5,5,9,19)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "5" }, + { "groupId": "OG001", "value": "5" }, + { "groupId": "OG002", "value": "9" }, + { "groupId": "OG003", "value": "19" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "2.6", + "spread": "6.11" + }, + { + "groupId": "OG001", + "value": "-0.6", + "spread": "4.72" + }, + { + "groupId": "OG002", + "value": "4.3", + "spread": "3.71" + }, + { "groupId": "OG003", "value": "2.6", "spread": "4.87" } + ] + } + ] + }, + { + "title": "Change from baseline at Week 52 (n=5,6,7,18)", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "5" }, + { "groupId": "OG001", "value": "6" }, + { "groupId": "OG002", "value": "7" }, + { "groupId": "OG003", "value": "18" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "3.0", + "spread": "5.24" + }, + { + "groupId": "OG001", + "value": "3.7", + "spread": "5.75" + }, + { + "groupId": "OG002", + "value": "4.3", + "spread": "4.07" + }, + { "groupId": "OG003", "value": "3.7", "spread": "4.73" } + ] + } + ] + } + ] + }, + { + "type": "SECONDARY", + "title": "Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age.", + "description": "The RULM assesses motor performance in the upper limbs from childhood through adulthood in ambulatory and non-ambulatory individuals with SMA. 'The scale consists of an entry item to establish functional levels and 19 items covering distal to proximal movements. The entry item is a modified version of the Brooke scale, including activities ranging from no functional use of hands (score 0) to full bilateral shoulder abduction (score 6). The entry item does not contribute to the total score but serves as a functional classification of overall upper limb functional ability. Of the remaining 19 items, 18 are scored on a 3 point scoring system and 1 item is scored on a 2 point scoring system. The test is performed unilaterally using the limb preferred by the participant. The total score ranges from 0, if all the items cannot be performed, to 37, if all the activities are achieved fully without any compensation. ' Higher scores indicate higher levels of motor ability.", + "populationDescription": "Participants in the Full Analysis Set (FAS) who had an available value for the outcome measure at the specified timepoints. The FAS includes all participants who were administered investigational drug.", + "reportingStatus": "POSTED", + "paramType": "MEAN", + "dispersionType": "Standard Deviation", + "unitOfMeasure": "RULM total score on a scale", + "timeFrame": "Baseline, Week 4, Week 13, Week 26, Week 39 and Week 52", + "groups": [ + { + "id": "OG000", + "title": "OAV101 1.1e14 vg/kg 8.5-13 kg", + "description": "8.5-13 kg" + }, + { + "id": "OG001", + "title": "OAV101 1.1e14 vg/kg >13-17 kg", + "description": "\\>13-17 kg" + }, + { + "id": "OG002", + "title": "OAV101 1.1e14 vg/kg >17-21 kg", + "description": "\\>17-21 kg" + }, + { + "id": "OG003", + "title": "OAV101 1.1e14 vg/kg 8.5-13 kg Overall", + "description": "Overall" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "4" }, + { "groupId": "OG001", "value": "6" }, + { "groupId": "OG002", "value": "8" }, + { "groupId": "OG003", "value": "18" } + ] + } + ], + "classes": [ + { + "title": "Change from 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However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial." + }, + "pointOfContact": { + "title": "Study Director", + "organization": "Novartis Pharmaceuticals", + "email": "Novartis.email@Novartis.com", + "phone": "+ 1 862 778 8300" + } + } + }, + "documentSection": { + "largeDocumentModule": { + "largeDocs": [ + { + "typeAbbrev": "Prot", + "hasProtocol": true, + "hasSap": false, + "hasIcf": false, + "label": "Study Protocol", + "date": "2022-02-28", + "uploadDate": "2023-11-27T14:50", + "filename": "Prot_000.pdf", + "size": 4530450 + }, + { + "typeAbbrev": "SAP", + "hasProtocol": false, + "hasSap": true, + "hasIcf": false, + "label": "Statistical Analysis Plan", + "date": "2023-07-03", + "uploadDate": "2023-11-27T14:50", + "filename": "SAP_001.pdf", + "size": 1532391 + } + ] + } + }, + "derivedSection": { + "miscInfoModule": { + "versionHolder": "2024-12-04", + "removedCountries": ["Switzerland"], + "submissionTracking": { + "firstMcpInfo": { + "postDateStruct": { "date": "2023-12-20", "type": "ACTUAL" } + } + } + }, + "conditionBrowseModule": { + "meshes": [ + { "id": "D009133", "term": "Muscular Atrophy" }, + { "id": "D009134", "term": "Muscular Atrophy, Spinal" }, + { "id": "D001284", "term": "Atrophy" } + ], + "ancestors": [ + { "id": "D020763", "term": "Pathological Conditions, Anatomical" }, + { "id": "D020879", "term": "Neuromuscular Manifestations" }, + { "id": "D009461", "term": "Neurologic Manifestations" }, + { "id": "D009422", "term": "Nervous System Diseases" }, + { "id": "D013118", "term": "Spinal Cord Diseases" }, + { "id": "D002493", "term": "Central Nervous System Diseases" }, + { "id": "D016472", "term": "Motor Neuron Disease" }, + { "id": "D019636", "term": "Neurodegenerative Diseases" }, + { "id": "D009468", "term": "Neuromuscular Diseases" } + ], + "browseLeaves": [ + { "id": "M21265", "name": "Wasting Syndrome", "relevance": "LOW" }, + { "id": "M5363", "name": "Cachexia", "relevance": "LOW" }, + { + "id": "M4589", + "name": "Atrophy", + "asFound": "Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12090", + "name": "Muscular Atrophy", + "asFound": "Muscular Atrophy", + "relevance": "HIGH" + }, + { "id": "M12092", "name": "Muscular Diseases", "relevance": "LOW" }, + { + "id": "M12091", + "name": "Muscular Atrophy, Spinal", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M28216", + "name": "Bulbo-Spinal Atrophy, X-Linked", + "relevance": "LOW" + }, + { + "id": "M22519", + "name": "Pathological Conditions, Anatomical", + "relevance": "LOW" + }, + { + "id": "M22619", + "name": "Neuromuscular Manifestations", + "relevance": "LOW" + }, + { + "id": "M12404", + "name": "Neurologic Manifestations", + "relevance": "LOW" + }, + { + "id": "M15915", + "name": "Spinal Cord Diseases", + "relevance": "LOW" + }, + { + "id": "M5742", + "name": "Central Nervous System Diseases", + "relevance": "LOW" + }, + { + "id": "M18879", + "name": "Motor Neuron Disease", + "relevance": "LOW" + }, + { + "id": "M4024", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + }, + { + "id": "M21558", + "name": "Neurodegenerative Diseases", + "relevance": "LOW" + }, + { + "id": "M12411", + "name": "Neuromuscular Diseases", + "relevance": "LOW" + }, + { "id": "T3215", "name": "Kennedy Disease", "relevance": "LOW" }, + { + "id": "T5342", + "name": "Spinal Muscular Atrophy", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "T349", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + } + ], + "browseBranches": [ + { "abbrev": "BC18", "name": "Nutritional and Metabolic Diseases" }, + { "abbrev": "All", "name": "All Conditions" }, + { "abbrev": "BC23", "name": "Symptoms and General Pathology" }, + { "abbrev": "BC10", "name": "Nervous System Diseases" }, + { "abbrev": "BC05", "name": "Musculoskeletal Diseases" }, + { + "abbrev": "BC16", + "name": "Diseases and Abnormalities at or Before Birth" + }, + { "abbrev": "Rare", "name": "Rare Diseases" } + ] + } + }, + "hasResults": true + }, + { + "protocolSection": { + "identificationModule": { + "nctId": "NCT03381729", + "orgStudyIdInfo": { "id": "AVXS-101-CL-102" }, + "secondaryIdInfos": [ + { "id": "2020-003678-28", "type": "EUDRACT_NUMBER" }, + { "id": "COAV101A12102", "type": "OTHER", "domain": "Novartis" } + ], + "organization": { "fullName": "Novartis", "class": "INDUSTRY" }, + "briefTitle": "Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy", + "officialTitle": "Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting But Non-ambulatory Patients With Spinal Muscular Atrophy", + "acronym": "STRONG" + }, + "statusModule": { + "statusVerifiedDate": "2023-04", + "overallStatus": "TERMINATED", + "whyStopped": "Based upon overall strategic objectives within the broader intrathecal clinical development program, Novartis Gene Therapies decided to terminate the study early.", + "expandedAccessInfo": { "hasExpandedAccess": false }, + "startDateStruct": { "date": "2017-12-14", "type": "ACTUAL" }, + "primaryCompletionDateStruct": { + "date": "2021-11-18", + "type": "ACTUAL" + }, + "completionDateStruct": { "date": "2021-11-18", "type": "ACTUAL" }, + "studyFirstSubmitDate": "2017-12-13", + "studyFirstSubmitQcDate": "2017-12-18", + "studyFirstPostDateStruct": { + "date": "2017-12-22", + "type": "ACTUAL" + }, + "resultsFirstSubmitDate": "2022-05-18", + "resultsFirstSubmitQcDate": "2022-05-18", + "resultsFirstPostDateStruct": { + "date": "2023-02-16", + "type": "ACTUAL" + }, + "lastUpdateSubmitDate": "2023-04-20", + "lastUpdatePostDateStruct": { "date": "2023-04-24", "type": "ACTUAL" } + }, + "sponsorCollaboratorsModule": { + "responsibleParty": { "type": "SPONSOR" }, + "leadSponsor": { + "name": "Novartis Gene Therapies", + "class": "INDUSTRY" + } + }, + "oversightModule": { + "oversightHasDmc": true, + "isFdaRegulatedDrug": true, + "isFdaRegulatedDevice": false + }, + "descriptionModule": { + "briefSummary": "The purpose of this trial is to evaluate the safety and tolerability of intrathecal administration of onasemnogene abeparvovec-xioi in infants and children with Spinal Muscular Atrophy with 3 copies of SMN2 and deletion of SMN1.", + "detailedDescription": "This is a Phase 1, single-dose administration study of infants and children with a genetic diagnosis consistent with spinal muscular atrophy (SMA), bi-allelic deletion of survival motor neuron 1 gene (SMN1) and 3 copies of survival motor neuron 2 gene (SMN2) without the genetic modifier who are able to sit but cannot stand or walk at the time of study entry. Patients will receive onasemnogene abeparvovec-xioi in a dose comparison safety study of two (or three) potential therapeutic doses. Patients will be stratified in two groups, those \u22656 months and \\< 24 months of age at time of dosing and those \u2265 24 months and \\< 60 months of age at time of dosing. At least 15 patients \u2265 6 months and \\< 24 months, and at least 12 patients \u2265 24 \\< 60 months will be enrolled.\n\nThe first cohort will enroll three patients (Cohort 1) \u2265 6 months and \\< 24 months of age who will receive administration of 6.0 \u00d7 1013 vg of onasemnogene abeparvovec-xioi (Dose A). There will be at least a four week interval between the dosing of each patient within the cohort. Novartis Gene Therapies, Inc. will confer with the Data Safety Monitoring Board (DSMB) on all Grade III or higher AEs within approximately 48 hours of awareness that are possibly, probably or definitely related to the study agent before continuing enrollment. Safety data will be reviewed by the DSMB during quarterly meetings; following enrollment of the three patients and based upon the available safety data a decision will be made whether to: a) stop due to toxicity, or b) proceed to Cohort 2 using Dose B.\n\nShould the determination be made to advance to Dose B, three patients \\< 60 months of age will be enrolled (Cohort 2) and will receive administration of 1.2 \u00d7 1014 vg of onasemnogene abeparvovec-xioi (Dose B). Again, there will be at least a four-week interval between dosing of the three patients within the cohort. Based on the available safety data from the three Cohort 2 patients and all of the Cohort 1 patients, the DSMB will decide and document during quarterly meetings whether further four-week intervals between patients dosing is necessary. Novartis Gene Therapies, Inc. will take this recommendation into consideration and will make the final determination whether to persist with four-week intervals between patients dosing going forward; the decision will be communicated to sites and Institutional Review Boards (IRBs) in a formal sponsor letter. Novartis Gene Therapies, Inc. will confer with the DSMB on all Grade III or higher AEs within approximately 48 hours of awareness that are possibly, probably or definitely related to the study agent before continuing enrollment. Safety data will be reviewed by the DSMB during quarterly meetings; following enrollment of the first six patients and based upon available safety data, a decision will be made whether to: a) stop due to toxicity, or b) continue to enroll an additional 21 patients until there are a total of 12 patients \\> 6 months and \\< 24 months and 12 patients \u2265 24 and \\< 60 months that have all received Dose B.\n\nBased upon an ongoing assessment of safety and efficacy data from patients treated with the 1.2 \u00d7 1014 vg dose, an option for testing of a third dose (Dose C), will be considered. If, based on all available data, this is judged to be safe and necessary, three patients \\< 60 months of age will receive Dose C, 2.4 \u00d7 1014 vg administered IT. A meeting of the DSMB will be called to obtain a recommendation on the safety of escalating to a higher dose prior to proceeding. If a decision is made to proceed to testing a higher dose, there will again be a four-week interval between dosing of the first three patients receiving Dose C, as in Cohorts 1 and 2. Safety data will be reviewed by the DSMB during quarterly meetings. Following enrollment of the first three Dose C patients and based upon available safety data, the DSMB will be consulted and a decision will be made whether to: a) stop dosing Dose C due to safety concerns, or b) continue to enroll an additional 21 patients until there are a total of 12 patients \\> 6 months and \\< 24 months and 12 patients \u2265 24 and \\< 60 months that have received Dose C.\n\nPatients from Cohort 3 will be followed for a total of 15 months post-dose. The primary analyses for efficacy will be assessed when all patients reach 12 months post-dose and the primary analyses for safety will be assessed when the last patient of Cohort 3 reaches 15 months post-dose (and database lock will be performed after the last patient reaches 15 months post-dose)." + }, + "conditionsModule": { + "conditions": ["Spinal Muscular Atrophy"], + "keywords": [ + "Gene Transfer", + "Gene Therapy", + "Adeno-associated virus", + "Survival Motor Neuron", + "SMN", + "AAV9" + ] + }, + "designModule": { + "studyType": "INTERVENTIONAL", + "phases": ["PHASE1"], + "designInfo": { + "allocation": "NON_RANDOMIZED", + "interventionModel": "PARALLEL", + "primaryPurpose": "TREATMENT", + "maskingInfo": { "masking": "NONE" } + }, + "enrollmentInfo": { "count": 32, "type": "ACTUAL" } + }, + "armsInterventionsModule": { + "armGroups": [ + { + "label": "Dose A", + "type": "EXPERIMENTAL", + "description": "Intrathecal administration 6.0 X 10\\^13 vg of onasemnogene abeparvovec-xioi", + "interventionNames": ["Biological: Onasemnogene Abeparvovec-xioi"] + }, + { + "label": "Dose B", + "type": "EXPERIMENTAL", + "description": "Intrathecal administration 1.2 X 10\\^14 vg of onasemnogene abeparvovec-xioi", + "interventionNames": ["Biological: Onasemnogene Abeparvovec-xioi"] + }, + { + "label": "Dose C", + "type": "EXPERIMENTAL", + "description": "Intrathecal administration 2.4 X 10\\^14 vg of onasemnogene abeparvovec-xioi", + "interventionNames": ["Biological: Onasemnogene Abeparvovec-xioi"] + } + ], + "interventions": [ + { + "type": "BIOLOGICAL", + "name": "Onasemnogene Abeparvovec-xioi", + "description": "Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-\u03b2-actin promoter", + "armGroupLabels": ["Dose A", "Dose B", "Dose C"], + "otherNames": ["Zolgensma"] + } + ] + }, + "outcomesModule": { + "primaryOutcomes": [ + { + "measure": "Age 6 to <24 Months Only: Number of Participants Who Achieved the Ability to Stand Alone", + "description": "Defined by the Bayley Scales of Infant and Toddler Development (BSID) Gross Motor (GM) subtest performance criteria number 40, confirmed by video recording, as a participant who stands alone for at least 3 seconds unsupported.", + "timeFrame": "From Day 1 up to Month 12" + }, + { + "measure": "Age 24 to <60 Months Only: Change From Baseline in Hammersmith Functional Motor Scale-Expanded (HFMSE) Score at Month 12", + "description": "The HFMSE contained 33 items which were scored on a scale of 0-2 with a total achievable score ranging from 0, if all activities are failed, to 66, if all the activities are achieved. A positive change from baseline indicates a better outcome.", + "timeFrame": "Baseline and Month 12" + }, + { + "measure": "Number of Participants Who Experienced a Treatment-emergent Adverse Event (TEAE)", + "description": "A TEAE was defined as any event that began or worsened in severity on or after the administration of AVXS-101 through the last study visit.\n\nEvaluation of TEAEs included the number of participants with at least one:\n\n* TEAE\n* Serious TEAE\n* TEAE related to AVXS-101\n* TEAE with Common Terminology Criteria for Adverse Events (CTCAE) grade \u2265 3 (grade 3 = severe or medically significant to grade 5 = death related to TEAE)", + "timeFrame": "Adverse events were collected from the single dose of study treatment until the end of study visit (12 months for Cohort 1 and 2 and 15 months for Cohort 3)" + } + ], + "secondaryOutcomes": [ + { + "measure": "Number of Participants Who Achieved the Ability to Walk Alone", + "description": "Defined by the BSID GM subtest performance criteria number 43, confirmed by video recording, as a participant who takes 5 coordinated independent steps.", + "timeFrame": "From Day 1 up to Month 12" + }, + { + "measure": "Average Number of Hours Per Day of Non-invasive Ventilatory Support", + "description": "Participants were assessed by a pulmonologist and may have been fitted with a non-invasive positive pressure ventilatory (e.g., Bilevel Positive Airway Pressure BiPAP) at the discretion of the pulmonologist and/or investigator. The number of hours per day of non-invasive ventilatory support was captured continuously by the device.", + "timeFrame": "Months 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, and 12" + } + ] + }, + "eligibilityModule": { + "eligibilityCriteria": "Key Inclusion Criteria\n\n* Patients \u22656 months and up to 60 months (1800 days) of age at time of dosing following diagnostic confirmation during screening period by genotype who demonstrate the ability to sit unassisted for 10 or more seconds but cannot stand or walk\n* Diagnostic confirmation by genotype includes lab documentation of homozygous absence of SMN1 exon 7; with exactly three copies of SMN2\n* Negative gene testing for SMN2 gene modifier mutation (c.859G\\>C)\n* Onset of clinical signs and symptoms consistent with spinal muscular atrophy (SMA) at \\< 12 months of age\n* Able to sit independently and not standing or walking independently. Definition of sitting independently is defined by the World Health Organization Multicentre Growth Reference Study (WHO-MGRS) criteria of being able to sit up unsupported with head erect for at least 10 seconds. Child should not use arms or hands to balance body or support position (Wijnhoven 2004)\n* Be up-to-date on childhood vaccines that include palivizumab prophylaxis (also known as Synagis) to prevent respiratory syncytial virus (RSV) infections are also recommended in accordance with American Academy of Pediatrics (AAP 2009)\n\nKey Exclusion Criteria\n\n* Current or historical ability to stand or walk independently\n* Contraindications for spinal tap procedure or administration of intrathecal therapy or presence of an implanted shunt for the drainage of CSF or an implanted central venous (CNS) catheter\n* Severe contractures as determined by designated Physical Therapist(s) at screening that interfere with either the ability to attain/demonstrate functional measures or interferes with ability to receive intrathecal (IT) dosing\n* Severe scoliosis (defined as \u2265 50\u00b0 curvature of spine) evident on X-ray examination\n* Previous, planned or expected scoliosis repair surgery/procedure within 1 year of dose administration\n* Use of invasive ventilatory support (tracheotomy with positive pressure) or pulse oximetry \\< 95% saturation at screening while the patient is awake, or for high altitudes \\> 1000 m, oxygen saturation \\< 92% while the patient is awake\n* Pulse oximetry saturation must not decrease \u2265 four (4) percentage points between screening and highest value on day of dosing\n* Use or requirement of non-invasive ventilatory support for 12 or more hours daily over the two (2) weeks prior to dosing\n* Medical necessity for a gastric feeding tube, where the majority of feedings are given by non-oral methods (i.e., nasogastric tube or nasojejunal tube) or patients whose weight-for-age falls below the 3rd percentile based on WHO Child Growth Standards (Onis 2006). Placement of a permanent gastrostomy prior to screening is not an exclusion\n* Use or requirement of non-invasive ventilatory support for 12 or more hours daily over the two (2) weeks prior to dosing\n* Medical necessity for a gastric feeding tube, where the majority of feedings are given by non-oral methods or patients whose weight-for-age falls below the 3rd percentile based on WHO Child Growth Standards (Onis 2006). Placement of a permanent gastrostomy prior to screening is not an exclusion\n* Active viral infection (includes human immunodeficiency virus (HIV) or serology positive for hepatitis B or C, or Zika virus)\n* Serious non-respiratory tract illness requiring systemic treatment and/or hospitalization within two (2) weeks prior to study entry\n* Respiratory infection requiring medical attention, medical intervention or increase in supportive care of any manner within four (4) weeks prior to study entry\n* Severe non-pulmonary/respiratory tract infection within four (4) weeks before study dosing or concomitant illness that in the opinion of the Principal Investigator (PI) creates unnecessary risks for gene transfer such as:\n\n * Major renal or hepatic impairment\n * Known seizure disorder\n * Diabetes mellitus\n * Idiopathic hypocalciuria\n * Symptomatic cardiomyopathy\n* History of bacterial meningitis or brain or spinal cord disease, including tumors, or abnormalities by magnetic resonance imaging (MRI) or computerized tomography (CT) that would interfere with the lumbar puncture (LP) procedures or CSF circulation\n* Known allergy or hypersensitivity to prednisolone or other glucocorticosteroids or their excipients\n* Known allergy or hypersensitivity to iodine or iodine-containing products\n* Concomitant use of any of the following: drugs for treatment of myopathy or neuropathy, agents used to treat diabetes mellitus, or ongoing immunosuppressive therapy, plasmapheresis, immunomodulators such as adalimumab, or immunosuppressive therapy within 3 months of study dosing\n* Inability to withhold use of laxatives or diuretics in the 24 hours prior to dose administration\n* Anti-AAV9 antibody titers \\>1:50 as determined by Enzyme-linked Immunosorbent Assay (ELISA) binding immunoassay\n\n * Should a potential patient demonstrate anti AAV9 antibody titer \\> 1:50, he or she may receive retesting within 30 days of the screening period and will be eligible to participate if the anti AAV9 antibody titer upon retesting is \u2264 1:50\n* Clinically significant abnormal laboratory values (GGT, ALT, and AST, or total bilirubin \\> 2 \u00d7 ULN, creatinine \u2265 1.0 mg/dL, hemoglobin \\[Hgb\\] \\< 8 or \\> 18 g/dL; white blood cell \\[WBC\\] \\> 20,000 per cmm) prior to gene replacement therapy. Patients with an elevated bilirubin level that is unequivocally the result of neonatal jaundice shall not be excluded\n* Participation in recent SMA treatment clinical trial or receipt of an investigational or approved compound product or therapy received with the intent to treat SMA at any time prior to screening for this study\n\n * Oral beta agonists must be discontinued 30 days prior to dosing.\n * Inhaled albuterol specifically prescribed for the purposes of respiratory (bronchodilator) management is acceptable and not a contraindication at any time prior to screening for this study\n* Expectation of major surgical procedures during the 1-year study assessment period", + "healthyVolunteers": false, + "sex": "ALL", + "minimumAge": "6 Months", + "maximumAge": "60 Months", + "stdAges": ["CHILD"] + }, + "contactsLocationsModule": { + "locations": [ + { + "facility": "UCLA", + "city": "Los Angeles", + "state": "California", + "zip": "90095", + "country": "United States", + "geoPoint": { "lat": 34.05223, "lon": -118.24368 } + }, + { + "facility": "Stanford University", + "city": "Stanford", + "state": "California", + "zip": "94305", + "country": "United States", + "geoPoint": { "lat": 37.42411, "lon": -122.16608 } + }, + { + "facility": "Nemours Children's Hospital", + "city": "Orlando", + "state": "Florida", + "zip": "32827", + "country": "United States", + "geoPoint": { "lat": 28.53834, "lon": -81.37924 } + }, + { + "facility": "Ann & Robert H. Lurie Children's Hospital", + "city": "Chicago", + "state": "Illinois", + "zip": "60611", + "country": "United States", + "geoPoint": { "lat": 41.85003, "lon": -87.65005 } + }, + { + "facility": "Johns Hopkins", + "city": "Baltimore", + "state": "Maryland", + "zip": "21287", + "country": "United States", + "geoPoint": { "lat": 39.29038, "lon": -76.61219 } + }, + { + "facility": "Boston Children's Hospital", + "city": "Boston", + "state": "Massachusetts", + "zip": "02115", + "country": "United States", + "geoPoint": { "lat": 42.35843, "lon": -71.05977 } + }, + { + "facility": "Washington University", + "city": "Saint Louis", + "state": "Missouri", + "zip": "63130", + "country": "United States", + "geoPoint": { "lat": 38.62727, "lon": -90.19789 } + }, + { + "facility": "Nationwide Children's Hospital", + "city": "Columbus", + "state": "Ohio", + "zip": "43205", + "country": "United States", + "geoPoint": { "lat": 39.96118, "lon": -82.99879 } + }, + { + "facility": "Children's Hospital of Philadelphia", + "city": "Philadelphia", + "state": "Pennsylvania", + "zip": "19104", + "country": "United States", + "geoPoint": { "lat": 39.95233, "lon": -75.16379 } + }, + { + "facility": "UT Southwestern", + "city": "Dallas", + "state": "Texas", + "zip": "75390", + "country": "United States", + "geoPoint": { "lat": 32.78306, "lon": -96.80667 } + }, + { + "facility": "University of Utah", + "city": "Salt Lake City", + "state": "Utah", + "zip": "84112", + "country": "United States", + "geoPoint": { "lat": 40.76078, "lon": -111.89105 } + } + ] + }, + "referencesModule": { + "references": [ + { + "pmid": "25516063", + "type": "BACKGROUND", + "citation": "Duque SI, Arnold WD, Odermatt P, Li X, Porensky PN, Schmelzer L, Meyer K, Kolb SJ, Schumperli D, Kaspar BK, Burghes AH. A large animal model of spinal muscular atrophy and correction of phenotype. Ann Neurol. 2015 Mar;77(3):399-414. doi: 10.1002/ana.24332. Epub 2015 Feb 9." + }, + { + "pmid": "20190738", + "type": "BACKGROUND", + "citation": "Foust KD, Wang X, McGovern VL, Braun L, Bevan AK, Haidet AM, Le TT, Morales PR, Rich MM, Burghes AH, Kaspar BK. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat Biotechnol. 2010 Mar;28(3):271-4. doi: 10.1038/nbt.1610. Epub 2010 Feb 28.", + "retractions": [ + { + "pmid": "36203015", + "source": "Nat Biotechnol. 2022 Nov;40(11):1692" + } + ] + }, + { + "pmid": "25358252", + "type": "BACKGROUND", + "citation": "Meyer K, Ferraiuolo L, Schmelzer L, Braun L, McGovern V, Likhite S, Michels O, Govoni A, Fitzgerald J, Morales P, Foust KD, Mendell JR, Burghes AH, Kaspar BK. Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates. Mol Ther. 2015 Mar;23(3):477-87. doi: 10.1038/mt.2014.210. Epub 2014 Oct 31." + } + ], + "seeAlsoLinks": [ + { + "label": "The Novartis Clinical Trial Results", + "url": "https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=17933" + } + ] + } + }, + "resultsSection": { + "participantFlowModule": { + "preAssignmentDetails": "A total of 38 participants were screened, of which 6 were screen failures and 32 were enrolled and received study drug.", + "recruitmentDetails": "A total of 32 participants took part in the trial at 11 sites in the United States between December 2017 and May 2021.", + "groups": [ + { + "id": "FG000", + "title": "Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 6.0E13 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "FG001", + "title": "Cohort 2: 1.2E14 vg - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 1.2E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "FG002", + "title": "Cohort 2: 1.2E14 vg - Age 24 to <60 Months", + "description": "Participants aged 24 to \\<60 months at time of AVXS-101 dosing received a single 1.2E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "FG003", + "title": "Cohort 3: 2.4E14 vg - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 2.4E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "FG004", + "title": "Cohort 3: 2.4E14 vg - Age 24 to <60 Months", + "description": "Participants aged 24 to \\<60 months at time of AVXS-101 dosing were planned to receive a single 2.4E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants were also planned to receive daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could have been tapered downwards. At week 9, prednisolone could have been discontinued. Due to early termination of the study, no participants aged 24 to \\<60 months were enrolled in Cohort 3." + } + ], + "periods": [ + { + "title": "Overall Study", + "milestones": [ + { + "type": "STARTED", + "achievements": [ + { "groupId": "FG000", "numSubjects": "3" }, + { "groupId": "FG001", "numSubjects": "13" }, + { "groupId": "FG002", "numSubjects": "12" }, + { "groupId": "FG003", "numSubjects": "4" }, + { "groupId": "FG004", "numSubjects": "0" } + ] + }, + { + "type": "Received AVXS-101", + "achievements": [ + { "groupId": "FG000", "numSubjects": "3" }, + { "groupId": "FG001", "numSubjects": "13" }, + { "groupId": "FG002", "numSubjects": "12" }, + { "groupId": "FG003", "numSubjects": "4" }, + { "groupId": "FG004", "numSubjects": "0" } + ] + }, + { + "type": "COMPLETED", + "achievements": [ + { "groupId": "FG000", "numSubjects": "3" }, + { "groupId": "FG001", "numSubjects": "13" }, + { "groupId": "FG002", "numSubjects": "12" }, + { "groupId": "FG003", "numSubjects": "4" }, + { "groupId": "FG004", "numSubjects": "0" } + ] + }, + { + "type": "NOT COMPLETED", + "achievements": [ + { "groupId": "FG000", "numSubjects": "0" }, + { "groupId": "FG001", "numSubjects": "0" }, + { "groupId": "FG002", "numSubjects": "0" }, + { "groupId": "FG003", "numSubjects": "0" }, + { "groupId": "FG004", "numSubjects": "0" } + ] + } + ] + } + ] + }, + "baselineCharacteristicsModule": { + "populationDescription": "Safety Analysis Set: All participants given an AVXS-101 intrathecal injection. Participants were analyzed according to actual dose received.", + "groups": [ + { + "id": "BG000", + "title": "Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 6.0E13 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "BG001", + "title": "Cohort 2: 1.2E14 vg - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 1.2E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "BG002", + "title": "Cohort 2: 1.2E14 vg - Age 24 to <60 Months", + "description": "Participants aged 24 to \\<60 months at time of AVXS-101 dosing received a single 1.2E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "BG003", + "title": "Cohort 3: 2.4E14 vg - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 2.4E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "BG004", + "title": "Total", + "description": "Total of all reporting groups" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "BG000", "value": "3" }, + { "groupId": "BG001", "value": "13" }, + { "groupId": "BG002", "value": "12" }, + { "groupId": "BG003", "value": "4" }, + { "groupId": "BG004", "value": "32" } + ] + } + ], + "measures": [ + { + "title": "Age, Categorical", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "title": "<=18 years", + "measurements": [ + { "groupId": "BG000", "value": "3" }, + { "groupId": "BG001", "value": "13" }, + { "groupId": "BG002", "value": "12" }, + { "groupId": "BG003", "value": "4" }, + { "groupId": "BG004", "value": "32" } + ] + }, + { + "title": "Between 18 and 65 years", + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" }, + { "groupId": "BG003", "value": "0" }, + { "groupId": "BG004", "value": "0" } + ] + }, + { + "title": ">=65 years", + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" }, + { "groupId": "BG003", "value": "0" }, + { "groupId": "BG004", "value": "0" } + ] + } + ] + } + ] + }, + { + "title": "Age, Continuous", + "paramType": "MEAN", + "dispersionType": "STANDARD_DEVIATION", + "unitOfMeasure": "months", + "classes": [ + { + "categories": [ + { + "measurements": [ + { + "groupId": "BG000", + "value": "17.2", + "spread": "4.1" + }, + { + "groupId": "BG001", + "value": "16.7", + "spread": "4.5" + }, + { + "groupId": "BG002", + "value": "37.5", + "spread": "10.6" + }, + { + "groupId": "BG003", + "value": "16.9", + "spread": "5.6" + }, + { + "groupId": "BG004", + "value": "24.6", + "spread": "12.5" + } + ] + } + ] + } + ] + }, + { + "title": "Sex: Female, Male", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "title": "Female", + "measurements": [ + { "groupId": "BG000", "value": "2" }, + { "groupId": "BG001", "value": "6" }, + { "groupId": "BG002", "value": "6" }, + { "groupId": "BG003", "value": "0" }, + { "groupId": "BG004", "value": "14" } + ] + }, + { + "title": "Male", + "measurements": [ + { "groupId": "BG000", "value": "1" }, + { "groupId": "BG001", "value": "7" }, + { "groupId": "BG002", "value": "6" }, + { "groupId": "BG003", "value": "4" }, + { "groupId": "BG004", "value": "18" } + ] + } + ] + } + ] + }, + { + "title": "Ethnicity (NIH/OMB)", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "title": "Hispanic or Latino", + "measurements": [ + { "groupId": "BG000", "value": "2" }, + { "groupId": "BG001", "value": "3" }, + { "groupId": "BG002", "value": "0" }, + { "groupId": "BG003", "value": "0" }, + { "groupId": "BG004", "value": "5" } + ] + }, + { + "title": "Not Hispanic or Latino", + "measurements": [ + { "groupId": "BG000", "value": "1" }, + { "groupId": "BG001", "value": "10" }, + { "groupId": "BG002", "value": "12" }, + { "groupId": "BG003", "value": "4" }, + { "groupId": "BG004", "value": "27" } + ] + }, + { + "title": "Unknown or Not Reported", + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" }, + { "groupId": "BG003", "value": "0" }, + { "groupId": "BG004", "value": "0" } + ] + } + ] + } + ] + }, + { + "title": "Race/Ethnicity, Customized", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "title": "White", + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "2" }, + { "groupId": "BG001", "value": "10" }, + { "groupId": "BG002", "value": "8" }, + { "groupId": "BG003", "value": "3" }, + { "groupId": "BG004", "value": "23" } + ] + } + ] + }, + { + "title": "Asian", + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "1" }, + { "groupId": "BG002", "value": "4" }, + { "groupId": "BG003", "value": "1" }, + { "groupId": "BG004", "value": "6" } + ] + } + ] + }, + { + "title": "Other", + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "1" }, + { "groupId": "BG002", "value": "0" }, + { "groupId": "BG003", "value": "0" }, + { "groupId": "BG004", "value": "1" } + ] + } + ] + }, + { + "title": "Multiple", + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "1" }, + { "groupId": "BG001", "value": "1" }, + { "groupId": "BG002", "value": "0" }, + { "groupId": "BG003", "value": "0" }, + { "groupId": "BG004", "value": "2" } + ] + } + ] + } + ] + } + ] + }, + "outcomeMeasuresModule": { + "outcomeMeasures": [ + { + "type": "PRIMARY", + "title": "Age 6 to <24 Months Only: Number of Participants Who Achieved the Ability to Stand Alone", + "description": "Defined by the Bayley Scales of Infant and Toddler Development (BSID) Gross Motor (GM) subtest performance criteria number 40, confirmed by video recording, as a participant who stands alone for at least 3 seconds unsupported.", + "populationDescription": "Intent-to-Treat (ITT) Set: All enrolled participants who were given an AVXS-101 intrathecal injection. Participants were analyzed according to the assigned dose. Data were only collected in participants aged 6 to \\<24 months at time of dosing.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "From Day 1 up to Month 12", + "groups": [ + { + "id": "OG000", + "title": "Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 6.0E13 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "OG001", + "title": "Cohort 2: 1.2E14 vg - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 1.2E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "OG002", + "title": "Cohort 3: 2.4E14 vg - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 2.4E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "3" }, + { "groupId": "OG001", "value": "13" }, + { "groupId": "OG002", "value": "4" } + ] + } + ], + "classes": [ + { + "categories": [ + { + "title": "Able to Stand Alone", + "measurements": [ + { "groupId": "OG000", "value": "1" }, + { "groupId": "OG001", "value": "1" }, + { "groupId": "OG002", "value": "0" } + ] + }, + { + "title": "Unable to Stand Alone", + "measurements": [ + { "groupId": "OG000", "value": "2" }, + { "groupId": "OG001", "value": "12" }, + { "groupId": "OG002", "value": "4" } + ] + } + ] + } + ], + "analyses": [ + { + "groupIds": ["OG001"], + "groupDescription": "Difference in the percentage of participants who achieved the ability to stand alone.", + "nonInferiorityType": "SUPERIORITY", + "pValue": ">0.9999", + "statisticalMethod": "Fisher Exact", + "paramType": "Difference in Percent", + "paramValue": "-6.0", + "ciPctValue": "95", + "ciNumSides": "TWO_SIDED", + "ciLowerLimit": "-21.8", + "ciUpperLimit": "22.8", + "otherAnalysisDescription": "Data for the current study were compared to historical control data (Finkel et al 2014 - PubMed 25080519) where 7 (13.7%) participants achieved the ability to stand alone and 44 (86.3%) participants did not achieve the ability to stand alone." + } + ] + }, + { + "type": "PRIMARY", + "title": "Age 24 to <60 Months Only: Change From Baseline in Hammersmith Functional Motor Scale-Expanded (HFMSE) Score at Month 12", + "description": "The HFMSE contained 33 items which were scored on a scale of 0-2 with a total achievable score ranging from 0, if all activities are failed, to 66, if all the activities are achieved. A positive change from baseline indicates a better outcome.", + "populationDescription": "ITT Set: All enrolled participants who were given an AVXS-101 intrathecal injection. Participants were analyzed according to the assigned dose. Data were only collected in participants aged 24 to \\<60 months at time of dosing.", + "reportingStatus": "POSTED", + "paramType": "LEAST_SQUARES_MEAN", + "dispersionType": "95% Confidence Interval", + "unitOfMeasure": "score on a scale", + "timeFrame": "Baseline and Month 12", + "groups": [ + { + "id": "OG000", + "title": "Cohort 2: 1.2E14 vg - Age 24 to <60 Months", + "description": "Participants aged 24 to \\<60 months at time of AVXS-101 dosing received a single 1.2E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "12" }] + } + ], + "classes": [ + { + "categories": [ + { + "measurements": [ + { + "groupId": "OG000", + "value": "6.0", + "lowerLimit": "3.7", + "upperLimit": "8.3" + } + ] + } + ] + } + ], + "analyses": [ + { + "groupIds": ["OG000"], + "nonInferiorityType": "SUPERIORITY", + "pValue": "0.0027", + "statisticalMethod": "Mixed-Model Repeat Measure", + "paramType": "Difference Between Least Squares Mean", + "paramValue": "5.5", + "ciPctValue": "95", + "ciNumSides": "TWO_SIDED", + "ciLowerLimit": "1.9", + "ciUpperLimit": "9.0", + "otherAnalysisDescription": "Data for the current study were compared to historical control data (Finkel et al 2014 - PubMed 25080519) where the least squares mean (95% confidence interval) of the change from baseline in HFMSE scores at 12 months was 0.5 (-2.2 to 3.2)." + } + ] + }, + { + "type": "PRIMARY", + "title": "Number of Participants Who Experienced a Treatment-emergent Adverse Event (TEAE)", + "description": "A TEAE was defined as any event that began or worsened in severity on or after the administration of AVXS-101 through the last study visit.\n\nEvaluation of TEAEs included the number of participants with at least one:\n\n* TEAE\n* Serious TEAE\n* TEAE related to AVXS-101\n* TEAE with Common Terminology Criteria for Adverse Events (CTCAE) grade \u2265 3 (grade 3 = severe or medically significant to grade 5 = death related to TEAE)", + "populationDescription": "Safety Analysis Set: All participants given an AVXS-101 intrathecal injection. Participants were analyzed according to actual dose received.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "Adverse events were collected from the single dose of study treatment until the end of study visit (12 months for Cohort 1 and 2 and 15 months for Cohort 3)", + "groups": [ + { + "id": "OG000", + "title": "Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 6.0E13 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "OG001", + "title": "Cohort 2: 1.2E14 vg - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 1.2E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "OG002", + "title": "Cohort 2: 1.2E14 vg - Age 24 to <60 Months", + "description": "Participants aged 24 to \\<60 months at time of AVXS-101 dosing received a single 1.2E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "OG003", + "title": "Cohort 3: 2.4E14 vg - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 2.4E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "3" }, + { "groupId": "OG001", "value": "13" }, + { "groupId": "OG002", "value": "12" }, + { "groupId": "OG003", "value": "4" } + ] + } + ], + "classes": [ + { + "title": "TEAE", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "3" }, + { "groupId": "OG001", "value": "13" }, + { "groupId": "OG002", "value": "12" }, + { "groupId": "OG003", "value": "4" } + ] + } + ] + }, + { + "title": "Serious TEAE", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "1" }, + { "groupId": "OG001", "value": "2" }, + { "groupId": "OG002", "value": "4" }, + { "groupId": "OG003", "value": "0" } + ] + } + ] + }, + { + "title": "TEAE Related to AVXS-101", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "7" }, + { "groupId": "OG002", "value": "4" }, + { "groupId": "OG003", "value": "1" } + ] + } + ] + }, + { + "title": "TEAE with CTCAE Grade \u2265 3", + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "1" }, + { "groupId": "OG001", "value": "4" }, + { "groupId": "OG002", "value": "4" }, + { "groupId": "OG003", "value": "0" } + ] + } + ] + } + ] + }, + { + "type": "SECONDARY", + "title": "Number of Participants Who Achieved the Ability to Walk Alone", + "description": "Defined by the BSID GM subtest performance criteria number 43, confirmed by video recording, as a participant who takes 5 coordinated independent steps.", + "populationDescription": "ITT Set: All enrolled participants who were given an AVXS-101 intrathecal injection. Participants were analyzed according to the assigned dose.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "From Day 1 up to Month 12", + "groups": [ + { + "id": "OG000", + "title": "Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 6.0E13 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "OG001", + "title": "Cohort 2: 1.2E14 vg - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 1.2E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "OG002", + "title": "Cohort 2: 1.2E14 vg - Age 24 to <60 Months", + "description": "Participants aged 24 to \\<60 months at time of AVXS-101 dosing received a single 1.2E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "OG003", + "title": "Cohort 3: 2.4E14 vg - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 2.4E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "3" }, + { "groupId": "OG001", "value": "13" }, + { "groupId": "OG002", "value": "12" }, + { "groupId": "OG003", "value": "4" } + ] + } + ], + "classes": [ + { + "categories": [ + { + "title": "Able to Walk Alone", + "measurements": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "1" }, + { "groupId": "OG002", "value": "0" }, + { "groupId": "OG003", "value": "0" } + ] + }, + { + "title": "Unable to Walk Alone", + "measurements": [ + { "groupId": "OG000", "value": "3" }, + { "groupId": "OG001", "value": "12" }, + { "groupId": "OG002", "value": "12" }, + { "groupId": "OG003", "value": "4" } + ] + } + ] + } + ], + "analyses": [ + { + "groupIds": ["OG001"], + "groupDescription": "Difference in the percentage of participants who achieved the ability to walk alone.", + "nonInferiorityType": "SUPERIORITY", + "pValue": ">0.9999", + "statisticalMethod": "Fisher Exact", + "paramType": "Percentage Difference", + "paramValue": "-2.1", + "ciPctValue": "95", + "ciNumSides": "TWO_SIDED", + "ciLowerLimit": "-17.2", + "ciUpperLimit": "27.0", + "otherAnalysisDescription": "Data for the current study were compared to historical control data (Finkel et al 2014 PubMed 25080519) where 5 (9.8%) participants achieved the ability to walk alone and 46 (90.2%) participants did not achieve the ability to walk alone." + } + ] + }, + { + "type": "SECONDARY", + "title": "Average Number of Hours Per Day of Non-invasive Ventilatory Support", + "description": "Participants were assessed by a pulmonologist and may have been fitted with a non-invasive positive pressure ventilatory (e.g., Bilevel Positive Airway Pressure BiPAP) at the discretion of the pulmonologist and/or investigator. The number of hours per day of non-invasive ventilatory support was captured continuously by the device.", + "populationDescription": "Data were only collected in participants requiring BiPAP support.", + "reportingStatus": "POSTED", + "paramType": "MEAN", + "dispersionType": "Standard Deviation", + "unitOfMeasure": "hours/day", + "timeFrame": "Months 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, and 12", + "groups": [ + { + "id": "OG000", + "title": "Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 6.0E13 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "OG001", + "title": "Cohort 2: 1.2E14 vg - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 1.2E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "OG002", + "title": "Cohort 2: 1.2E14 vg - Age 24 to <60 Months", + "description": "Participants aged 24 to \\<60 months at time of AVXS-101 dosing received a single 1.2E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "OG003", + "title": "Cohort 3: 2.4E14 vg - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 2.4E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "1" }, + { "groupId": "OG002", "value": "1" }, + { "groupId": "OG003", "value": "0" } + ] + } + ], + "classes": [ + { + "title": "Month 2", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "0" }, + { "groupId": "OG002", "value": "1" }, + { "groupId": "OG003", "value": "0" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG002", + "value": "10.530", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + } + ] + } + ] + }, + { + "title": "Month 3", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "0" }, + { "groupId": "OG002", "value": "1" }, + { "groupId": "OG003", "value": "0" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG002", + "value": "2.930", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + } + ] + } + ] + }, + { + "title": "Month 4", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "1" }, + { "groupId": "OG002", "value": "1" }, + { "groupId": "OG003", "value": "0" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG001", + "value": "0.000", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + }, + { + "groupId": "OG002", + "value": "5.860", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + } + ] + } + ] + }, + { + "title": "Month 5", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "1" }, + { "groupId": "OG002", "value": "1" }, + { "groupId": "OG003", "value": "0" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG001", + "value": "0.000", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + }, + { + "groupId": "OG002", + "value": "4.610", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + } + ] + } + ] + }, + { + "title": "Month 6", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "1" }, + { "groupId": "OG002", "value": "1" }, + { "groupId": "OG003", "value": "0" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG001", + "value": "2.590", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + }, + { + "groupId": "OG002", + "value": "4.490", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + } + ] + } + ] + }, + { + "title": "Month 7", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "1" }, + { "groupId": "OG002", "value": "1" }, + { "groupId": "OG003", "value": "0" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG001", + "value": "4.550", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + }, + { + "groupId": "OG002", + "value": "7.490", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + } + ] + } + ] + }, + { + "title": "Month 8", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "1" }, + { "groupId": "OG002", "value": "1" }, + { "groupId": "OG003", "value": "0" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG001", + "value": "7.170", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + }, + { + "groupId": "OG002", + "value": "8.290", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + } + ] + } + ] + }, + { + "title": "Month 9", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "1" }, + { "groupId": "OG002", "value": "1" }, + { "groupId": "OG003", "value": "0" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG001", + "value": "8.690", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + }, + { + "groupId": "OG002", + "value": "7.690", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + } + ] + } + ] + }, + { + "title": "Month 10", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "1" }, + { "groupId": "OG002", "value": "1" }, + { "groupId": "OG003", "value": "0" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG001", + "value": "10.320", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + }, + { + "groupId": "OG002", + "value": "9.900", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + } + ] + } + ] + }, + { + "title": "Month 11", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "1" }, + { "groupId": "OG002", "value": "1" }, + { "groupId": "OG003", "value": "0" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG001", + "value": "14.050", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + }, + { + "groupId": "OG002", + "value": "3.460", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + } + ] + } + ] + }, + { + "title": "Month 12", + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "1" }, + { "groupId": "OG002", "value": "1" }, + { "groupId": "OG003", "value": "0" } + ] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "OG001", + "value": "10.120", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + }, + { + "groupId": "OG002", + "value": "0.040", + "spread": "NA", + "comment": "Only 1 participant had available data so standard deviation could not be calculated." + } + ] + } + ] + } + ] + } + ] + }, + "adverseEventsModule": { + "frequencyThreshold": "0", + "timeFrame": "Adverse events were collected from the single dose of study treatment until the end of study visit (12 months for Cohort 1 and 2 and 15 months for Cohort 3)", + "description": "Safety Analysis Set: All participants given an AVXS-101 intrathecal injection. Participants were analyzed according to actual dose received.", + "eventGroups": [ + { + "id": "EG000", + "title": "Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 6.0E13 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued.", + "deathsNumAffected": 0, + "deathsNumAtRisk": 3, + "seriousNumAffected": 1, + "seriousNumAtRisk": 3, + "otherNumAffected": 3, + "otherNumAtRisk": 3 + }, + { + "id": "EG001", + "title": "Cohort 2: 1.2E14 vg - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 1.2E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued.", + "deathsNumAffected": 0, + "deathsNumAtRisk": 13, + "seriousNumAffected": 2, + "seriousNumAtRisk": 13, + "otherNumAffected": 13, + "otherNumAtRisk": 13 + }, + { + "id": "EG002", + "title": "Cohort 2: 1.2E14 vg - Age 24 to <60 Months", + "description": "Participants aged 24 to \\<60 months at time of AVXS-101 dosing received a single 1.2E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued.", + "deathsNumAffected": 0, + "deathsNumAtRisk": 12, + "seriousNumAffected": 4, + "seriousNumAtRisk": 12, + "otherNumAffected": 12, + "otherNumAtRisk": 12 + }, + { + "id": "EG003", + "title": "Cohort 3: 2.4E14 vg - Age 6 to <24 Months", + "description": "Participants aged 6 to \\<24 months at time of AVXS-101 dosing received a single 2.4E14 vg dose of AVXS-101 administered as an intrathecal injection on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1 mg/kg/day from 1 day prior to AVXS-101 injection until at least 30 days post-injection at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued.", + "deathsNumAffected": 0, + "deathsNumAtRisk": 4, + "seriousNumAffected": 0, + "seriousNumAtRisk": 4, + "otherNumAffected": 4, + "otherNumAtRisk": 4 + } + ], + "seriousEvents": [ + { + "term": "Bronchitis", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 0, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 1, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Influenza", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 0, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 1, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Pneumonia", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 0, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 1, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Pneumonia respiratory syncytial viral", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 0, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 1, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Respiratory tract infection viral", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 0, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 1, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Rhinovirus infection", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 0, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 1, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Blood alkaline phosphatase increased", + "organSystem": "Investigations", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 1, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 1, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 0, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Alanine aminotransferase increased", + "organSystem": "Investigations", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 1, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 0, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Aspartate aminotransferase increased", + "organSystem": "Investigations", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 1, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 0, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Acute respiratory failure", + "organSystem": "Respiratory, thoracic and mediastinal disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 0, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 1, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Asthma", + "organSystem": "Respiratory, thoracic and mediastinal disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 0, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 1, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Respiratory failure", + "organSystem": "Respiratory, thoracic and mediastinal disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 1, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 0, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 0, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + } + ], + "otherEvents": [ + { + "term": "Lymphadenopathy", + "organSystem": "Blood and lymphatic system disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 2, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 1, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Leukopenia", + "organSystem": "Blood and lymphatic system disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 1, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 0, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Tachycardia", + "organSystem": "Cardiac disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 2, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 2, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Mitral valve incompetence", + "organSystem": "Cardiac disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 1, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 1, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Sinus tachycardia", + "organSystem": "Cardiac disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 1, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 1, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Cardiomegaly", + "organSystem": "Cardiac disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 0, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 1, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Pericardial effusion", + "organSystem": "Cardiac disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 1, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 0, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Pulmonary valve incompetence", + "organSystem": "Cardiac disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 0, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 1, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Pectus carinatum", + "organSystem": "Congenital, familial and genetic disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 1, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 0, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Pectus excavatum", + "organSystem": "Congenital, familial and genetic disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 1, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 0, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Ear discomfort", + "organSystem": "Ear and labyrinth disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 0, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 1, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Motion sickness", + "organSystem": "Ear and labyrinth disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 0, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 1, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Cushingoid", + "organSystem": "Endocrine disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 0, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 1, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Vomiting", + "organSystem": "Gastrointestinal disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 5, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 3, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 2, "numAtRisk": 4 } + ] + }, + { + "term": "Constipation", + "organSystem": "Gastrointestinal disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 2, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 3, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 2, "numAtRisk": 4 } + ] + }, + { + "term": "Teething", + "organSystem": "Gastrointestinal disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 1, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 2, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 0, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 1, "numAtRisk": 4 } + ] + }, + { + "term": "Abdominal pain", + "organSystem": "Gastrointestinal disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, "numAtRisk": 3 }, + { "groupId": "EG001", "numAffected": 0, "numAtRisk": 13 }, + { "groupId": "EG002", "numAffected": 1, "numAtRisk": 12 }, + { "groupId": "EG003", "numAffected": 0, "numAtRisk": 4 } + ] + }, + { + "term": "Anal fissure", + "organSystem": "Gastrointestinal disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { "groupId": "EG000", "numAffected": 0, 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These full results are available via this link: https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=17933" + }, + "certainAgreement": { + "piSponsorEmployee": false, + "restrictionType": "OTHER", + "restrictiveAgreement": true, + "otherDetails": "Depending on local requirements, Sponsor's consent necessary before publication of study, or Sponsor can review results communications before public release with a right to request changes to communications regarding trial results between 40 to 60 and up to 90 or 120 days, as applicable, from the time submitted to Sponsor for review to remove references to Sponsor's Confidential Information or delay results communications to permit Sponsor to obtain appropriate Intellectual Property protection." + }, + "pointOfContact": { + "title": "EMEA Medical Information", + "organization": "Novartis Gene Therapies EU Limited", + "email": "medinfoemea.gtx@novartis.com", + "phone": "+353 (1) 566-2364" + } + } + }, + "documentSection": { + "largeDocumentModule": { + "largeDocs": [ + { + "typeAbbrev": "Prot", + "hasProtocol": true, + "hasSap": false, + "hasIcf": false, + "label": "Study Protocol", + "date": "2019-06-17", + "uploadDate": "2023-04-19T15:33", + "filename": "Prot_002.pdf", + "size": 1778854 + }, + { + "typeAbbrev": "SAP", + "hasProtocol": false, + "hasSap": true, + "hasIcf": false, + "label": "Statistical Analysis Plan", + "date": "2021-11-22", + "uploadDate": "2023-04-19T15:39", + "filename": "SAP_003.pdf", + "size": 17685553 + } + ] + } + }, + "derivedSection": { + "miscInfoModule": { "versionHolder": "2024-12-04" }, + "conditionBrowseModule": { + "meshes": [ + { "id": "D009133", "term": "Muscular Atrophy" }, + { "id": "D009134", "term": "Muscular Atrophy, Spinal" }, + { "id": "D001284", "term": "Atrophy" } + ], + "ancestors": [ + { "id": "D020763", "term": "Pathological Conditions, Anatomical" }, + { "id": "D020879", "term": "Neuromuscular Manifestations" }, + { "id": "D009461", "term": "Neurologic Manifestations" }, + { "id": "D009422", "term": "Nervous System Diseases" }, + { "id": "D013118", "term": "Spinal Cord Diseases" }, + { "id": "D002493", "term": "Central Nervous System Diseases" }, + { "id": "D016472", "term": "Motor Neuron Disease" }, + { "id": "D019636", "term": "Neurodegenerative Diseases" }, + { "id": "D009468", "term": "Neuromuscular Diseases" } + ], + "browseLeaves": [ + { "id": "M17522", "name": "Virus Diseases", "relevance": "LOW" }, + { + "id": "M4589", + "name": "Atrophy", + "asFound": "Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12090", + "name": "Muscular Atrophy", + "asFound": "Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12091", + "name": "Muscular Atrophy, Spinal", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M22519", + "name": "Pathological Conditions, Anatomical", + "relevance": "LOW" + }, + { + "id": "M22619", + "name": "Neuromuscular Manifestations", + "relevance": "LOW" + }, + { + "id": 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"abbrev": "BC10", "name": "Nervous System Diseases" }, + { "abbrev": "BC18", "name": "Nutritional and Metabolic Diseases" }, + { "abbrev": "Rare", "name": "Rare Diseases" } + ] + } + }, + "hasResults": true + }, + { + "protocolSection": { + "identificationModule": { + "nctId": "NCT05386680", + "orgStudyIdInfo": { "id": "COAV101B12302" }, + "organization": { "fullName": "Novartis", "class": "INDUSTRY" }, + "briefTitle": "Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam", + "officialTitle": "Phase IIIb, Open-label, Single-arm, Multi-center Study to Evaluate the Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally (1.2 x 10^14 Vector Genomes) to Participants 2 to < 18 Years of Age With Spinal Muscular Atrophy (SMA) Who Have Discontinued Treatment With Nusinersen (Spinraza\u00ae) or Risdiplam (Evrysdi\u00ae)", + "acronym": "STRENGTH" + }, + "statusModule": { + "statusVerifiedDate": "2024-01", + "overallStatus": "ACTIVE_NOT_RECRUITING", + "expandedAccessInfo": { "hasExpandedAccess": false }, + "startDateStruct": { "date": "2023-01-12", "type": "ACTUAL" }, + "primaryCompletionDateStruct": { + "date": "2024-12-03", + "type": "ESTIMATED" + }, + "completionDateStruct": { "date": "2024-12-03", "type": "ESTIMATED" }, + "studyFirstSubmitDate": "2022-05-18", + "studyFirstSubmitQcDate": "2022-05-18", + "studyFirstPostDateStruct": { + "date": "2022-05-23", + "type": "ACTUAL" + }, + "lastUpdateSubmitDate": "2024-01-17", + "lastUpdatePostDateStruct": { "date": "2024-01-19", "type": "ACTUAL" } + }, + "sponsorCollaboratorsModule": { + "responsibleParty": { "type": "SPONSOR" }, + "leadSponsor": { + "name": "Novartis Pharmaceuticals", + "class": "INDUSTRY" + } + }, + "oversightModule": { + "oversightHasDmc": true, + "isFdaRegulatedDrug": true, + "isFdaRegulatedDevice": false + }, + "descriptionModule": { + "briefSummary": "This is an open-label, single arm, multi-center study. Approximately 28 participants aged 2 to \\<18 years will be enrolled stratified as 2 to 5 years and 6 to \\< 18 years. The study is comprised of 3 periods, Screening (up to 45 days), Treatment (1 day), and Follow-up (52 weeks).", + "detailedDescription": "During the Screening period and on Day -1 (Baseline), eligibility will be assessed, including confirmation that nusinersen (Spinraza\u00ae) or risdiplam (Evrysdi\u00ae) have not been used for the defined period (4 month and 15 days prior to Day -1 respectively). On Day - 1 (Baseline) participants will be admitted to the hospital for pre-treatment baseline procedures. Prednisolone (or equivalent) will be given and continued as per the study protocol.\n\nParticipants who meet eligibility criteria at Screening and Baseline will receive a single dose of OAV101 (1.2 x 10\\^14 vector genomes) by lumbar intrathecal injection on Day 1 (Treatment) and will then undergo in-patient safety monitoring atleast the next 48 hours, after which the participant may be discharged according to Investigator judgement.\n\nDuring Follow-up, safety monitoring will continue as per the visits defined in the Assessment Schedule. Safety for participants enrolled will be evaluated by the study team together with the Data Monitoring Committee (DMC).\n\nFinal analysis will be performed after all participants have completed Week 52 or discontinued prior to Week 52. At the end of study, participants will be invited to enroll in a Novartis-sponsored long-term follow-up study to monitor long-term safety and efficacy." + }, + "conditionsModule": { + "conditions": ["Spinal Muscular Atrophy"], + "keywords": [ + "Zolgensma", + "OAV101", + "AVXS 101", + "gene therapy", + "Muscle atrophy", + "SBMA", + "spinal and bulbar muscular atrophy", + "spinal muscular atrophy", + "bulbar muscular atrophy", + "muscle function", + "myopathy", + "muscle wasting", + "atrophied muscle", + "loss of muscle strength", + "SMA" + ] + }, + "designModule": { + "studyType": "INTERVENTIONAL", + "phases": ["PHASE3"], + "designInfo": { + "allocation": "NON_RANDOMIZED", + "interventionModel": "SINGLE_GROUP", + "primaryPurpose": "TREATMENT", + "maskingInfo": { "masking": "NONE" } + }, + "enrollmentInfo": { "count": 27, "type": "ACTUAL" } + }, + "armsInterventionsModule": { + "armGroups": [ + { + "label": "OAV-101", + "type": "EXPERIMENTAL", + "description": "Intrathecal administration of OAV101 at a dose of 1.2 x 10\\^14 vector genomes, one time dose", + "interventionNames": ["Genetic: OAV101"] + } + ], + "interventions": [ + { + "type": "GENETIC", + "name": "OAV101", + "description": "Intrathecal administration of OAV101 at a dose of 1.2 x 10\\^14 vector genomes, one time dose", + "armGroupLabels": ["OAV-101"], + "otherNames": ["AVXS-101", "Zolgensma"] + } + ] + }, + "outcomesModule": { + "primaryOutcomes": [ + { + "measure": "Number and percentage of participants reporting AEs, related AEs, SAEs, and AESIs", + "description": "An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign \\[including abnormal laboratory findings\\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study. The occurrence of AEs must be sought by non-directive questioning of the participant at each visit during the study. Adverse events also may be detected when they are volunteered by the participant during or between visits or through physical examination findings, laboratory test findings, or other assessments.\n\nAn AESI is primarily defined by using standard Medical Dictionary for Regulatory Activities (MedDRA) queries, and identified as follows: Hepatotoxicity, Thrombocytopenia, Thrombotic microangiopathy, Cardiac adverse events, and Dorsal root ganglia toxicity", + "timeFrame": "52 weeks" + } + ], + "secondaryOutcomes": [ + { + "measure": "Change from baseline to Week 52 visit in the HFMSE total score", + "description": "The Hammersmith Functional Motor Scale Expanded (HFMSE) is a SMA-specific 33-item assessment that is administered by clinical evaluators in a short period of time, requires minimal equipment, and is designed to factor in patient fatigue. Each motor skill item is scored on a 3-point Likert scale from 0 (no response) to 2 (full response), with a total score range of 0 to 66. A higher score indicates a higher ability level.", + "timeFrame": "52 weeks" + }, + { + "measure": "Change from baseline to Week 52 visit in the RULM total Score", + "description": "The Revised Upper Limb Model (RULM) is a validated, SMA-specific assessment that measures motor performance in the upper limbs from childhood through adulthood in ambulatory and never ambulatory individuals with SMA. The revised version of the test consists of 19 scorable items: 18 items scored on a 0 (unable) to 2 (full achievement) scale, and one item that is scored from 0 (unable) to 1 (able). These item scores are summed to give a total score ranging from 0 to 37 points with lower scores reflecting poorer ability.", + "timeFrame": "52 weeks" + }, + { + "measure": "Change from baseline to Week 52 visit in Assessment of Caregiver Experience in ACEND instrument score", + "description": "The Assessment of Caregiver Experience in Neuromuscular Disease (ACEND) instrument quantifies the caregiver impact experienced by parents/caregivers of children affected with severe neuromuscular diseases, including children with SMA The total score is on a scale of 0 to 100 with a higher score indicating a greater impact on the caregiver.", + "timeFrame": "52 weeks" + } + ] + }, + "eligibilityModule": { + "eligibilityCriteria": "Inclusion Criteria\n\n* SMA diagnosis\n* Aged 2 to \\< 18 years\n* Have had at least four loading doses of nusinersen (Spinraza\u00ae) or at least 3 months of treatment with risdiplam (Evrysdi\u00ae) at Screening\n* Must have symptoms of SMA as defined in the protocol\n\nExclusion Criteria:\n\n* Anti Adeno Associated Virus Serotype 9 (AAV9) antibody titer using an immunoassay is reported as elevated\n* Clinically significant abnormalities in test results during screening\n* Contraindications for lumbar puncture procedure\n* At Baseline, participants are excluded if they received:\n\n * nusinersen (Spinraza\u00ae) or\n * risdiplam (Evrysdi\u00ae) within a defined timeframe\n* Vaccinations 2 weeks prior to administration of OAV101\n* Hospitalization for a pulmonary event, or for nutritional support within 2 months prior to Screening or inpatient major surgery planned.\n* Presence of an infection or febrile illness up to 30 days prior to administration of OAV101\n* Requiring invasive ventilation", + "healthyVolunteers": false, + "sex": "ALL", + "minimumAge": "2 Years", + "maximumAge": "17 Years", + "stdAges": ["CHILD"] + }, + "contactsLocationsModule": { + "locations": [ + { + "facility": "Novartis Investigative Site", + "city": "Boston", + "state": "Massachusetts", + "zip": "02215", + "country": "United States", + "geoPoint": { "lat": 42.35843, "lon": -71.05977 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Norfolk", + "state": "Virginia", + "zip": "23507", + "country": "United States", + "geoPoint": { "lat": 36.84681, "lon": -76.28522 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Madison", + "state": "Wisconsin", + "zip": "53792-7375", + "country": "United States", + "geoPoint": { "lat": 43.07305, "lon": -89.40123 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Parkville", + "state": "Victoria", + "zip": "3052", + "country": "Australia", + "geoPoint": { "lat": -37.78333, "lon": 144.95 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Leuven", + "zip": "3000", + "country": "Belgium", + "geoPoint": { "lat": 50.87959, "lon": 4.70093 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Montreal", + "state": "Quebec", + "zip": "H4A 3J1", + "country": "Canada", + "geoPoint": { "lat": 45.50884, "lon": -73.58781 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Bron Cedex", + "zip": "69677", + "country": "France", + "geoPoint": { "lat": 45.73333, "lon": 4.91667 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Toulouse Cedex", + "zip": "31059", + "country": "France", + "geoPoint": { "lat": 43.60426, "lon": 1.44367 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Roma", + "state": "RM", + "zip": "00168", + "country": "Italy", + "geoPoint": { "lat": 41.89193, "lon": 12.51133 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Kurume city", + "state": "Fukuoka", + "zip": "830-0011", + "country": "Japan", + "geoPoint": { "lat": 33.31667, "lon": 130.51667 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Shinjuku ku", + "state": "Tokyo", + "zip": "162 8666", + "country": "Japan", + "geoPoint": { "lat": 35.2946, "lon": 139.57059 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Utrecht", + "zip": "3584CX", + "country": "Netherlands", + "geoPoint": { "lat": 52.09083, "lon": 5.12222 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Barcelona", + "state": "Catalunya", + "zip": "08035", + "country": "Spain", + "geoPoint": { "lat": 41.38879, "lon": 2.15899 } + } + ] + }, + "ipdSharingStatementModule": { + "ipdSharing": "YES", + "description": "Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on https://www.clinicalstudydatarequest.com/.", + "url": "https://www.clinicalstudydatarequest.com/" + } + }, + "derivedSection": { + "miscInfoModule": { "versionHolder": "2024-12-04" }, + "conditionBrowseModule": { + "meshes": [ + { "id": "D009133", "term": "Muscular Atrophy" }, + { "id": "D009134", "term": "Muscular Atrophy, Spinal" }, + { "id": "D001284", "term": "Atrophy" } + ], + "ancestors": [ + { "id": "D020763", "term": "Pathological Conditions, Anatomical" }, + { "id": "D020879", "term": "Neuromuscular Manifestations" }, + { "id": "D009461", "term": "Neurologic Manifestations" }, + { "id": "D009422", "term": "Nervous System Diseases" }, + { "id": "D013118", "term": "Spinal Cord Diseases" }, + { "id": "D002493", "term": "Central Nervous System Diseases" }, + { "id": "D016472", "term": "Motor Neuron Disease" }, + { "id": "D019636", "term": "Neurodegenerative Diseases" }, + { "id": "D009468", "term": "Neuromuscular Diseases" } + ], + "browseLeaves": [ + { "id": "M21265", "name": "Wasting Syndrome", "relevance": "LOW" }, + { "id": "M5363", "name": "Cachexia", "relevance": "LOW" }, + { + "id": "M4589", + "name": "Atrophy", + "asFound": "Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12090", + "name": "Muscular Atrophy", + "asFound": "Muscular Atrophy", + "relevance": "HIGH" + }, + { "id": "M12092", "name": "Muscular Diseases", "relevance": "LOW" }, + { + "id": "M12091", + "name": "Muscular Atrophy, Spinal", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M28216", + "name": "Bulbo-Spinal Atrophy, X-Linked", + "relevance": "LOW" + }, + { + "id": "M22519", + "name": "Pathological Conditions, Anatomical", + "relevance": "LOW" + }, + { + "id": "M22619", + "name": "Neuromuscular Manifestations", + "relevance": "LOW" + }, + { + "id": "M12404", + "name": "Neurologic Manifestations", + "relevance": "LOW" + }, + { + "id": "M15915", + "name": "Spinal Cord Diseases", + "relevance": "LOW" + }, + { + "id": "M5742", + "name": "Central Nervous System Diseases", + "relevance": "LOW" + }, + { + "id": "M18879", + "name": "Motor Neuron Disease", + "relevance": "LOW" + }, + { + "id": "M4024", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + }, + { + "id": "M21558", + "name": "Neurodegenerative Diseases", + "relevance": "LOW" + }, + { + "id": "M12411", + "name": "Neuromuscular Diseases", + "relevance": "LOW" + }, + { "id": "T3215", "name": "Kennedy Disease", "relevance": "LOW" }, + { + "id": "T5342", + "name": "Spinal Muscular Atrophy", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "T349", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + } + ], + "browseBranches": [ + { "abbrev": "BC18", "name": "Nutritional and Metabolic Diseases" }, + { "abbrev": "All", "name": "All Conditions" }, + { "abbrev": "BC23", "name": "Symptoms and General Pathology" }, + { "abbrev": "BC10", "name": "Nervous System Diseases" }, + { "abbrev": "BC05", "name": "Musculoskeletal Diseases" }, + { + "abbrev": "BC16", + "name": "Diseases and Abnormalities at or Before Birth" + }, + { "abbrev": "Rare", "name": "Rare Diseases" } + ] + }, + "interventionBrowseModule": { + "browseLeaves": [ + { "id": "M264314", "name": "Risdiplam", "relevance": "LOW" } + ], + "browseBranches": [ + { "abbrev": "All", "name": "All Drugs and Chemicals" } + ] + } + }, + "hasResults": false + }, + { + "protocolSection": { + "identificationModule": { + "nctId": "NCT05335876", + "orgStudyIdInfo": { "id": "COAV101A12308" }, + "organization": { "fullName": "Novartis", "class": "INDUSTRY" }, + "briefTitle": "Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials", + "officialTitle": "Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials", + "acronym": "SPECTRUM" + }, + "statusModule": { + "statusVerifiedDate": "2024-05", + "overallStatus": "RECRUITING", + "expandedAccessInfo": { "hasExpandedAccess": false }, + "startDateStruct": { "date": "2022-12-19", "type": "ACTUAL" }, + "primaryCompletionDateStruct": { + "date": "2039-10-18", + "type": "ESTIMATED" + }, + "completionDateStruct": { "date": "2039-10-18", "type": "ESTIMATED" }, + "studyFirstSubmitDate": "2022-04-12", + "studyFirstSubmitQcDate": "2022-04-12", + "studyFirstPostDateStruct": { + "date": "2022-04-20", + "type": "ACTUAL" + }, + "lastUpdateSubmitDate": "2024-05-22", + "lastUpdatePostDateStruct": { "date": "2024-05-24", "type": "ACTUAL" } + }, + "sponsorCollaboratorsModule": { + "responsibleParty": { "type": "SPONSOR" }, + "leadSponsor": { + "name": "Novartis Pharmaceuticals", + "class": "INDUSTRY" + } + }, + "oversightModule": { + "oversightHasDmc": true, + "isFdaRegulatedDrug": true, + "isFdaRegulatedDevice": false + }, + "descriptionModule": { + "briefSummary": "This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will continue for 15 years from the date of OAV101 administration in the previous clinical trial.", + "detailedDescription": "The study is comprised of a Baseline Period and 3 Follow-up Periods. Follow-up Periods 1 and 2 consist of in-person visits and Period 3 consists of tele-visits. Follow-up Periods 1 and 2, which includes Baseline through Year 5 visits, assessments will be performed at the Investigational site. During Follow-up Period 3 (Year 6 to up to Year 15 after OAV101 administration), participants/caregivers will be contacted using tele-visits annually for remote assessments. All patients will enter the study at the baseline visit and continue until 15 years since OAV101 administration is reached. Total duration of participation in the study will be dependent upon time of enrollment relative to OAV101 administration and will vary by participant." + }, + "conditionsModule": { + "conditions": ["Spinal Muscular Atrophy (SMA)"], + "keywords": [ + "Zolgensma", + "OAV101", + "AVXS 101", + "gene therapy", + "Muscle atrophy", + "SBMA", + "spinal and bulbar muscular atrophy", + "spinal muscular atrophy", + "bulbar muscular atrophy", + "muscle function", + "myopathy", + "muscle wasting", + "atrophied muscle", + "loss of muscle strength", + "Spinal Muscular Atrophy (SMA)", + "survival motor neuron 1 gene (SMN1)", + "SMN protein depletion", + "survival motor neuron 2 gene (SMN2)", + "chromosome 5q13", + "neurogenetic disorder", + "onasemnogene abeparvovec" + ] + }, + "designModule": { + "studyType": "INTERVENTIONAL", + "phases": ["PHASE3"], + "designInfo": { + "allocation": "NA", + "interventionModel": "SINGLE_GROUP", + "primaryPurpose": "TREATMENT", + "maskingInfo": { "masking": "NONE" } + }, + "enrollmentInfo": { "count": 260, "type": "ESTIMATED" } + }, + "armsInterventionsModule": { + "armGroups": [ + { + "label": "Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec", + "type": "EXPERIMENTAL", + "description": "Patients who received OAV101 IT or OAV101 IV in clinical trials (COAV101A12306, COAV101B12301 and COAV101B12302)", + "interventionNames": ["Biological: onasemnogene abeparvovec"] + } + ], + "interventions": [ + { + "type": "BIOLOGICAL", + "name": "onasemnogene abeparvovec", + "description": "Onasemnogene abeparvovec is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the ytomegalovirus enhancer/chicken \u03b2-actin-hybrid promoter. Onasemnogene abeparvovec is administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.", + "armGroupLabels": [ + "Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec" + ], + "otherNames": ["Zolgensma"] + } + ] + }, + "outcomesModule": { + "primaryOutcomes": [ + { + "measure": "Number of participants with treatment-emergent serious adverse events (SAEs)", + "description": "An SAE is defined as any adverse event \\[appearance of (or worsening of any pre-existing)\\] undesirable sign(s), symptom(s), or medical conditions(s) which meets any one of the following criteria:\n\n* fatal\n* life-threatening\n* results in persistent or significant disability/incapacity\n* constitutes a congenital anomaly/birth defect, fetal death or congenital abnormality or birth defect\n* requires in-patient hospitalization or prolongation of existing hospitalization, unless hospitalization is for routine treatment or monitoring of the studied indication, not associated with any deterioration in condition\n* is medically significant, e.g. defined as an event that jeopardizes the participant or may require medical or surgical intervention to prevent one of the outcomes listed above", + "timeFrame": "Up to Year 15" + }, + { + "measure": "Number of participants with treatment emergent Adverse Events of Special Interest (AESI)", + "description": "The following are important identified and important potential risks (AESI) associated with OAV101: Hepatotoxicity, Transient Thrombocytopenia, Cardiac adverse events, Sensory abnormalities suggestive of ganglionopathy, and Thrombotic microangiopathy. These will be assessed by the investigator.", + "timeFrame": "Up to Year 15" + } + ], + "secondaryOutcomes": [ + { + "measure": "The number of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist", + "description": "The Developmental Milestone Checklist is a sponsor created list of items using relevant definitions obtained from World Health Organization Multicentre Growth Reference Study (WHO-MGRS). These will be assessed via the milestone checklist, formed of 6 yes/no questions. The developmental milestones are: sitting with support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone. A yes response indicates that the patient reached a particular development milestone.", + "timeFrame": "Up to Year 5" + }, + { + "measure": "The number of participants demonstrating maintenance of each developmental milestone according to the Developmental Milestone Checklist", + "description": "The Developmental Milestone Checklist is a sponsor created list of items using relevant definitions obtained from World Health Organization Multicentre Growth Reference Study (WHO-MGRS). These will be assessed via the milestone checklist, formed of 6 yes/no questions. The developmental milestones are: sitting with support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone. A yes response indicates that the patient reached a particular development milestone.", + "timeFrame": "Up to Year 5" + }, + { + "measure": "Change from Baseline in the Hammersmith Functional Motor Scale - Expanded (HFMSE) total score", + "description": "The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability.", + "timeFrame": "Up to Year 5" + }, + { + "measure": "Change from Baseline in the Revised Upper Limb Module (RULM) total score", + "description": "The RULM is a validated SMA specific assessment of motor performance in the upper limbs from childhood through adulthood in ambulatory and non-ambulatory individuals with SMA. The scale consists of 19 scorable items: 18 items scored on 0 (unable) to 2 (full achievement) scale, and one item that is scored from 0 (unable) to 1 (able). Total scores range from 0-37 points. Higher scores reflect higher level of motor ability.", + "timeFrame": "Up to Year 5" + }, + { + "measure": "Systolic and diastolic blood pressure (mmHg)", + "timeFrame": "Up to Year 15" + }, + { + "measure": "Number of patients with potentialy clinically significant vital sign findings - Respiratory Rate (breaths/min)", + "timeFrame": "Up to Year 15" + }, + { + "measure": "Number of patients with potentialy clinically significant vital sign findings -Pulse Rate (beats/min)", + "timeFrame": "Up to Year 15" + }, + { + "measure": "Number of patients with potentialy clinically significant vital sign findings -Temperature (Degrees Celsius)", + "timeFrame": "Up to Year 15" + }, + { + "measure": "Number of patients with potentialy clinically significant vital sign findings -Oxygen saturation level (%).", + "description": "Oxygen saturation is the fraction of oxygen-saturated hemoglobin relative to total hemoglobin (unsaturated+saturated) in the blood and then multiplied by 100.", + "timeFrame": "Up to Year 15" + } + ] + }, + "eligibilityModule": { + "eligibilityCriteria": "Inclusion Criteria:\n\n1. Participated in an OAV101 clinical trial.\n2. Written informed consent must be obtained before any assessment is performed.\n3. Patient/Parent/legal guardian willing and able to comply with study procedures.\n\nExclusion Criteria:\n\nThere are no exclusion criteria for this study.", + "healthyVolunteers": false, + "sex": "ALL", + "stdAges": ["CHILD", "ADULT", "OLDER_ADULT"] + }, + "contactsLocationsModule": { + "centralContacts": [ + { + "name": "Novartis Gene Therapies Med Info (US, Latin America, Canada)", + "role": "CONTACT", + "phone": "+1-833-828-3947", + "email": "medinfo.gtx@novartis.com" + }, + { + "name": "Novartis Gene Therapies Med Info (Europe, Middle East, Africa, Asia-Pacific)", + "role": "CONTACT", + "phone": "+353 (1) 566-2364", + "email": "medinfoemea.gtx@novartis.com" + } + ], + "locations": [ + { + "facility": "Child Hosp of the Kings Daughters", + "status": "RECRUITING", + "city": "Norfolk", + "state": "Virginia", + "zip": "23507", + "country": "United States", + "contacts": [ + { + "name": "Erin Smith", + "role": "CONTACT", + "phone": "757-668-9026", + "email": "erin.smith@chkd.org" + }, + { "name": "Crystal Proud", "role": "PRINCIPAL_INVESTIGATOR" } + ], + "geoPoint": { "lat": 36.84681, "lon": -76.28522 } + }, + { + "facility": "Novartis Investigative Site", + "status": "RECRUITING", + "city": "Randwick", + "state": "New South Wales", + "zip": "2031", + "country": "Australia", + "geoPoint": { "lat": -33.91439, "lon": 151.24895 } + }, + { + "facility": "Novartis Investigative Site", + "status": "RECRUITING", + "city": "Leuven", + "zip": "3000", + "country": "Belgium", + "geoPoint": { "lat": 50.87959, "lon": 4.70093 } + }, + { + "facility": "Novartis Investigative Site", + "status": "RECRUITING", + "city": "Montreal", + "state": "Quebec", + "zip": "H4A 3J1", + "country": "Canada", + "geoPoint": { "lat": 45.50884, "lon": -73.58781 } + }, + { + "facility": "Novartis Investigative Site", + "status": "RECRUITING", + "city": "Chengdu", + "state": "Sichuan", + "zip": "610041", + "country": "China", + "geoPoint": { "lat": 30.66667, "lon": 104.06667 } + }, + { + "facility": 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"lat": 54.97328, "lon": -1.61396 } + } + ] + }, + "ipdSharingStatementModule": { + "ipdSharing": "YES", + "description": "Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.\n\nThis trial data availability is according to the criteria and process described on https://www.clinicalstudydatarequest.com/.", + "url": "https://www.clinicalstudydatarequest.com/" + } + }, + "derivedSection": { + "miscInfoModule": { "versionHolder": "2024-12-04" }, + "conditionBrowseModule": { + "meshes": [ + { "id": "D009133", "term": "Muscular Atrophy" }, + { "id": "D009134", "term": "Muscular Atrophy, Spinal" }, + { "id": "D001284", "term": "Atrophy" } + ], + "ancestors": [ + { "id": "D020763", "term": "Pathological Conditions, Anatomical" }, + { "id": "D020879", "term": "Neuromuscular Manifestations" }, + { "id": "D009461", "term": "Neurologic Manifestations" }, + { "id": "D009422", "term": "Nervous System Diseases" }, + { "id": "D013118", "term": "Spinal Cord Diseases" }, + { "id": "D002493", "term": "Central Nervous System Diseases" }, + { "id": "D016472", "term": "Motor Neuron Disease" }, + { "id": "D019636", "term": "Neurodegenerative Diseases" }, + { "id": "D009468", "term": "Neuromuscular Diseases" } + ], + "browseLeaves": [ + { "id": "M21265", "name": "Wasting Syndrome", "relevance": "LOW" }, + { "id": "M5363", "name": "Cachexia", "relevance": "LOW" }, + { + "id": "M4589", + "name": "Atrophy", + "asFound": "Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12090", + "name": "Muscular Atrophy", + "asFound": "Muscular Atrophy", + "relevance": "HIGH" + }, + { "id": "M12092", "name": "Muscular Diseases", "relevance": "LOW" }, + { + "id": "M12091", + "name": "Muscular Atrophy, Spinal", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M28216", + "name": "Bulbo-Spinal Atrophy, X-Linked", + "relevance": "LOW" + }, + { + "id": "M22519", + "name": "Pathological Conditions, Anatomical", + "relevance": "LOW" + }, + { + "id": "M22619", + "name": "Neuromuscular Manifestations", + "relevance": "LOW" + }, + { + "id": "M12404", + "name": "Neurologic Manifestations", + "relevance": "LOW" + }, + { + "id": "M15915", + "name": "Spinal Cord Diseases", + "relevance": "LOW" + }, + { + "id": "M5742", + "name": "Central Nervous System Diseases", + "relevance": "LOW" + }, + { + "id": "M18879", + "name": "Motor Neuron Disease", + "relevance": "LOW" + }, + { + "id": "M4024", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + }, + { + "id": "M21558", + "name": "Neurodegenerative Diseases", + "relevance": "LOW" + }, + { + "id": "M12411", + "name": "Neuromuscular Diseases", + "relevance": "LOW" + }, + { "id": "T3215", "name": "Kennedy Disease", "relevance": "LOW" }, + { + "id": "T5342", + "name": "Spinal Muscular Atrophy", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "T349", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + } + ], + "browseBranches": [ + { "abbrev": "BC18", "name": "Nutritional and Metabolic Diseases" }, + { "abbrev": "All", "name": "All Conditions" }, + { "abbrev": "BC23", "name": "Symptoms and General Pathology" }, + { "abbrev": "BC10", "name": "Nervous System Diseases" }, + { "abbrev": "BC05", "name": "Musculoskeletal Diseases" }, + { + "abbrev": "BC16", + "name": "Diseases and Abnormalities at or Before Birth" + }, + { "abbrev": "Rare", "name": "Rare Diseases" } + ] + } + }, + "hasResults": false + }, + { + "protocolSection": { + "identificationModule": { + "nctId": "NCT06532474", + "orgStudyIdInfo": { "id": "ML43225" }, + "organization": { + "fullName": "St. Jude Children's Research Hospital", + "class": "OTHER" + }, + "briefTitle": "Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies", + "officialTitle": "Pilot Study Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies" + }, + "statusModule": { + "statusVerifiedDate": "2024-11", + "overallStatus": "NOT_YET_RECRUITING", + "expandedAccessInfo": { "hasExpandedAccess": false }, + "startDateStruct": { "date": "2025-01", "type": "ESTIMATED" }, + "primaryCompletionDateStruct": { + "date": "2026-05", + "type": "ESTIMATED" + }, + "completionDateStruct": { "date": "2026-11", "type": "ESTIMATED" }, + "studyFirstSubmitDate": "2024-07-29", + "studyFirstSubmitQcDate": "2024-07-29", + "studyFirstPostDateStruct": { + "date": "2024-08-01", + "type": "ACTUAL" + }, + "lastUpdateSubmitDate": "2024-11-07", + "lastUpdatePostDateStruct": { "date": "2024-11-08", "type": "ACTUAL" } + }, + "sponsorCollaboratorsModule": { + "responsibleParty": { "type": "SPONSOR" }, + "leadSponsor": { + "name": "St. Jude Children's Research Hospital", + "class": "OTHER" + }, + "collaborators": [{ "name": "Genentech, Inc.", "class": "INDUSTRY" }] + }, + "oversightModule": { + "oversightHasDmc": false, + "isFdaRegulatedDrug": false, + "isFdaRegulatedDevice": false + }, + "descriptionModule": { + "briefSummary": "In this observational study, researchers are looking at the effects of spinal muscular atrophy (SMA) drugs on the muscles and nerve cells in patients with SMA.\n\nPrimary Objectives\n\n* To evaluate the feasibility and reliability of performing MR functional imaging in exercising muscle in patients with SMA.\n* To evaluate patients with SMA types 2 and 3 at baseline and longitudinally at 6 and 12 months\n\nSecondary Objectives\n\n* To describe the MR functional bioenergetics response in the leg muscles in four potential groups of patients with spinal muscular atrophy: untreated, actively treated with nusinersen (Spinraza\u00ae) or onasemnogene abeparvovec (Zolgensma\u00ae), actively treated with risdiplam (Evrysdi\u00ae), and switching from Spinraza or Zolgensma to Evrysdi.\n* To identify changes in motor function in patients with SMA types 2 and 3 who initiate treatment with risdiplam.\n* To obtain biomarkers in blood, urine, and muscle tissue to provide evidence for risdiplam effect on skeletal muscle.\n* To obtain quality of life and disability data from participants in this study.", + "detailedDescription": "This is an observational study to demonstrate the feasibility of performing MR functional imaging in exercising muscle in patients with SMA. The participants will be prescribed medication by their treating physician, they will not receive any drug as part of this study.\n\nParticipants participating in the ML43225 study, will be put into one of four groups depending on their type of SMA and the drugs they may or may not be taking. They will be asked to come to clinic 3 times over one year. Each visit will include magnetic resonance (MR) studies, a muscle ultrasound, a nerve test, muscle function testing, lung function testing, blood work, vital signs, and participants will be asked about their quality of life and daily life activities. After participants have completed the 3 required visits, they will be taken off study." + }, + "conditionsModule": { + "conditions": ["Spinal Muscular Atrophy"], + "keywords": [ + "Spinal Muscular Atrophy (SMA)", + "SMA drugs", + "Responses of Skeletal Muscle", + "SMN-directed therapies", + "Eyrysdi", + "Spinraza", + "Zolgensma" + ] + }, + "designModule": { + "studyType": "OBSERVATIONAL", + "patientRegistry": false, + "designInfo": { + "observationalModel": "CASE_ONLY", + "timePerspective": "PROSPECTIVE" + }, + "bioSpec": { + "retention": "SAMPLES_WITH_DNA", + "description": "blood and urine" + }, + "enrollmentInfo": { "count": 24, "type": "ESTIMATED" } + }, + "armsInterventionsModule": { + "armGroups": [ + { + "label": "Cohort 1", + "description": "Current Evrysdi prescription" + }, + { + "label": "Cohort 2", + "description": "Current Spinraza or Zolgensma prescription" + }, + { + "label": "Cohort 3", + "description": "Changing from Spinraza or Zolgensma to Evrysdi" + }, + { + "label": "Cohort 4", + "description": "Have never received any SMN-directed therapies" + } + ] + }, + "outcomesModule": { + "primaryOutcomes": [ + { + "measure": "Feasibility of performing MR functional imaging in SMA patients", + "description": "MR functional imaging is considered feasible if \u2265 80% of MRI protocol eligible patients can complete 100% of imaging assessments at baseline and 6 months.", + "timeFrame": "At baseline and 6 months" + }, + { + "measure": "Reliability of performing MR functional imaging in SMA patients", + "description": "MR functional imaging is considered reliable if the test-retest reliability is \u2265 0.80 for key imaging biomarkers.", + "timeFrame": "At baseline and 6 months" + }, + { + "measure": "Compare skeletal muscle oxidative phosphorylation bioenergetics in patients with SMA types 2 and 3 (phosphocreatine)", + "description": "Real-time 31P MR spectroscopy and CrCEST MRI will be used to measure phosphocreatine within the gastrocnemius-soleus leg muscles at rest, during an exercise protocol, and during post-exercise recovery to baseline. Both measure the recovery time in seconds.", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "Compare skeletal muscle oxidative phosphorylation bioenergetics in patients with SMA types 2 and 3 (creatine concentrations)", + "description": "Real-time 31P MR spectroscopy and CrCEST MRI will be used to measure creatine concentrations within the gastrocnemius-soleus leg muscles at rest, during an exercise protocol, and during post-exercise recovery to baseline. Both measure the recovery time in seconds.", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "Measure intramuscular fat fraction in major muscle groups of the lower extremity in patients with SMA types 2 and 3", + "description": "Measurement of thickness of muscle compared to fat on MRI, measured in percentage.", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "Measure electrophysiological tests of motor neuron function to repetitive nerve stimulation in patients with SMA types 2 and 3", + "description": "Electrophysiological testing: Compound motor action potential (CMAP, measured in millivolts), motor unit number estimate (MUNE, average 200-400 for most limb muscles), and repetitive stimulation at 3 Hz - right ulnar to abductor digiti minimus and right fibular/peroneal nerve to tibialis anterior muscle.\n\nA decrease of more than 40% in the amplitude of CMAP is considered abnormal.", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + } + ], + "secondaryOutcomes": [ + { + "measure": "Identify changes in motor function in non-ambulant patients and ambulant patients with SMA types 2 and 3", + "description": "The Hammersmith Functional Motor Scale Expanded (HFMSE) has a score range from 0-66 with lower scores indicating poorer overall motor function.", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "Identify changes in motor function in non-ambulant patients with SMA types 2 and 3 - Revised Upper Limb Module", + "description": "The Revised Upper Limb Module (RULM) has a score range 0-37 with lower scores indicating poorer upper limb motor function.", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "Identify changes in motor function in non-ambulant patients with SMA types 2 and 3 - Block and Box Test", + "description": "The Block and Box Test (BBT) is a measure of gross manual dexterity and is scored by counting the number of blocks carried from one compartment to another. Higher scores are indicative of better manual dexterity.", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "Identify changes in motor function in ambulant patients with SMA types 2 and 3 - 6-Minute Walk", + "description": "The 6-Minute Walk (6MW) Test measures how far someone can walk in six minutes, a higher score indicates better exercise tolerance score. A low score correlates with lower function.", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "Identify changes in motor function in ambulant patients with SMA types 2 and 3 - 10 Meter Walk/Run", + "description": "The 10 Meter Walk/Run (10MW) Test is measured in seconds and assesses walking speed over a short distance. Longer times indicate lower motor function", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "Identify changes in motor function in ambulant patients with SMA types 2 and 3 - 4-Stair Climb", + "description": "The 4-Stair Climb (4SC) Test is measured in seconds and assesses lower extremity power and motor function. Longer times indicate lower motor function", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "Identify changes in motor function in ambulant patients with SMA types 2 and 3 - Supine-to-Stand Test", + "description": "The Supine-to-Stand (STS) Test is measured in seconds and assesses the ability to move from laying on your back to standing up straight. Longer times indicate lower motor function.", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "Identify changes in motor function in ambulant patients with SMA types 2 and 3 - Timed Up-and-Go Test", + "description": "The Timed Up-and-Go (TUG) Test is measured in seconds and assesses the ability to stand up from sitting in a chair, walk 10 feet, turn around, walk back, and sit down. Longer times indicate lower motor function", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "Assess myometry, or measurement of muscle strength", + "description": "Myometry measures force in pounds or kilograms and measures the strength of a muscle group. The more force measured indicates more muscle strength.", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "Muscle ultrasound thickness and echogenicity of 5 muscles - 2 upper limb and 3 lower limb", + "description": "Muscle thickness is measured in centimeters and echogenicity is measured in gray-scale value. Muscle thickness and echogenicity are related to muscle strength, physical function, muscle mass, and quality of muscle.", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "Plasma neurofilament light (NF-L) and phosphorylated heavy chain (pNF-H) levels", + "description": "The NF-L and pNF-H levels are measured in nanograms per milliliter from blood samples and can be an indicator of nerve cell damage. Higher values normally mean more nerve damage is occurring.", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "Blood SMN protein levels", + "description": "SMN protein levels are measured in nanograms per milliliter from blood samples. Higher values indicate more SMN protein production which is normally deficit in people with SMA.", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "Trough risdiplam drug levels", + "description": "Trough risdiplam drug level is measured in nanograms per milliliter from blood samples and indicates the concentration of risdiplam in someone's blood stream. Higher values mean someone has more risdiplam exposure.\n\nThis will only be tested on participants who are taking the drug risdiplam.", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "Quality of life (QOL) and disability information - Pediatric Quality of Life Inventory (PedsQL)", + "description": "The Pediatric Quality of Life Inventory (PedsQL) is a 23-item questionnaire measuring health-related quality of life in children and adolescents aged 2-18. Higher scores indicate better quality of life.", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "QOL and disability information - Pediatric Outcomes Data Collection Instrument (PODCI)", + "description": "The Pediatric Outcomes Data Collection Instrument (PODCI) is an 83-86 item questionnaire that measures the health-related quality of life for children and adolescents with musculoskeletal disorders aged 2-18. Higher scores indicate better quality of life.", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + }, + { + "measure": "QOL and disability information - SMA Health Index (SMA-HI)", + "description": "The SMA Health Index (SMA-HI) is a 107 item questionnaire that measures a patient's perception of disease burden in 15 areas of health related to SMA. Higher scores indicate more SMA disease burden", + "timeFrame": "At baseline and longitudinally at 6 and 12 months" + } + ] + }, + "eligibilityModule": { + "eligibilityCriteria": "Inclusion Criteria:\n\n* Genetic confirmation of SMA with homozygous deletion of SMN1 or compound heterozygous deletion/mutation of SMN1\n* Two, three, or four copies of SMN2\n* Age 5 to 20 years\n* Non-ambulatory participants: maximum function sitting or standing with support, never walked independently, still able to sit independently for 5 seconds at screening, with active ankle plantar flexion strength of at least 3 N with hand-held myometry and capable of performing repetitive maximal plantar flexion effort for 120 seconds. HFMSE score at screening between 10 and 45 points.\n* Ambulatory participants: minimum function of independent walking, able to walk unassisted a minimum of 100 meters at screening, ankle plantar flexion strength of at least 10 N with hand-held myometry and capable of performing repetitive maximal plantar flexion for 120 seconds. HFMSE score at screening between 40 and 60.\n* SMN-directed therapy inclusion:\n\n * Current Evrysdi prescription\n\n * Must have Evrysdi prescription through their treating physician but have not yet initiated treatment OR\n * Current Spinraza or Zolgensma prescription\n\n * For patients on Spinraza, must have been taking Spinraza for at least 12 months at screening (4 loading and 2 maintenance doses) and following the FDA-recommended dosing schedule\n * For patients on Zolgensma, must have been dosed at least one year prior to screening\n * Must have Spinraza or Zolgensma prescription through their treating physician OR\n * Changing from Spinraza or Zolgensma to Evrysdi\n\n * For patients on Spinraza, must have been taking Spinraza for at least 12 months at screening (4 loading and 2 maintenance doses) and following the FDA-recommended dosing schedule\n * For patients on Zolgensma, must have been dosed at least one year prior to screening\n * Must have voluntarily decided to switch therapies based on discussion with their treating physician\n * Must have Evrysdi prescription through their treating physician but have not yet initiated treatment OR\n * Have never received any SMN-directed therapies\n\nExclusion Criteria:\n\n* Labs at screening that are abnormal and identified as clinically significant by the PI: CBC, and CMP, liver function tests (over twice the upper limit of normal), PT/PTT, urine protein of 2+ or greater.\n* Inability to perform reliably the motor function testing or the exercise testing in the MR scanner.\n* Treatment with a possible SMA-enhancing or mitochondrial-enhancing medication, unless discontinued within 3 months prior to screening: oral albuterol, hydroxyurea, phenylbutryate, valproic acid, creatine, l-carnitine, or other mitochondrial type supplement (riboflavin, lipoic acid, etc.). A daily multivitamin and Vitamin D supplement and intermittent inhaled albuterol are permitted if the dosage is unchanged during the study.\n* Need for routine non-invasive ventilation support.\n* Non-oral nutritional support, e.g., gastrostomy tube feeding.\n* Any ferrous metal implants (e.g., spinal rods) that preclude testing in a MR scanner.", + "sex": "ALL", + "minimumAge": "5 Years", + "maximumAge": "20 Years", + "stdAges": ["CHILD", "ADULT"], + "studyPopulation": "Those who meet the Eligibility criteria and consent to enrollment on the study.", + "samplingMethod": "NON_PROBABILITY_SAMPLE" + }, + "contactsLocationsModule": { + "centralContacts": [ + { + "name": "Jean Laboe, RN", + "role": "CONTACT", + "phone": "901-595-1693", + "email": "referralinfo@stjude.org" + } + ], + "overallOfficials": [ + { + "name": "Richard Finkel, MD", + "affiliation": "St. Jude Children's Research Hospital", + "role": "PRINCIPAL_INVESTIGATOR" + } + ], + "locations": [ + { + "facility": "St. Jude Children's Research Hospital", + "city": "Memphis", + "state": "Tennessee", + "zip": "38105", + "country": "United States", + "contacts": [ + { + "name": "Jean Laboe, RN", + "role": "CONTACT", + "phone": "901-595-1693", + "email": "referralinfo@stjude.org" + }, + { + "name": "Richard Finkel, MD", + "role": "PRINCIPAL_INVESTIGATOR" + } + ], + "geoPoint": { "lat": 35.14953, "lon": -90.04898 } + } + ] + }, + "referencesModule": { + "seeAlsoLinks": [ + { + "label": "Description St. Jude Children's Research Hospital", + "url": "http://www.stjude.org" + }, + { + "label": "Clinical Trials Open at St. Jude", + "url": "http://www.stjude.org/protocols" + } + ] + } + }, + "derivedSection": { + "miscInfoModule": { "versionHolder": "2024-12-04" }, + "conditionBrowseModule": { + "meshes": [ + { "id": "D009133", "term": "Muscular Atrophy" }, + { "id": "D009134", "term": "Muscular Atrophy, Spinal" }, + { "id": "D001284", "term": "Atrophy" } + ], + "ancestors": [ + { "id": "D020763", "term": "Pathological Conditions, Anatomical" }, + { "id": "D020879", "term": "Neuromuscular Manifestations" }, + { "id": "D009461", "term": "Neurologic Manifestations" }, + { "id": "D009422", "term": "Nervous System Diseases" }, + { "id": "D013118", "term": "Spinal Cord Diseases" }, + { "id": "D002493", "term": "Central Nervous System Diseases" }, + { "id": "D016472", "term": "Motor Neuron Disease" }, + { "id": "D019636", "term": "Neurodegenerative Diseases" }, + { "id": "D009468", "term": "Neuromuscular Diseases" } + ], + "browseLeaves": [ + { + "id": "M4589", + "name": "Atrophy", + "asFound": "Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12090", + "name": "Muscular Atrophy", + "asFound": "Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12091", + "name": "Muscular Atrophy, Spinal", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M22519", + "name": "Pathological Conditions, Anatomical", + "relevance": "LOW" + }, + { + "id": "M22619", + "name": "Neuromuscular Manifestations", + "relevance": "LOW" + }, + { + "id": "M12404", + "name": "Neurologic Manifestations", + "relevance": "LOW" + }, + { + "id": "M15915", + "name": "Spinal Cord Diseases", + "relevance": "LOW" + }, + { + "id": "M5742", + "name": "Central Nervous System Diseases", + "relevance": "LOW" + }, + { + "id": "M18879", + "name": "Motor Neuron Disease", + "relevance": "LOW" + }, + { + "id": "M4024", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + }, + { + "id": "M21558", + "name": "Neurodegenerative Diseases", + "relevance": "LOW" + }, + { + "id": "M12411", + "name": "Neuromuscular Diseases", + "relevance": "LOW" + }, + { + "id": "T5342", + "name": "Spinal Muscular Atrophy", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "T349", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + } + ], + "browseBranches": [ + { "abbrev": "BC23", "name": "Symptoms and General Pathology" }, + { "abbrev": "All", "name": "All Conditions" }, + { "abbrev": "BC10", "name": "Nervous System Diseases" }, + { "abbrev": "BC18", "name": "Nutritional and Metabolic Diseases" }, + { "abbrev": "Rare", "name": "Rare Diseases" } + ] + } + }, + "hasResults": false + }, + { + "protocolSection": { + "identificationModule": { + "nctId": "NCT04174157", + "orgStudyIdInfo": { "id": "COAV101A12001" }, + "organization": { "fullName": "Novartis", "class": "INDUSTRY" }, + "briefTitle": "Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)", + "officialTitle": "A Prospective, Long-Term Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)" + }, + "statusModule": { + "statusVerifiedDate": "2024-10", + "overallStatus": "RECRUITING", + "expandedAccessInfo": { + "hasExpandedAccess": true, + "nctId": "NCT03955679", + "statusForNctId": "APPROVED_FOR_MARKETING" + }, + "startDateStruct": { "date": "2018-09-25", "type": "ACTUAL" }, + "primaryCompletionDateStruct": { + "date": "2038-06-30", + "type": "ESTIMATED" + }, + "completionDateStruct": { "date": "2038-06-30", "type": "ESTIMATED" }, + "studyFirstSubmitDate": "2019-09-12", + "studyFirstSubmitQcDate": "2019-11-20", + "studyFirstPostDateStruct": { + "date": "2019-11-22", + "type": "ACTUAL" + }, + "lastUpdateSubmitDate": "2024-10-31", + "lastUpdatePostDateStruct": { "date": "2024-11-04", "type": "ACTUAL" } + }, + "sponsorCollaboratorsModule": { + "responsibleParty": { "type": "SPONSOR" }, + "leadSponsor": { + "name": "Novartis Pharmaceuticals", + "class": "INDUSTRY" + } + }, + "oversightModule": { + "isFdaRegulatedDrug": true, + "isFdaRegulatedDevice": false + }, + "descriptionModule": { + "briefSummary": "Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor neurons. SMA is an autosomal recessive, early childhood disease with an incidence of 1:10,000 live births. SMA is the leading cause of infant mortality due to genetic diseases.\n\nThe purpose of this registry is to assess the long term outcomes of patients with SMA in the context of advances in treatment options and also to characterize and assess long-term safety and effectiveness of OAV-101.", + "detailedDescription": "This is a prospective, multi center, multinational, non-interventional observational study. All patients will be managed according to the clinical site's normal clinical practice, i.e., the diagnostic and clinical treatment/practice process that a clinician chooses according to their clinical judgement for an SMA patient. Clinical care will not be driven by the protocol. No additional visits or investigations will be performed beyond normal clinical practice. Patients will be followed for 15 years from enrolment or until death, whichever is sooner." + }, + "conditionsModule": { "conditions": ["Spinal Muscular Atrophy (SMA)"] }, + "designModule": { + "studyType": "OBSERVATIONAL", + "patientRegistry": true, + "targetDuration": "15 Years", + "designInfo": { + "observationalModel": "ECOLOGIC_OR_COMMUNITY", + "timePerspective": "PROSPECTIVE" + }, + "enrollmentInfo": { "count": 500, "type": "ESTIMATED" } + }, + "armsInterventionsModule": { + "armGroups": [ + { + "label": "Prospective observational registry", + "description": "This is a prospective, multi center, multinational, non-interventional observational registry.", + "interventionNames": [ + "Other: Prospective observational registry", + "Drug: Zolgensma" + ] + } + ], + "interventions": [ + { + "type": "OTHER", + "name": "Prospective observational registry", + "description": "This prospective observational registry will assess long-term outcomes of patients with a diagnosis of SMA.", + "armGroupLabels": ["Prospective observational registry"] + }, + { + "type": "DRUG", + "name": "Zolgensma", + "description": "Zolgensma will be given to patients as per normal clinical practice and clinical care will not be mandated by the protocol. As such, the decision to prescribe Zolgensma is separate from the decision to include the patient in this study", + "armGroupLabels": ["Prospective observational registry"] + } + ] + }, + "outcomesModule": { + "primaryOutcomes": [ + { + "measure": "Change in probability of survival of all patients with SMA using Kaplan Meier method to estimate", + "timeFrame": "Based on information collected at Baseline and every 6 months through 2 years of follow-up, then annually through 15 years of follow up." + }, + { + "measure": "Change from baseline Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) in infants with pre-symptomatic or type I SMA", + "description": "CHOP INTEND score ranges from 0 to 64 with higher scores indicating higher motor function", + "timeFrame": "Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up" + }, + { + "measure": "Change from baseline Hammersmith Infant Neurological Examination (HINE) in infants with pre-symptomatic, type I or type II SMA", + "description": "HINE score range from 0 to 26 with higher scores indicating more development.", + "timeFrame": "Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up" + }, + { + "measure": "Change from baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) for patients with type II and III SMA", + "description": "HFMSE score range from 0 to 66 with the higher scores indicating more development.", + "timeFrame": "Baseline and every 6months through 2 years of follow up, then annually through 15 years of follow up" + }, + { + "measure": "Incidence of treatment emergent adverse events", + "timeFrame": "Through 15 years of follow up" + }, + { + "measure": "Incidence of treatment emergent serious adverse events", + "timeFrame": "Through 15 years of follow up" + }, + { + "measure": "Incidence of treatment emergent adverse events related to therapy", + "timeFrame": "Through 15 years of follow up" + }, + { + "measure": "Incidence of treatment emergent thrombocytopenia, hepatotoxicity and cardiac adverse events", + "timeFrame": "Through 15 years of follow up" + } + ], + "secondaryOutcomes": [ + { + "measure": "Change from baseline in rates of hospitalization", + "timeFrame": "Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up" + }, + { + "measure": "Change from baseline in Zarit Burden Interview", + "description": "Zarit Burden Total Score ranges from 0 to 88. A higher score correlates with higher level of burden.", + "timeFrame": "Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up" + }, + { + "measure": "Change from baseline in PedsQL Patient interview", + "description": "PedsQL Total Scale Score is average of all items and ranges from 0 to 100. A higher score correlates with better Health-Related Quality of Life", + "timeFrame": "Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up" + }, + { + "measure": "Change from baseline in PedsQL Parent interview", + "description": "PedsQL Total Scale Score is average of all items and ranges from 0 to 100. A higher score correlates with better Health-Related Quality of Life", + "timeFrame": "Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up" + }, + { + "measure": "Change from baseline in percent of patients requiring ventilator support (BiPAP, Endotracheal tube)", + "timeFrame": ": Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up" + }, + { + "measure": "Change from baseline in in percent of patients requiring nutritional support (Gastrostomy Tube, Gastrojejunal tube (GT) with Nissen fundoplication, GT without Nissen fundoplication, Nasogastrictube, Nasojejunaltube or Percutaneous endoscopic gastrostomy)", + "timeFrame": "Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up" + }, + { + "measure": "Change from baseline in in percent of patients requiring mobility device support (Ankle-Foot Orthoses, Supramalleolar Orthosis, Orthotic/shoe inserts, Knee immobilizers, Knee-Ankle-Foot Orthoses , Hand splints, Spinal bracing)", + "timeFrame": ": Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up" + } + ] + }, + "eligibilityModule": { + "eligibilityCriteria": "Inclusion Criteria:\n\n* Patients treated with OAV-101 with a genetically confirmed diagnosis of SMA regardless of the date of diagnosis.\n* Appropriate consent/assent has been obtained for participation in the registry\n\nExclusion Criteria:\n\n- Currently enrolled in an interventional clinical trial involving an investigational medicinal product to treat SMA.\n\nNote: Patients who are participating in a Compassionate Use Program (CUP) for OAV-101 (Zolgensma) such as a Managed Access Program (MAP), an Expanded Access Program (EAP), Single Patient Investigational New Drug (IND) (SPI) or Named Patient Program (NPP) are eligible to enroll in the registry regardless of the date of a genetic or clinical diagnosis of SMA.", + "healthyVolunteers": false, + "sex": "ALL", + "stdAges": ["CHILD", "ADULT", "OLDER_ADULT"], + "studyPopulation": "The study will enroll at least 500 patients with a genetically confirmed diagnosis of SMA. 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"email": "margarcia@chla.usc.edu" + }, + { + "name": "Claudia Dozal", + "role": "CONTACT", + "phone": "(323) 361-5825", + "email": "cdozal@chla.usc.edu" + }, + { + "name": "Leigh Maria Ramos-Platt, MD", + "role": "PRINCIPAL_INVESTIGATOR" + } + ], + "geoPoint": { "lat": 34.05223, "lon": -118.24368 } + }, + { + "facility": "University of California Los Angeles Health", + "status": "RECRUITING", + "city": "Los Angeles", + "state": "California", + "zip": "90095", + "country": "United States", + "contacts": [ + { + "name": "Huynh Jennifer", + "role": "CONTACT", + "phone": "310-825-3564", + "email": "JenniferH@mednet.ucla.edu" + }, + { + "name": "Perry Shieh, MD, PhD", + "role": "PRINCIPAL_INVESTIGATOR" + } + ], + "geoPoint": { "lat": 34.05223, "lon": -118.24368 } + }, + { + "facility": "Valley Children's Healthcare", + "status": "RECRUITING", + "city": "Madera", + "state": "California", + "zip": "93636", + "country": "United States", + "contacts": [ + { + "name": "Raymund David, MD", + "role": "CONTACT", + "phone": "559-353-6215", + "email": "RDavid@valleychildrens.org" + }, + { + "name": "Raymund David, MD", + "role": "PRINCIPAL_INVESTIGATOR" + } + ], + "geoPoint": { "lat": 36.96134, "lon": -120.06072 } + }, + { + "facility": "Children's Hospital of Orange County", + "status": "RECRUITING", + "city": "Madera", + "state": "California", + "zip": "93638", + "country": "United States", + "contacts": [ + { + "name": "Amanda Fernandez, MD", + "role": "CONTACT", + "phone": "559-353-6215", + "email": "afernandez@choc.org" + }, + { + "name": "Linda Do", + "role": "CONTACT", + "phone": "(714) 696-2665", + "email": "Linh.Do@choc.org" + }, + { + "name": "Amanda Fernandez, MD", + "role": "PRINCIPAL_INVESTIGATOR" + } + ], + "geoPoint": { "lat": 36.96134, "lon": -120.06072 } + }, + { + "facility": "University of California Davis Health System", + "status": "RECRUITING", + "city": "Sacramento", + "state": "California", + "zip": "95817", + "country": "United States", + "contacts": [ 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"80045", + "country": "United States", + "contacts": [ + { + "name": "Margaret Finlay", + "role": "CONTACT", + "phone": "720-777-1234", + "email": "margaret.finlay@childrenscolorado.org" + }, + { + "name": "Julie Parsons, MD", + "role": "PRINCIPAL_INVESTIGATOR" + } + ], + "geoPoint": { "lat": 39.72943, "lon": -104.83192 } + }, + { + "facility": "Connecticut Children's Medical Center", + "status": "RECRUITING", + "city": "Farmington", + "state": "Connecticut", + "zip": "06034", + "country": "United States", + "contacts": [ + { + "name": "Hendriana Nielsen", + "role": "CONTACT", + "phone": "860-837-5881", + "email": "HNielsen@connecticutchildrens.org" + }, + { + "name": "James Santanelli", + "role": "CONTACT", + "phone": "(860) 837-5873", + "email": "jsantanelli@connecticutchidlrens.org" + }, + { + "name": "Gyula Acsadi, MD, PhD", + "role": "PRINCIPAL_INVESTIGATOR" + } + ], + "geoPoint": { "lat": 41.71982, "lon": -72.83204 } + }, + { + "facility": "Yale-New Haven Health System", + "status": "RECRUITING", + "city": "New Haven", + "state": "Connecticut", + "zip": "06510", + "country": "United States", + "contacts": [ + { + "name": "Catherine Tsao", + "role": "CONTACT", + "phone": "203-785-4390", + "email": "catherine.tsao@yale.edu" + }, + { + "name": "Cristian Ionita, MD", + "role": "PRINCIPAL_INVESTIGATOR" + } + ], + "geoPoint": { "lat": 41.30815, "lon": -72.92816 } + }, + { + "facility": "Golisano Children's Hospital of Southwest Florida", + "status": "RECRUITING", + "city": "Fort Myers", + "state": "Florida", + "zip": "33908", + "country": "United States", + "contacts": [ + { + "name": "Britt Stroud, MD", + "role": "CONTACT", + "phone": "239-343-5437", + "email": "britt.stroud@leehealth.org" + }, + { + "name": "Molly Arnstrom", + "role": "CONTACT", + "phone": "(239) 343-5437", + "email": "Molly.arnstrom@leehealth.org" + }, + { "name": "Britt Stroud, MD", "role": "PRINCIPAL_INVESTIGATOR" } + ], + "geoPoint": { "lat": 26.62168, "lon": -81.84059 } + }, + { + "facility": "Nicklaus Children's Hospital", + "status": "COMPLETED", + "city": "Miami", + "state": "Florida", + "zip": "33155", + "country": "United States", + "geoPoint": { "lat": 25.77427, "lon": -80.19366 } + }, + { + "facility": "Adventist Health System - Florida", + "status": "COMPLETED", + "city": "Orlando", + "state": "Florida", + "zip": "32803", + "country": "United States", + "geoPoint": { "lat": 28.53834, "lon": -81.37924 } + }, + { + "facility": "Jackson South Medical Center", + "status": "RECRUITING", + "city": "Palmetto Bay", + "state": "Florida", + "zip": "33157", + "country": "United States", + "contacts": [ + { + "name": "Paula Schleifer, MD", + "role": "CONTACT", + "phone": "305-279-2226", + "email": "pschleifer@fcneurology.net" + }, + { + "name": "Paula Schleifer, MD", + "role": "PRINCIPAL_INVESTIGATOR" + } + ], + "geoPoint": { "lat": 25.62177, "lon": -80.32477 } + }, + { + "facility": "Indiana University Health University Hospital (IUHUH)", + "status": "RECRUITING", 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"RECRUITING", + "city": "Minneapolis", + "state": "Minnesota", + "zip": "55455", + "country": "United States", + "contacts": [ + { + "name": "Seth Stafki, MD", + "role": "CONTACT", + "phone": "218-298-2747", + "email": "stafk006@umn.edu" + }, + { + "name": "Peter Karachunski, MD", + "role": "PRINCIPAL_INVESTIGATOR" + } + ], + "geoPoint": { "lat": 44.97997, "lon": -93.26384 } + }, + { + "facility": "University of Missouri Health System", + "status": "RECRUITING", + "city": "Columbia", + "state": "Missouri", + "zip": "65212", + "country": "United States", + "contacts": [ + { + "name": "Anne Bonnett", + "role": "CONTACT", + "phone": "573-884-6119", + "email": "bonnetta@health.missouri.edu" + }, + { "name": "Jane Emerson, MD", "role": "PRINCIPAL_INVESTIGATOR" } + ], + "geoPoint": { "lat": 38.95171, "lon": -92.33407 } + }, + { + "facility": "Washington University School of Medicine in St. Louis", + "status": "RECRUITING", + "city": "Saint Louis", + "state": "Missouri", + "zip": "63110", + "country": "United States", + "contacts": [ + { + "name": "Natalie Goedeker", + "role": "CONTACT", + "phone": "314-362-4919", + "email": "ngoedeker@wustl.edu" + }, + { + "name": "Craig Zaidman, MD", + "role": "PRINCIPAL_INVESTIGATOR" + } + ], + "geoPoint": { "lat": 38.62727, "lon": -90.19789 } + }, + { + "facility": "Atlantic Health System", + "status": "RECRUITING", + "city": "Morristown", + "state": "New Jersey", + "zip": "07960", + "country": "United States", + "contacts": [ + { + "name": "Darius Adams, MD", + "role": "CONTACT", + "phone": "518-262-5120", + "email": "adamsd@mail.amc.edu" + }, + { "name": "Darius Adams, MD", "role": "PRINCIPAL_INVESTIGATOR" } + ], + "geoPoint": { "lat": 40.79677, "lon": -74.48154 } + }, + { + "facility": "University of Rochester Medical Center", + "status": "RECRUITING", + "city": "Rochester", + "state": "New York", + "zip": "14642", + "country": "United States", + "contacts": [ + { + "name": "Bohoon Lee", + "role": "CONTACT", + "phone": "585-275-2776", + "email": "Bohoon_Lee@URMC.Rochester.edu" + }, + { + "name": "Kim Hart Kim_", + "role": "CONTACT", + "email": "Hart@URMC.Rochester.edu" + }, + { "name": "Bohoon Lee, MD", "role": "PRINCIPAL_INVESTIGATOR" } + ], + "geoPoint": { "lat": 43.15478, "lon": -77.61556 } + }, + { + "facility": "The State University of New York", + "status": "RECRUITING", + "city": "Stony Brook", + "state": "New York", + "zip": "11794", + "country": "United States", + "contacts": [ + { + "name": "Christine Pol", + "role": "CONTACT", + "phone": "631-444-9083", + "email": "Christiana.Pol@stonybrookmedicine.edu" + }, + { + "name": "Simona Treidler, MD", + "role": "PRINCIPAL_INVESTIGATOR" + } + ], + "geoPoint": { "lat": 40.92565, "lon": -73.14094 } + }, + { + "facility": "Duke Health", + "status": "RECRUITING", + "city": "Durham", + "state": "North Carolina", + "zip": "27705", + "country": "United States", + "contacts": [ + { + "name": "Karen Cornett", + "role": "CONTACT", + "phone": "919-684-1143", + "email": "k.cornett@duke.edu" + }, + { "name": "Mai Elmallah, MD", "role": "PRINCIPAL_INVESTIGATOR" } + ], + "geoPoint": { "lat": 35.99403, "lon": -78.89862 } + }, + { + "facility": "Akron Children's Hospital", + "status": "COMPLETED", + "city": "Akron", + "state": "Ohio", + "zip": "44308-1062", + "country": "United States", + "geoPoint": { "lat": 41.08144, "lon": -81.51901 } + }, + { + "facility": "Cincinnati Children's Hospital Medical Center", + "status": "RECRUITING", + "city": "Cincinnati", + "state": "Ohio", + "zip": "45229-3026", + "country": "United States", + "contacts": [ + { + "name": "Emily Hunsaker", + "role": "CONTACT", + "phone": "513-636-5511", + "email": "emily.hunsaker@cchmc.org" + }, + { "name": "Cuixia Tian, MD", "role": "PRINCIPAL_INVESTIGATOR" } + ], + "geoPoint": { "lat": 39.12713, "lon": -84.51435 } + }, + { + "facility": "University Hospitals", + "status": "COMPLETED", + "city": "Cleveland", + "state": "Ohio", + "zip": "44106", + "country": "United States", + 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Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72,400 specimens. Eur J Hum Genet. 2012 Jan;20(1):27-32. doi: 10.1038/ejhg.2011.134. Epub 2011 Aug 3." + } + ] + }, + "ipdSharingStatementModule": { + "ipdSharing": "UNDECIDED", + "description": "Authorship of planned manuscripts for submission to medical journals shall be determined in accordance with the International Committee of Medical Journal Editors (ICMJE) Uniform Requirements for Manuscripts Submitted to Biomedical Journals (www.icmje.org).\n\nThe physician agrees that if the physician is part of a multi-center registry, the physician shall coordinate in advance any intended disclosure of the results of the registry with Novartis Gene Therapies to ensure that the results of individual physicians are not published or presented before those of the multi-center registry, unless otherwise agreed to in writing by Novartis Gene Therapies." + } + }, + "derivedSection": { + "miscInfoModule": { "versionHolder": "2024-12-04" }, + "conditionBrowseModule": { + "meshes": [ + { "id": "D009133", "term": "Muscular Atrophy" }, + { "id": "D009134", "term": "Muscular Atrophy, 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1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion", + "acronym": "STRIVE-EU" + }, + "statusModule": { + "statusVerifiedDate": "2022-08", + "overallStatus": "COMPLETED", + "expandedAccessInfo": { "hasExpandedAccess": false }, + "startDateStruct": { "date": "2018-08-16", "type": "ACTUAL" }, + "primaryCompletionDateStruct": { + "date": "2020-09-11", + "type": "ACTUAL" + }, + "completionDateStruct": { "date": "2020-09-11", "type": "ACTUAL" }, + "studyFirstSubmitDate": "2018-03-04", + "studyFirstSubmitQcDate": "2018-03-08", + "studyFirstPostDateStruct": { + "date": "2018-03-12", + "type": "ACTUAL" + }, + "resultsFirstSubmitDate": "2021-03-09", + "resultsFirstSubmitQcDate": "2021-03-09", + "resultsFirstPostDateStruct": { + "date": "2021-04-02", + "type": "ACTUAL" + }, + "lastUpdateSubmitDate": "2022-08-12", + "lastUpdatePostDateStruct": { "date": "2022-08-16", "type": "ACTUAL" } + }, + "sponsorCollaboratorsModule": { + "responsibleParty": { "type": "SPONSOR" }, + 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Up to 30 patients \\< 6 months (\\< 180 days) of age at the time of gene replacement therapy (Day 1) will be enrolled.", + "detailedDescription": "Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in patients with spinal muscular atrophy (SMA) Type 1 who meet enrollment criteria and are genetically defined by a biallelic pathogenic mutation of the survival motor neuron 1 gene (SMN1) with one or two copies of survival motor neuron 2 gene (SMN2). 30 patients \\< 6 months (\\< 180 days) of age at the time of gene replacement therapy (Day 1) will be enrolled.\n\nThe trial includes a screening period, a gene replacement therapy period, and a follow-up period. During the screening period (Days -30 to -2), patients whose parent(s)/legal guardian(s) provide informed consent will complete screening procedures to determine eligibility for trial enrollment. Patients who meet the entry criteria will enter the in-patient gene replacement therapy period (Day -1 to Day 3). On Day -1, patients will be admitted to the hospital for pre-treatment baseline procedures. On Day 1, patients will receive a one-time intravenous (IV) infusion of onasemnogene abeparvovec-xioi, and will undergo in-patient safety monitoring over the next 48 hours. Patients may be discharged 48 hours after the infusion, based on Investigator judgment. During the outpatient follow-up period (Days 4 to End of Trial at 18 months of age), patients will return at regularly scheduled intervals for efficacy and safety assessments until the End of Trial when the patient reaches 18 months of age. After the End of Trial visit, eligible patients will be asked to participate into the long-term follow up trial.\n\nAll post-treatment visits will be relative to the date on which gene replacement therapy is administered, until the patient is 14 months of age, after which they will be relevant to the patient's date of birth." + }, + "conditionsModule": { "conditions": ["SMA"] }, + "designModule": { + "studyType": "INTERVENTIONAL", + "phases": ["PHASE3"], + "designInfo": { + "allocation": "NA", + "interventionModel": "SINGLE_GROUP", + "interventionModelDescription": "Open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi", + "primaryPurpose": "TREATMENT", + "maskingInfo": { "masking": "NONE" } + }, + "enrollmentInfo": { "count": 33, "type": "ACTUAL" } + }, + "armsInterventionsModule": { + "armGroups": [ + { + "label": "Onasemnogene Abeparvovec-xioi", + "type": "EXPERIMENTAL", + "description": "Onasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the human survival motor neuron (SMN) gene under the control of the cytomegalovirus (CMV) enhancer/chicken \u03b2-actin-hybrid promoter (CB).", + "interventionNames": ["Biological: Onasemnogene Abeparvovec-xioi"] + } + ], + "interventions": [ + { + "type": "BIOLOGICAL", + "name": "Onasemnogene Abeparvovec-xioi", + "description": "Onasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the human survival motor neuron (SMN) gene under the control of the cytomegalovirus (CMV) enhancer/chicken \u03b2-actin-hybrid promoter (CB).", + "armGroupLabels": ["Onasemnogene Abeparvovec-xioi"], + "otherNames": ["AVXS-101", "OAV101", "Zolgensma"] + } + ] + }, + "outcomesModule": { + "primaryOutcomes": [ + { + "measure": "Number of Participants Who Achieve Independent Sitting for at Least 10 Seconds", + "description": "Independent sitting is defined by the World Health Organization Multicentre Growth Reference Study, confirmed by video recording, as a participant who sits up straight with head erect for at least 10 seconds; participant does not use arms or hands to balance body or support position.", + "timeFrame": "From Day 1 up to 18 Months of Age Visit (Up to a Maximum of Approximately 17 Months)" + } + ], + "secondaryOutcomes": [ + { + "measure": "Event-free Survival at 14 Months of Age", + "description": "Event-free survival at 14 months of age was defined as the number of participants who did not die, did not require permanent ventilation and did not withdraw from the study by 14 months of age.", + "timeFrame": "Up to 14 months of age" + } + ] + }, + "eligibilityModule": { + "eligibilityCriteria": "Inclusion Criteria:\n\n* Patients with SMA Type 1 as determined by diagnosis of SMA based on gene mutation analysis with biallelic SMN1 mutations (deletion or point mutations) and one or two copies of SMN2 \\[inclusive of the known SMN2 gene modifier mutation (c.859G\\>C)\\]\n* Patients must be \\< 6 months (\\< 180 days) of age at the time of onasemnogene abeparvovec-xioi infusion\n* Patients must have a swallowing evaluation test performed prior to administration of gene replacement therapy\n\nExclusion Criteria:\n\n* Previous, planned or expected scoliosis repair surgery/procedure prior to 18 months of age\n* Use of invasive ventilatory support (tracheotomy with positive pressure) or pulse oximetry \\< 95% saturation at screening\n* Use or requirement of non-invasive ventilatory support for 12 or more hours daily in the two weeks prior to dosing\n* Patient with signs of aspiration based on a swallowing test or whose weight-for-age falls below the 3rd percentile based on World Health Organization (WHO) Child Growth Standards and unwilling to use an alternative method to oral feeding\n* Participation in recent SMA treatment clinical trial (with the exception of observational cohort studies or non-interventional studies) or receipt of an investigational or commercial compound, product or therapy administered with the intent to treat SMA (eg, nusinersen, valproic acid,) at any time prior to screening for this trial.", + "healthyVolunteers": false, + "sex": "ALL", + "maximumAge": "6 Months", + "stdAges": ["CHILD"] + }, + "contactsLocationsModule": { + "overallOfficials": [ + { + "name": "AveXis Medinfo", + "affiliation": "Sponsor GmbH", + "role": "STUDY_CHAIR" + } + ], + "locations": [ + { + "facility": "University Hospital Ghent Neuromuscular reference center", + "city": "Ghent", + "country": "Belgium", + "geoPoint": { "lat": 51.05, "lon": 3.71667 } + }, + { + "facility": "Neurop\u00e9diatrie - Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires", + "city": "Li\u00e8ge", + "country": "Belgium", + "geoPoint": { "lat": 50.63373, "lon": 5.56749 } + }, + { + "facility": "H\u00f4pital Armand Trousseau", + "city": "Paris", + "country": "France", + "geoPoint": { "lat": 48.85341, "lon": 2.3488 } + }, + { + "facility": "Istituto Gianninia Gaslini", + "city": "Genova", + "country": "Italy", + "geoPoint": { "lat": 44.40478, "lon": 8.94438 } + }, + { + "facility": "Policlinico \"G. 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Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial. Lancet Neurol. 2021 Oct;20(10):832-841. doi: 10.1016/S1474-4422(21)00251-9." + }, + { + "pmid": "34383289", + "type": "DERIVED", + "citation": "Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Oct;44(10):1109-1119. doi: 10.1007/s40264-021-01107-6. Epub 2021 Aug 12. Erratum In: Drug Saf. 2022 Feb;45(2):191-192. doi: 10.1007/s40264-021-01130-7." + } + ], + "seeAlsoLinks": [ + { + "label": "The Novartis Clinical Trial Results", + "url": "https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=17805" + } + ] + }, + "ipdSharingStatementModule": { + "ipdSharing": "YES", + "description": "Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.\n\nThis trial data availability is according to the criteria and process described on https://www.clinicalstudydatarequest.com/.", + "url": "https://www.clinicalstudydatarequest.com/." + } + }, + "resultsSection": { + "participantFlowModule": { + "groups": [ + { + "id": "FG000", + "title": "Onasemnogene Abeparvovec-xioi", + "description": "Participants received a single dose of onasemnogene abeparvovec-xioi administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 \u00d7 10\\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study." + } + ], + "periods": [ + { + "title": "Overall Study", + "milestones": [ + { + "type": "STARTED", + "achievements": [{ "groupId": "FG000", "numSubjects": "33" }] + }, + { + "type": "COMPLETED", + "achievements": [{ "groupId": "FG000", "numSubjects": "32" }] + }, + { + "type": "NOT COMPLETED", + "achievements": [{ "groupId": "FG000", "numSubjects": "1" }] + } + ], + "dropWithdraws": [ + { + "type": "Death", + "reasons": [{ "groupId": "FG000", "numSubjects": "1" }] + } + ] + } + ] + }, + "baselineCharacteristicsModule": { + "groups": [ + { + "id": "BG000", + "title": "Onasemnogene Abeparvovec-xioi", + "description": "Participants received a single dose of onasemnogene abeparvovec-xioi administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 \u00d7 10\\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "BG000", "value": "33" }] + } + ], + "measures": [ + { + "title": "Age, Categorical", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "BG000", "value": "33" }] + } + ], + "categories": [ + { + "title": "<=18 years", + "measurements": [{ "groupId": "BG000", "value": "33" }] + }, + { + "title": "Between 18 and 65 years", + "measurements": [{ "groupId": "BG000", "value": "0" }] + }, + { + "title": ">=65 years", + "measurements": [{ "groupId": "BG000", "value": "0" }] + } + ] + } + ] + }, + { + "title": "Age, Continuous", + "paramType": "MEAN", + "dispersionType": "STANDARD_DEVIATION", + "unitOfMeasure": "months", + "classes": [ + { + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "BG000", "value": "33" }] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "BG000", + "value": "4.055", + "spread": "1.2799" + } + ] + } + ] + } + ] + }, + { + "title": "Sex: Female, Male", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "BG000", "value": "33" }] + } + ], + "categories": [ + { + "title": "Female", + "measurements": [{ "groupId": "BG000", "value": "19" }] + }, + { + "title": "Male", + "measurements": [{ "groupId": "BG000", "value": "14" }] + } + ] + } + ] + }, + { + "title": "Race and Ethnicity Not Collected", + "populationDescription": "Race and Ethnicity were not collected from any participant.", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "BG000", "value": "0" }] + } + ] + } + ] + }, + { + "title": "Region of Enrollment", + "paramType": "NUMBER", + "unitOfMeasure": "participants", + "classes": [ + { + "title": "Belgium", + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "BG000", "value": "33" }] + } + ], + "categories": [ + { "measurements": [{ "groupId": "BG000", "value": "4" }] } + ] + }, + { + "title": "Italy", + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "BG000", "value": "33" }] + } + ], + "categories": [ + { "measurements": [{ "groupId": "BG000", "value": "23" }] } + ] + }, + { + "title": "United Kingdom", + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "BG000", "value": "33" }] + } + ], + "categories": [ + { "measurements": [{ "groupId": "BG000", "value": "4" }] } + ] + }, + { + "title": "France", + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "BG000", "value": "33" }] + } + ], + "categories": [ + { "measurements": [{ "groupId": "BG000", "value": "2" }] } + ] + } + ] + } + ] + }, + "outcomeMeasuresModule": { + "outcomeMeasures": [ + { + "type": "PRIMARY", + "title": "Number of Participants Who Achieve Independent Sitting for at Least 10 Seconds", + "description": "Independent sitting is defined by the World Health Organization Multicentre Growth Reference Study, confirmed by video recording, as a participant who sits up straight with head erect for at least 10 seconds; participant does not use arms or hands to balance body or support position.", + "populationDescription": "The analysis population was the Intent-to-Treat (ITT) population which consisted of symptomatic participants with bi-allelic deletion of SMN1 (exon 7/8 common homozygous deletions) and 2 copies of SMN2 without the known gene modifier mutation (c.859G\\>C) who received an IV infusion of onasemnogene abeparvovec-xioi at less than 180 days of age.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "From Day 1 up to 18 Months of Age Visit (Up to a Maximum of Approximately 17 Months)", + "groups": [ + { + "id": "OG000", + "title": "Onasemnogene Abeparvovec-xioi", + "description": "Participants received a single dose of onasemnogene abeparvovec-xioi administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 \u00d7 10\\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "32" }] + } + ], + "classes": [ + { + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "14" }] } + ] + } + ] + }, + { + "type": "SECONDARY", + "title": "Event-free Survival at 14 Months of Age", + "description": "Event-free survival at 14 months of age was defined as the number of participants who did not die, did not require permanent ventilation and did not withdraw from the study by 14 months of age.", + "populationDescription": "The analysis population was the ITT population which consisted of symptomatic participants with bi-allelic deletion of SMN1 (exon 7/8 common homozygous deletions) and 2 copies of SMN2 without the known gene modifier mutation (c.859G\\>C) who received an IV infusion of onasemnogene abeparvovec-xioi at less than 180 days of age.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "Up to 14 months of age", + "groups": [ + { + "id": "OG000", + "title": "Onasemnogene Abeparvovec-xioi", + "description": "Participants received a single dose of onasemnogene abeparvovec-xioi administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 \u00d7 10\\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "32" }] + } + ], + "classes": [ + { + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "31" }] } + ] + } + ], + "analyses": [ + { + "groupIds": ["OG000"], + "nonInferiorityType": "SUPERIORITY", + "pValue": "<0.0001", + "statisticalMethod": "Fisher Exact", + "paramType": "Difference of Proportion", + "paramValue": "0.71", + "ciPctValue": "95", + "ciNumSides": "TWO_SIDED", + "ciLowerLimit": "0.48", + "ciUpperLimit": "0.87", + "otherAnalysisDescription": "Data for the current study were compared to historical control data (Finkel et al 2014 - PubMed 25080519) where 6 out of 23 (26.1%) babies were alive and did not need mechanical ventilation at 14 months of age." + } + ] + } + ] + }, + "adverseEventsModule": { + "frequencyThreshold": "0", + "timeFrame": "From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)", + "description": "Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.", + "eventGroups": [ + { + "id": "EG000", + "title": "Onasemnogene Abeparvovec-xioi", + "description": "Participants received a single dose of onasemnogene abeparvovec-xioi administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 \u00d7 10\\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study.", + "deathsNumAffected": 1, + "deathsNumAtRisk": 33, + "seriousNumAffected": 19, + "seriousNumAtRisk": 33, + "otherNumAffected": 32, + "otherNumAtRisk": 33 + } + ], + "seriousEvents": [ + { + "term": "Gastrostomy", + "organSystem": "Surgical and medical procedures", + "sourceVocabulary": "MedDRA 23.0", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": 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"numAffected": 20, + "numAtRisk": 33 + } + ] + }, + { + "term": "Crying", + "organSystem": "General disorders", + "sourceVocabulary": "MedDRA 23.0", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 2, + "numAffected": 1, + "numAtRisk": 33 + } + ] + }, + { + "term": "Extravasation", + "organSystem": "General disorders", + "sourceVocabulary": "MedDRA 23.0", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 33 + } + ] + }, + { + "term": "Generalised oedema", + "organSystem": "General disorders", + "sourceVocabulary": "MedDRA 23.0", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 33 + } + ] + }, + { + "term": "Hypothermia", + "organSystem": "General disorders", + "sourceVocabulary": "MedDRA 23.0", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + 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"assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 4, + "numAffected": 4, + "numAtRisk": 33 + } + ] + }, + { + "term": "Ear infection", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 23.0", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 3, + "numAffected": 3, + "numAtRisk": 33 + } + ] + }, + { + "term": "Gastroenteritis", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 23.0", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 3, + "numAffected": 3, + "numAtRisk": 33 + } + ] + }, + { + "term": "Candida infection", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 23.0", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 2, + "numAffected": 2, + "numAtRisk": 33 + } + ] + }, + { + "term": "Impetigo", + 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"Infections and infestations", + "sourceVocabulary": "MedDRA 23.0", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 33 + } + ] + }, + { + "term": "Influenza", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 23.0", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 33 + } + ] + }, + { + "term": "Laryngitis", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 23.0", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 33 + } + ] + }, + { + "term": "Lower respiratory tract infection", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 23.0", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 2, + "numAffected": 1, + "numAtRisk": 33 + } + ] + }, + { + "term": "Otitis media", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 23.0", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 33 + } + ] + }, + { + "term": "Pharyngitis bacterial", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 23.0", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 33 + } + ] + }, + { + "term": "Respiratory tract infection viral", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 23.0", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 33 + } + ] + }, + { + "term": "Roseola", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 23.0", + 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Clinical Trial Results, as a historical control. These full results are available via this link: https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=17805." + }, + "certainAgreement": { + "piSponsorEmployee": false, + "restrictionType": "OTHER", + "restrictiveAgreement": true, + "otherDetails": "Sponsor can review results communications prior to public release \\& has the right to request changes to communications regarding trial results for a period of 30 to 60 days with an additional 6 months, if needed, from the time submitted to the Sponsor for review in order to either remove references to Sponsor's Confidential Information or delay such results communications to permit Sponsor to obtain appropriate Intellectual Property protection as set forth in each of the agreements." + }, + "pointOfContact": { + "title": "EMEA Medical Information", + "organization": "Novartis Gene Therapies EU Limited", + "email": "medinfoemea.gtx@novartis.com", + "phone": "+353 (1) 566-2364" + } + } + }, + "documentSection": { + "largeDocumentModule": { + "largeDocs": [ + { + "typeAbbrev": "Prot", + "hasProtocol": true, + "hasSap": false, + "hasIcf": false, + "label": "Study Protocol", + "date": "2020-06-25", + "uploadDate": "2021-03-04T14:02", + "filename": "Prot_001.pdf", + "size": 1436699 + }, + { + "typeAbbrev": "SAP", + "hasProtocol": false, + "hasSap": true, + "hasIcf": false, + "label": "Statistical Analysis Plan", + "date": "2020-11-03", + "uploadDate": "2021-03-04T13:52", + "filename": "SAP_000.pdf", + "size": 1042261 + } + ] + } + }, + "derivedSection": { + "miscInfoModule": { + "versionHolder": "2024-12-04", + "removedCountries": ["Germany", "Netherlands", "Spain", "Sweden"] + }, + "conditionBrowseModule": { + "meshes": [ + { "id": "D009133", "term": "Muscular Atrophy" }, + { "id": "D009134", "term": "Muscular Atrophy, Spinal" }, + { "id": "D001284", "term": "Atrophy" } + ], + "ancestors": [ + { "id": "D020763", "term": "Pathological Conditions, Anatomical" }, + { "id": "D020879", "term": "Neuromuscular Manifestations" }, + { "id": "D009461", "term": "Neurologic Manifestations" }, + { "id": "D009422", "term": "Nervous System Diseases" }, + { "id": "D013118", "term": "Spinal Cord Diseases" }, + { "id": "D002493", "term": "Central Nervous System Diseases" }, + { "id": "D016472", "term": "Motor Neuron Disease" }, + { "id": "D019636", "term": "Neurodegenerative Diseases" }, + { "id": "D009468", "term": "Neuromuscular Diseases" } + ], + "browseLeaves": [ + { + "id": "M4589", + "name": "Atrophy", + "asFound": "Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12090", + "name": "Muscular Atrophy", + "asFound": "Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12091", + "name": "Muscular Atrophy, Spinal", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M22519", + "name": "Pathological Conditions, Anatomical", + "relevance": "LOW" + }, + { + "id": "M22619", + "name": "Neuromuscular Manifestations", + "relevance": "LOW" + }, + { + "id": 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Diseases" }, + { "abbrev": "BC18", "name": "Nutritional and Metabolic Diseases" }, + { "abbrev": "Rare", "name": "Rare Diseases" } + ] + } + }, + "hasResults": true + }, + { + "protocolSection": { + "identificationModule": { + "nctId": "NCT04042025", + "orgStudyIdInfo": { "id": "AVXS-101-LT-002" }, + "secondaryIdInfos": [ + { "id": "2019-002611-26", "type": "EUDRACT_NUMBER" }, + { "id": "205305", "type": "OTHER", "domain": "JapicCTI" }, + { + "id": "COAV101A12103", + "type": "OTHER", + "domain": "Novartis Pharmaceuticals" + } + ], + "organization": { "fullName": "Novartis", "class": "INDUSTRY" }, + "briefTitle": "Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi", + "officialTitle": "A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101" + }, + "statusModule": { + "statusVerifiedDate": "2024-02", + "overallStatus": "ACTIVE_NOT_RECRUITING", + "expandedAccessInfo": { "hasExpandedAccess": false }, + "startDateStruct": { "date": "2020-02-10", "type": "ACTUAL" }, + "primaryCompletionDateStruct": { + "date": "2035-12-31", + "type": "ESTIMATED" + }, + "completionDateStruct": { "date": "2035-12-31", "type": "ESTIMATED" }, + "studyFirstSubmitDate": "2019-07-31", + "studyFirstSubmitQcDate": "2019-07-31", + "studyFirstPostDateStruct": { + "date": "2019-08-01", + "type": "ACTUAL" + }, + "lastUpdateSubmitDate": "2024-02-21", + "lastUpdatePostDateStruct": { "date": "2024-02-22", "type": "ACTUAL" } + }, + "sponsorCollaboratorsModule": { + "responsibleParty": { "type": "SPONSOR" }, + "leadSponsor": { + "name": "Novartis Gene Therapies", + "class": "INDUSTRY" + } + }, + "oversightModule": { + "oversightHasDmc": true, + "isFdaRegulatedDrug": true, + "isFdaRegulatedDevice": false + }, + "descriptionModule": { + "briefSummary": "This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment." + }, + "conditionsModule": { + "conditions": [ + "Spinal Muscular Atrophy Type I", + "Spinal Muscular Atrophy Type II", + "Spinal Muscular Atrophy Type III", + "SMA" + ], + "keywords": ["Gene replacement"] + }, + "designModule": { + "studyType": "INTERVENTIONAL", + "phases": ["PHASE3"], + "designInfo": { + "allocation": "NA", + "interventionModel": "SINGLE_GROUP", + "primaryPurpose": "TREATMENT", + "maskingInfo": { "masking": "NONE" } + }, + "enrollmentInfo": { "count": 85, "type": "ACTUAL" } + }, + "armsInterventionsModule": { + "armGroups": [ + { + "label": "Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec-xioi", + "type": "OTHER", + "description": "Participants received treatment with IV onasemnogene abeparvovec-xioi in an onasemnogene abeparvovec-xioi or received treatment with IT onasemnogene abeparvovec-xioi in an onasemnogene.", + "interventionNames": ["Biological: Onasemnogene Abeparvovec-xioi"] + } + ], + "interventions": [ + { + "type": "BIOLOGICAL", + "name": "Onasemnogene Abeparvovec-xioi", + "description": "Onasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the cytomegalovirus enhancer/chicken \u03b2-actin-hybrid promoter. Onasemnogene abeparvovec-xioi administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.", + "armGroupLabels": [ + "Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec-xioi" + ], + "otherNames": ["Zolgensma"] + } + ] + }, + "outcomesModule": { + "primaryOutcomes": [ + { + "measure": "Number of Participants Who Reach Developmental Milestones", + "description": "Assessed via the developmental milestone checklist, formed of 10 yes/no questions. The developmental milestones are: head control, sitting with support, sitting without support, sitting without support for 30 seconds, hands-and-knees crawling, pulls to stand, standing with assistance, walking with assistance, standing alone and walking alone.", + "timeFrame": "Up to 5 years" + }, + { + "measure": "Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score", + "description": "The HFMSE was devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE is formed of 33 assessments rated from 0 (unable to perform functional task) to 2 (able to perform functional task unassisted). Higher scores on the total scale of 0-66 indicates higher levels of motor ability.", + "timeFrame": "Up to 5 years" + }, + { + "measure": "Number of Participants Who Experience a Clinically Significant Change From Baseline in Pulmonary Assessment Results and Require Ventilatory Support", + "description": "Participants will receive pulmonary assessments by a pulmonologist or appropriate clinician. Respiratory device data will be reviewed for participants receiving non-invasive ventilatory support.", + "timeFrame": "Up to 15 years" + }, + { + "measure": "Number of Participants Who Experience Swallowing Dysfunction and Require Nutritional Support", + "description": "Assessed via the swallowing function questionnaire, formed of 4 yes/ no questions and 1 body weight question.", + "timeFrame": "Up to 5 years" + }, + { + "measure": "Number of Participants Who Experience a Clinically Significant Change from Baseline in Physical Examination Findings", + "description": "The physical examination includes review of the following systems: head, ears, eyes, nose and throat, lungs/thorax, cardiovascular, abdomen, musculoskeletal, neurologic, dermatologic, lymphatic, and genitourinary. In addition, visual inspection of the spine, back, shoulders, and hips looking for spinal curvature and asymmetry will be carried out. Joints will be assessed for loss of mobility and contractures.", + "timeFrame": "Up to 5 years" + }, + { + "measure": "Number of Participants Who Experience a Clinically Significant Change From Baseline in Vital Signs Measurements", + "description": "Vital sign measurements will include blood pressure, respiratory rate, pulse, axillary temperature, and pulse oximetry.", + "timeFrame": "Up to 5 years" + }, + { + "measure": "Change From Baseline in Height Measurements", + "timeFrame": "Up to 5 years" + }, + { + "measure": "Change From Baseline in Weight Measurements", + "timeFrame": "Up to 5 years" + }, + { + "measure": "Number of Participants Who Experience a Clinically Significant Change From Baseline in Clinical Laboratory Assessments", + "description": "Blood samples will be collected for hematology (including complete blood cell count) and chemistry.", + "timeFrame": "Up to 5 years" + }, + { + "measure": "Number of Participants Who Experience a Clinically Significant Change From Baseline in Cardiac Assessments", + "description": "Cardiac assessments will include a 12-lead electrocardiogram, transthoracic echocardiogram and Troponin-I.", + "timeFrame": "Up to 5 years" + }, + { + "measure": "Number of Participants Who Experience a Clinically Significant Change From Baseline in Observational Phase Questionnaire Results", + "description": "The observational phase questionnaire includes 7 yes/no questions. Observation categories include: adverse events, hospitalizations, concomitant medications, ventilatory support and feeding support.", + "timeFrame": "Year 6 to Year 15" + }, + { + "measure": "Number of Participants Who Experience at Least One Serious Adverse Event (SAE)", + "description": "An SAE is defined as any adverse event (appearance of \\[or worsening of any pre existing\\]) undesirable sign(s), symptom(s), or medical conditions(s) which meets any one of the following criteria:\n\n* Fatal\n* Life-threatening\n* Results in persistent or significant disability/incapacity\n* Constitutes a congenital abnormality or birth defect\n* Requires in-patient hospitalization or prolongation of existing hospitalization\n* Is medically significant e.g. defined as an event that jeopardizes the participant or may require medical or surgical intervention to prevent one of the outcomes listed above", + "timeFrame": "Up to 15 years" + }, + { + "measure": "Number of Participants Who Experience at Least One Adverse Event of Special Interest (AESI)", + "description": "An AESI is defined as an AE occurring during any study phase that fulfills one of the following criteria:\n\n* Hepatotoxicity\n* Thrombotic microangiopathy\n* Cardiac adverse events\n* Dorsal root ganglia toxicity\n* New malignancies\n* New incidence of a neurologic disorder\n* New incidence of an autoimmune disorder\n* New incidence of hematologic disorder", + "timeFrame": "Up to 15 years" + }, + { + "measure": "Change From Baseline in Bayley Scales of Infant and Toddler Development", + "description": "Third Edition (Bayley-III) to be performed in all patients up to 42 months, 15 days of age.", + "timeFrame": "Up to 42 months, 15 days of age" + }, + { + "measure": "Change From Baseline in Revised Upper Limb Module (RULM) Score", + "description": "RULM score is based on a scale from 0 to 37 where lower scores reflect poorer upper limb functional ability.", + "timeFrame": "Up to 5 years" + }, + { + "measure": "Change From Baseline in Cogstate Computerized Cognitive Battery Performed in Age 48 Months and Older", + "description": "The Cogstate Computerized Cognitive Battery consists of the Identification Test (scored 0 (best) to 1.5708 (worst)), the International Shopping List Test (scored 0 (worst) to 999 (best)), the International Shopping List Test-Delayed Recall (scored 0 (worst) to 999 (best)), the One Card Learning Test (scored 0 (worst) to 1.5708 (best)), and the One Back Test (scored 0 (worst) to 1.5708 (best)).", + "timeFrame": "Up to 5 years" + }, + { + "measure": "Change From Baseline in Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5) Performed in All Participants 5 to 21 Years of Age", + "description": "The CELF-5 Following Directions and Sentence Repetition subtests use scoring that varies based on age, but will be administered to participants 5-21 years of age. The Following Directions subtest will be scored from 0-33 with higher score being more advanced and the Recalling Sentences subtest will be scored from 0-78 with higher score being more advanced.", + "timeFrame": "Up to 5 years" + }, + { + "measure": "Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND)", + "description": "ACEND score is based on a scale from 1 to 41 where higher scores represent a better caregiver experience", + "timeFrame": "Up to 5 years" + }, + { + "measure": "Number of Participants With Concomitant Medications Overall and by Type of Medications", + "timeFrame": "Up to 5 years" + }, + { + "measure": "Number of Participants With Other SMA Therapies Overall and by Type of Medications", + "timeFrame": "Year 6 to Year 15" + } + ] + }, + "eligibilityModule": { + "eligibilityCriteria": "Inclusion Criteria:\n\n* Any participant with SMA who received onasemnogene abeparvovec-xioi gene replacement therapy in a Novartis Gene Therapies-sponsored clinical study\n* Participant/parent/legal guardian willing and able to complete the informed consent process and comply with study procedures and visit schedule\n\nExclusion Criteria:\n\n* Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study", + "healthyVolunteers": false, + "sex": "ALL", + "stdAges": ["CHILD", "ADULT", "OLDER_ADULT"] + }, + "contactsLocationsModule": { + "overallOfficials": [ + { + "name": "Sitra Tauscher-Wisniewski, MD", + "affiliation": "Novartis Gene Therapies, Inc.", + "role": "STUDY_CHAIR" + } + ], + "locations": [ + { + "facility": "Stanford University Medical Center", + "city": "Palo Alto", + "state": "California", + "zip": "94304", + "country": "United States", + "geoPoint": { "lat": 37.44188, "lon": -122.14302 } + }, + { + "facility": "Children's Hospital Colorado", + "city": "Aurora", + "state": "Colorado", + "zip": "80045", + "country": "United States", + "geoPoint": { "lat": 39.72943, "lon": -104.83192 } + }, + { + "facility": "Ann Robert H. Lurie Children's Hospital of Chicago", + "city": "Chicago", + "state": "Illinois", + "zip": "60611", + "country": "United States", + "geoPoint": { "lat": 41.85003, "lon": -87.65005 } + }, + { + "facility": "John Hopkins Hospital - David M. 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Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Oct;44(10):1109-1119. doi: 10.1007/s40264-021-01107-6. Epub 2021 Aug 12. Erratum In: Drug Saf. 2022 Feb;45(2):191-192. doi: 10.1007/s40264-021-01130-7." + } + ] + }, + "ipdSharingStatementModule": { "ipdSharing": "YES" } + }, + "derivedSection": { + "miscInfoModule": { + "versionHolder": "2024-12-04", + "removedCountries": ["Korea, Republic of", "Spain"] + }, + "conditionBrowseModule": { + "meshes": [ + { "id": "D009133", "term": "Muscular Atrophy" }, + { "id": "D009134", "term": "Muscular Atrophy, Spinal" }, + { + "id": "D014897", + "term": "Spinal Muscular Atrophies of Childhood" + }, + { "id": "D001284", "term": "Atrophy" } + ], + "ancestors": [ + { "id": "D020763", "term": "Pathological Conditions, Anatomical" }, + { "id": "D020879", "term": "Neuromuscular Manifestations" }, + { "id": "D009461", "term": "Neurologic Manifestations" }, + { "id": "D009422", "term": "Nervous System Diseases" }, + { "id": "D013118", "term": "Spinal Cord Diseases" }, + { "id": "D002493", "term": "Central Nervous System Diseases" }, + { "id": "D016472", "term": "Motor Neuron 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II", + "relevance": "HIGH" + }, + { + "id": "T5343", + "name": "Spinal Muscular Atrophy 1", + "asFound": "Spinal Muscular Atrophy Type I", + "relevance": "HIGH" + }, + { + "id": "T5347", + "name": "Spinal Muscular Atrophy Type 3", + "asFound": "Spinal Muscular Atrophy Type III", + "relevance": "HIGH" + }, + { + "id": "T349", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + } + ], + "browseBranches": [ + { "abbrev": "BC23", "name": "Symptoms and General Pathology" }, + { "abbrev": "All", "name": "All Conditions" }, + { "abbrev": "BC10", "name": "Nervous System Diseases" }, + { + "abbrev": "BC16", + "name": "Diseases and Abnormalities at or Before Birth" + }, + { "abbrev": "BC18", "name": "Nutritional and Metabolic Diseases" }, + { "abbrev": "Rare", "name": "Rare Diseases" } + ] + } + }, + "hasResults": false + }, + { + "protocolSection": { + "identificationModule": { + "nctId": "NCT03837184", + "orgStudyIdInfo": { "id": "AVXS-101-CL-306" }, + "secondaryIdInfos": [ + { "id": "194664", "type": "OTHER", "domain": "JapicCTI" }, + { + "id": "201900208", + "type": "OTHER", + "domain": "Ministry of Food and Drug Safety (South Korea)" + }, + { + "id": "COAV101A12304", + "type": "OTHER", + "domain": "Novartis Pharmaceuticals" + } + ], + "organization": { "fullName": "Novartis", "class": "INDUSTRY" }, + "briefTitle": "Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies", + "officialTitle": "Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion" + }, + "statusModule": { + "statusVerifiedDate": "2022-11", + "overallStatus": "COMPLETED", + "expandedAccessInfo": { "hasExpandedAccess": false }, + "startDateStruct": { "date": "2019-05-31", "type": "ACTUAL" }, + "primaryCompletionDateStruct": { + "date": "2021-06-29", + "type": "ACTUAL" + }, + "completionDateStruct": { "date": "2021-06-29", "type": "ACTUAL" }, + "studyFirstSubmitDate": "2019-02-08", + "studyFirstSubmitQcDate": "2019-02-08", + "studyFirstPostDateStruct": { + "date": "2019-02-12", + "type": "ACTUAL" + }, + "resultsFirstSubmitDate": "2021-12-14", + "resultsFirstSubmitQcDate": "2021-12-14", + "resultsFirstPostDateStruct": { + "date": "2022-01-11", + "type": "ACTUAL" + }, + "lastUpdateSubmitDate": "2022-11-18", + "lastUpdatePostDateStruct": { "date": "2022-11-22", "type": "ACTUAL" } + }, + "sponsorCollaboratorsModule": { + "responsibleParty": { "type": "SPONSOR" }, + "leadSponsor": { + "name": "Novartis Gene Therapies", + "class": "INDUSTRY" + }, + "collaborators": [ + { "name": "PRA Health Sciences", "class": "INDUSTRY" } + ] + }, + "oversightModule": { + "oversightHasDmc": true, + "isFdaRegulatedDrug": true, + "isFdaRegulatedDevice": false, + "isUsExport": true + }, + "descriptionModule": { + "briefSummary": "This is a Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in participants with spinal muscular atrophy (SMA) Type 1 and who are genetically defined by a biallelic pathogenic mutation of the survival motor neuron 1 gene (SMN1) with one or two copies of survival motor neuron 2 gene (SMN2). The primary objective of the study is to evaluate the efficacy of onasemnogene abeparvovec-xioi by assessing the proportion of symptomatic SMA Type 1 participants who achieve the ability to sit unaided for at least 10 seconds up to and including the 18 months of age trial visit. At least 6 participants aged \\< 6 months (\\< 180 days) at the time of gene replacement therapy (Day 1) will be enrolled.", + "detailedDescription": "This is a Phase 3, open-label, single-arm, single-dose trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in participants with SMA Type 1 with one or 2 copies of SMN2. At least 6 participants \\< 6 months (\\< 180 days) of age at the time of gene replacement therapy (Day 1) will be enrolled.\n\nThe trial includes 3 trial periods: screening, gene replacement therapy, and follow-up. During the screening period (Days -30 to -2), participants whose parent(s)/legal guardian(s) provide informed consent will undergo screening procedures to determine eligibility for trial enrollment. participants who meet the entry criteria will enter the in-patient gene replacement therapy period (Day -1 to Day 3). On Day -1, participants will be admitted to the hospital for pre-treatment baseline procedures. On Day 1, participants will receive a one-time intravenous (IV) infusion of the equivalent of onasemnogene abeparvovec-xioi cohort 2 dose received in the AVXS-101-CL-101 trial over approximately 60 minutes and will undergo in-patient safety monitoring over the next 48 hours. Participants may be discharged 48 hours after gene replacement therapy, based on Investigator judgment. During the outpatient follow-up period (Days 4 to End of Trial at 18 months of age), participants will return at regularly scheduled intervals for efficacy and safety assessments until the participant reaches 18 months of age." + }, + "conditionsModule": { + "conditions": ["Spinal Muscular Atrophy Type I"], + "keywords": [ + "Spinal Muscular Atrophy", + "SMN2", + "Onasemnogene Abeparvovec-xioi", + "Gene replacement therapy" + ] + }, + "designModule": { + "studyType": "INTERVENTIONAL", + "phases": ["PHASE3"], + "designInfo": { + "allocation": "NA", + "interventionModel": "SINGLE_GROUP", + "primaryPurpose": "TREATMENT", + "maskingInfo": { "masking": "NONE" } + }, + "enrollmentInfo": { "count": 2, "type": "ACTUAL" } + }, + "armsInterventionsModule": { + "armGroups": [ + { + "label": "Onasemnogene Abeparvovec-xioi", + "type": "EXPERIMENTAL", + "description": "Participants will receive a single dose of onasemnogene abeparvovec-xioi, administered intravenously.", + "interventionNames": ["Biological: Onasemnogene Abeparvovec-xioi"] + } + ], + "interventions": [ + { + "type": "BIOLOGICAL", + "name": "Onasemnogene Abeparvovec-xioi", + "description": "Onasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the human survival motor neuron (SMN) gene under the control of the cytomegalovirus (CMV) enhancer/chicken \u03b2-actin-hybrid promoter (CB). Onasemnogene abeparvovec-xioi will be administered as a one-time intravenous infusion over approximately 60 minutes. Dosage will be determined by the participants weight.", + "armGroupLabels": ["Onasemnogene Abeparvovec-xioi"], + "otherNames": ["Zolgensma"] + } + ] + }, + "outcomesModule": { + "primaryOutcomes": [ + { + "measure": "Number of Participants Who Achieved Sitting Alone for at Least 10 Seconds", + "description": "Independent sitting is defined by the World Health Organization Multicentre Growth Reference Study, confirmed by video recording, as a participant who sits up straight unsupported for at least 10 seconds.", + "timeFrame": "From Baseline up to 18 Months of Age Visit" + } + ], + "secondaryOutcomes": [ + { + "measure": "Event-free Survival at 14 Months of Age", + "description": "Event-free survival at 14 months of age was defined as the number of participants who did not die, did not require permanent ventilation and did not withdraw from the study by 14 months of age.", + "timeFrame": "From Baseline up to 14 Months of Age" + } + ] + }, + "eligibilityModule": { + "eligibilityCriteria": "Inclusion Criteria:\n\n* Participants with SMA Type 1 as determined by diagnosis of SMA based on gene mutation analysis with biallelic SMN1 mutations (deletion or point mutations) and one or 2 copies of SMN2 \\[inclusive of the known SMN2 gene modifier mutation (c.859G\\>C)\\]\n* Participants must be \\< 6 months (\\< 180 days) of age at the time of onasemnogene abeparvovec-xioi infusion\n* Participants must have a swallowing evaluation test performed prior to administration of gene replacement therapy\n* Up-to-date on childhood vaccinations as per local health authorities.\n* Parent(s)/legal guardian(s) willing and able to complete the informed consent process and comply with trial procedures and visit schedule\n\nExclusion Criteria:\n\n* Previous, planned or expected scoliosis repair surgery/procedure prior to 18 months of age\n* Use of invasive ventilatory support (tracheotomy with positive pressure) or pulse oximetry \\< 95% saturation at screening:\n\n 1. Pulse oximetry saturation must not decrease \u2265 4 percentage points between screening and dosing with confirmatory oximetry reading\n 2. Participants may be put on non-invasive ventilatory support for less than 12 hours per day at the discretion of their physician or trial staff\n* Use or requirement of non-invasive ventilatory support for greater than or equal to 12 hours daily in the two weeks prior to dosing\n* Participant with signs of aspiration based on a swallowing test or whose weight-for-age falls below the 3rd percentile based on World Health Organization (WHO) Child Growth Standards and unwilling to use an alternative method to oral feeding\n* Active viral infection (includes human immunodeficiency virus \\[HIV\\] or positive serology for hepatitis B, C, or E, or known Zika virus infection)\n* Serious non-respiratory tract illness requiring systemic treatment and/or hospitalization within 2 weeks prior to screening\n* Upper or lower respiratory infection requiring medical attention, medical intervention, or increase in supportive care of any manner within 4 weeks prior to screening\n* Severe non-pulmonary/respiratory tract infection (eg, pyelonephritis, or meningitis) within 4 weeks before administration of gene replacement therapy or concomitant illness that, in the opinion of the Principal Investigator, creates unnecessary risks for gene replacement such as:\n\n 1. Major renal or hepatic impairment\n 2. Known seizure disorder\n 3. Diabetes mellitus\n 4. Idiopathic hypocalcuria\n 5. Symptomatic cardiomyopathy\n* Known allergy or hypersensitivity to prednisolone or other glucocorticosteroids or their excipients, or human, animal biological raw materials (human transferrin, human insulin, trypsin derived from porcine spleen, bovine derived protein (FBS, bovine milk-derived Benzonase, casamino acid, bovine pancreas), HEK 293 cells, Cosmic Calf Serum, HyQtase) used in manufacturing of onasemnogene abeparvovec-xioi product\n* Concomitant use of any of the following: drugs for treatment of myopathy or neuropathy, agents used to treat diabetes mellitus, or ongoing immunosuppressive therapy, plasmapheresis, immunomodulators such as adalimumab, or immunosuppressive therapy within 3 months prior to gene replacement therapy (e.g., corticosteroids, cyclosporine, tacrolimus, methotrexate, cyclophosphamide, IV immunoglobulin, rituximab)\n* Anti-AAV9 antibody titer \\> 1:50 as determined by Enzyme-linked Immunosorbent Assay (ELISA) binding immunoassay. Should a potential participant demonstrate Anti-AAV9 antibody titer \\> 1:50, he or she may receive retesting within 30 days of the screening period and will be eligible to participate if the Anti-AAV9 antibody titer upon retesting is \u2264 1:50\n* Clinically significant abnormal laboratory values (gamma-glutamyl transpeptidase \\[GGT\\], ALT, AST, total bilirubin \\> 2x the ULN, creatinine \u2265 1.0 mg/dL, hemoglobin \\[Hgb\\] \\< 8 or \\> 18 g/dL; white blood cell \\[WBC\\] \\> 20,000 per cmm) prior to gene replacement therapy. Patients with an elevated bilirubin level that is unequivocally the result of neonatal jaundice shall not be excluded\n* Participation in recent SMA treatment clinical trial (with the exception of observational cohort studies or non-interventional studies) or receipt of an investigational or commercial compound, product or therapy administered with the intent to treat SMA (e.g., nusinersen, valproic acid) at any time prior to screening for this trial. Oral beta-agonists must be discontinued at least 30 days prior to dosing. Inhaled albuterol specifically prescribed for the purposes of respiratory (bronchodilator) management is acceptable and not a contraindication at any time prior to screening for this trial\n* Expectation of major surgical procedures during the trial assessment period (e.g., spinal surgery or tracheostomy)\n* Parent(s)/legal guardian(s) unable or unwilling to comply with trial procedures or inability to travel for repeat visits\n* Parent(s)/legal guardian(s) unwilling to keep trial results/observations confidential or to refrain from posting confidential trial results/observations on social media sites\n* Parent(s)/legal guardian(s) refuses to sign consent form\n* Participants \\< 35 weeks gestational age at time of birth", + "healthyVolunteers": false, + "sex": "ALL", + "minimumAge": "0 Days", + "maximumAge": "6 Months", + "stdAges": ["CHILD"] + }, + "contactsLocationsModule": { + "overallOfficials": [ + { + "name": "Novartis Pharmaceuticals", + "affiliation": "Novartis Pharmaceuticals", + "role": "STUDY_DIRECTOR" + } + ], + "locations": [ + { + "facility": "Tokyo Women's Medical University", + "city": "Tokyo", + "country": "Japan", + "geoPoint": { "lat": 35.6895, "lon": 139.69171 } + }, + { + "facility": "Pusan National University Yangsan Hospital", + "city": "Yangsan", + "state": "Gyeongsangnam-do", + "country": "Korea, Republic of", + "geoPoint": { "lat": 35.34199, "lon": 129.03358 } + }, + { + "facility": "Seoul National University Hospital", + "city": "Seoul", + "country": "Korea, Republic of", + "geoPoint": { "lat": 37.566, "lon": 126.9784 } + }, + { + "facility": "National Taiwan University Hospital", + "city": "Taipei", + "country": "Taiwan", + "geoPoint": { "lat": 25.04776, "lon": 121.53185 } + } + ] + }, + "referencesModule": { + "references": [ + { + "pmid": "34383289", + "type": "DERIVED", + "citation": "Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Oct;44(10):1109-1119. doi: 10.1007/s40264-021-01107-6. Epub 2021 Aug 12. Erratum In: Drug Saf. 2022 Feb;45(2):191-192. doi: 10.1007/s40264-021-01130-7." + } + ], + "seeAlsoLinks": [ + { + "label": "Novartis Clinical Trial Results", + "url": "https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=17903" + } + ] + }, + "ipdSharingStatementModule": { + "ipdSharing": "YES", + "description": "Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. 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"assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 2 + } + ] + }, + { + "term": "Glycosuria", + "organSystem": "Renal and urinary disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 2 + } + ] + }, + { + "term": "Upper respiratory tract infection", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 4, + "numAffected": 1, + "numAtRisk": 2 + } + ] + }, + { + "term": "Eczema", + "organSystem": "Skin and subcutaneous tissue disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 2 + } + ] + }, + { + "term": 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"documentSection": { + "largeDocumentModule": { + "largeDocs": [ + { + "typeAbbrev": "Prot", + "hasProtocol": true, + "hasSap": false, + "hasIcf": false, + "label": "Study Protocol", + "date": "2020-11-11", + "uploadDate": "2021-12-14T13:37", + "filename": "Prot_000.pdf", + "size": 1209562 + }, + { + "typeAbbrev": "SAP", + "hasProtocol": false, + "hasSap": true, + "hasIcf": false, + "label": "Statistical Analysis Plan", + "date": "2021-07-30", + "uploadDate": "2021-12-14T13:43", + "filename": "SAP_001.pdf", + "size": 608622 + } + ] + } + }, + "derivedSection": { + "miscInfoModule": { "versionHolder": "2024-12-04" }, + "conditionBrowseModule": { + "meshes": [ + { "id": "D009133", "term": "Muscular Atrophy" }, + { "id": "D009134", "term": "Muscular Atrophy, Spinal" }, + { + "id": "D014897", + "term": "Spinal Muscular Atrophies of Childhood" + }, + { "id": "D001284", "term": "Atrophy" } + ], + "ancestors": [ + { "id": "D020763", "term": "Pathological Conditions, Anatomical" }, + { "id": "D020879", "term": "Neuromuscular Manifestations" }, + { "id": "D009461", "term": "Neurologic Manifestations" }, + { "id": "D009422", "term": "Nervous System Diseases" }, + { "id": "D013118", "term": "Spinal Cord Diseases" }, + { "id": "D002493", "term": "Central Nervous System Diseases" }, + { "id": "D016472", "term": "Motor Neuron Disease" }, + { "id": "D019636", "term": "Neurodegenerative Diseases" }, + { "id": "D009468", "term": "Neuromuscular Diseases" }, + { + "id": "D020271", + "term": "Heredodegenerative Disorders, Nervous System" + }, + { "id": "D030342", "term": "Genetic Diseases, Inborn" } + ], + "browseLeaves": [ + { + "id": "M4589", + "name": "Atrophy", + "asFound": "Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12090", + "name": "Muscular Atrophy", + "asFound": "Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12091", + "name": "Muscular Atrophy, Spinal", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M17637", + "name": 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"orgStudyIdInfo": { "id": "AVXS-101-MAP-001" }, + "organization": { + "fullName": "United BioSource, LLC", + "class": "INDUSTRY" + }, + "briefTitle": "AveXis Managed Access Program Cohort for Access to AVXS-101", + "officialTitle": "A Managed Access Program (MAP) Cohort Treatment Protocol to Provide AVXS-101 to Patients With a Genetic Diagnosis of Spinal Muscular Atrophy (SMA) With 1, 2 or 3 Copies of SMN2" + }, + "statusModule": { + "statusVerifiedDate": "2020-08", + "overallStatus": "APPROVED_FOR_MARKETING", + "studyFirstSubmitDate": "2019-05-15", + "studyFirstSubmitQcDate": "2019-05-15", + "studyFirstPostDateStruct": { + "date": "2019-05-20", + "type": "ACTUAL" + }, + "lastUpdateSubmitDate": "2020-08-26", + "lastUpdatePostDateStruct": { "date": "2020-08-28", "type": "ACTUAL" } + }, + "sponsorCollaboratorsModule": { + "responsibleParty": { "type": "SPONSOR" }, + "leadSponsor": { + "name": "United BioSource, LLC", + "class": "INDUSTRY" + }, + "collaborators": [{ "name": "AveXis, Inc.", "class": "INDUSTRY" }] + }, + "descriptionModule": { + "briefSummary": "The purpose of this Cohort Treatment Protocol will allow access to AVXS-101 for eligible patients diagnosed with SMA.", + "detailedDescription": "The purpose of this Cohort Treatment Protocol will allow access to AVXS-101 for eligible patients diagnosed with SMA.\n\nThe requesting Physician submits a request for access to drug (often referred to as Compassionate Use) to AveXis which is reviewed and approved by the medical team experienced with the drug and indication. The requesting Physician should refer to the latest Investigator's Brochure (IB) or approved label for overview of drug including: nonclinical and clinical experience, risk and benefits." + }, + "conditionsModule": { + "conditions": ["Spinal Muscular Atrophy"], + "keywords": [ + "Spinal Muscular Atrophy", + "SMA", + "Gene replacement therapy", + "AVXS-101", + "Compassionate Use", + "Managed Access Program", + "MAP" + ] + }, + "designModule": { + "studyType": "EXPANDED_ACCESS", + "nPtrsToThisExpAccNctId": 1, + "expandedAccessTypes": { "treatment": true } + }, + "armsInterventionsModule": { + "interventions": [ + { + "type": "GENETIC", + "name": "AVXS-101", + "description": "AVXS-101 is a non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the human survival motor neuron (SMN) gene under the control of the cytomegalovirus (CMV) enhancer/chicken \u03b2-actin-hybrid promoter (CB). AVXS-101 will be administered as a one-time intravenous infusion over approximately 60 minutes. Dosage will be determined by the participants weight." + } + ] + }, + "eligibilityModule": { + "eligibilityCriteria": "Inclusion Criteria:\n\n1. Patients with SMA must satisfy both a. and b. of the following specified criteria:\n\n 1. Diagnosis of SMA based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and 1, 2, or 3 copies of SMN2.\n 2. Either patients with onset of symptoms prior to \\< 6 months (\\< 180 days) of age or pre-symptomatic patients, less than 6 months of age, with 1, 2, or 3 copies of SMN2 who are not excluded for other reasons.\n2. Weight \u2265 2.6 kg to \u2264 13.5 kg at dosing.\n3. Patients must have a pre-treatment swallowing evaluation test performed prior to administration of AVXS-101.\n4. Patients must have a formal pulmonary evaluation including documentation of non-invasive ventilatory use prior to administration of AVXS-101. Ventilation should be actively managed by an appropriately trained specialist per the published standard of care.\n5. Up-to-date on childhood vaccinations. Seasonal vaccinations and palivizumab prophylaxis (also known as Synagis) to prevent respiratory syncytial virus (RSV) infections have been administered as recommended by the American Academy of Pediatrics.\n6. Parent(s)/legal guardian(s) willing and able to complete the informed consent process and comply with study procedures and visit schedule.\n\nExclusion Criteria:\n\n1. Tracheostomy.\n2. Contraindication to receiving glucocorticosteroids or their excipients.\n3. Anti Adeno Associated Virus Serotype 9 (AAV9) antibody titer \\> 1:50 (or any value reported as elevated for the laboratory) as determined by Enzyme-linked Immunosorbent Assay (ELISA) binding immunoassay. Should a potential patient demonstrate AntiAAV9 antibody titer \\> 1:50, he or she may be retested and will be eligible to participate if the AntiAAV9 antibody titer upon retesting is \u2264 1:50.\n4. Clinically significant abnormal laboratory values for troponin-I, and platelets. ALT, AST, bilirubin or gamma glutamyl transferase (GGT) \\> 2 x the upper limit of normal (ULN) prior to gene replacement therapy that in the judgment of the Treating Physician or AveXis would create too great a risk for the patient to be treated with AVXS-101 or prophylactic prednisolone. Note: Elevated bilirubin \\> 2 x ULN if associated with neonatal jaundice is not considered exclusionary.\n5. Medical conditions, diagnoses (especially cardiac), or on concurrent medications prior to gene replacement therapy that in the judgment of the treating physician or sponsor would create too great a risk for the patient to be treated with AVXS-101 or prophylactic prednisolone.\n6. Participation or expected participation in current treatment clinical study (with the exception of observational cohort studies or non-interventional studies) for an unapproved investigational agent.\n7. Parent(s)/legal guardian(s) unwilling to keep study results/observations confidential or to refrain from posting confidential study results/observations on social media sites.\n8. Parent(s)/legal guardian(s) refuses to sign consent form.", + "sex": "ALL", + "stdAges": ["CHILD", "ADULT", "OLDER_ADULT"] + }, + "contactsLocationsModule": { + "locations": [ + { + "facility": "Nationwide Children's Hospital", + "city": "Columbus", + "state": "Ohio", + "zip": "43205", + "country": "United States", + "geoPoint": { "lat": 39.96118, "lon": -82.99879 } + } + ] + }, + "referencesModule": { + "references": [ + { + "pmid": "29290580", + "type": "BACKGROUND", + "citation": "Mercuri E, Finkel RS, Muntoni F, Wirth B, Montes J, Main M, Mazzone ES, Vitale M, Snyder B, Quijano-Roy S, Bertini E, Davis RH, Meyer OH, Simonds AK, Schroth MK, Graham RJ, Kirschner J, Iannaccone ST, Crawford TO, Woods S, Qian Y, Sejersen T; SMA Care Group. Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. Neuromuscul Disord. 2018 Feb;28(2):103-115. doi: 10.1016/j.nmd.2017.11.005. Epub 2017 Nov 23." + }, + { + "pmid": "29305137", + "type": "BACKGROUND", + "citation": "Finkel RS, Mercuri E, Meyer OH, Simonds AK, Schroth MK, Graham RJ, Kirschner J, Iannaccone ST, Crawford TO, Woods S, Muntoni F, Wirth B, Montes J, Main M, Mazzone ES, Vitale M, Snyder B, Quijano-Roy S, Bertini E, Davis RH, Qian Y, Sejersen T; SMA Care group. Diagnosis and management of spinal muscular atrophy: Part 2: Pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics. Neuromuscul Disord. 2018 Mar;28(3):197-207. doi: 10.1016/j.nmd.2017.11.004. Epub 2017 Nov 23." + }, + { + "pmid": "19736258", + "type": "BACKGROUND", + "citation": "Committee on Infectious Diseases. From the American Academy of Pediatrics: Policy statements--Modified recommendations for use of palivizumab for prevention of respiratory syncytial virus infections. Pediatrics. 2009 Dec;124(6):1694-701. doi: 10.1542/peds.2009-2345. Epub 2009 Sep 7." + }, + { + "pmid": "29091557", + "type": "BACKGROUND", + "citation": "Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Wells C, Cardenas JA, Heitzer MD, Kaspar A, Corcoran S, Braun L, Likhite S, Miranda C, Meyer K, Foust KD, Burghes AHM, Kaspar BK. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy. N Engl J Med. 2017 Nov 2;377(18):1713-1722. doi: 10.1056/NEJMoa1706198." + }, + { + "pmid": "29064732", + "type": "BACKGROUND", + "citation": "Fu H, Meadows AS, Pineda RJ, Kunkler KL, Truxal KV, McBride KL, Flanigan KM, McCarty DM. Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy. Hum Gene Ther Clin Dev. 2017 Dec;28(4):187-196. doi: 10.1089/humc.2017.109. Epub 2017 Oct 24." + } + ], + "seeAlsoLinks": [ + { + "label": "Kroger A, Duchin J, Vazquez M. General Best Practice Guidelines for Immunization (ACIP). Accessed on 2018.", + "url": "http://www.cdc.gov/vaccines/hcp/acip-recs/general-recs/downloads/general-recs.pdf" + } + ] + } + }, + "derivedSection": { + "miscInfoModule": { "versionHolder": "2024-12-04" }, + "conditionBrowseModule": { + "meshes": [ + { "id": "D009133", "term": "Muscular Atrophy" }, + { "id": "D009134", "term": "Muscular Atrophy, Spinal" }, + { "id": "D001284", "term": "Atrophy" } + ], + "ancestors": [ + { "id": "D020763", "term": "Pathological Conditions, Anatomical" }, + { "id": "D020879", "term": "Neuromuscular Manifestations" }, + { "id": "D009461", "term": "Neurologic Manifestations" }, + { "id": "D009422", "term": "Nervous System Diseases" }, + { "id": "D013118", "term": "Spinal Cord Diseases" }, + { "id": "D002493", "term": "Central Nervous System Diseases" }, + { "id": "D016472", "term": "Motor Neuron Disease" }, + { "id": "D019636", "term": "Neurodegenerative Diseases" }, + { "id": "D009468", "term": "Neuromuscular Diseases" } + ], + "browseLeaves": [ + { + "id": "M4589", + "name": "Atrophy", + "asFound": "Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12090", + "name": "Muscular Atrophy", + "asFound": "Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12091", + "name": "Muscular Atrophy, Spinal", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M22519", + "name": "Pathological Conditions, Anatomical", + "relevance": "LOW" + }, + { + "id": "M22619", + "name": "Neuromuscular Manifestations", + "relevance": "LOW" + }, + { + "id": "M12404", + "name": "Neurologic Manifestations", + "relevance": "LOW" + }, + { + "id": "M15915", + "name": "Spinal Cord Diseases", + "relevance": "LOW" + }, + { + "id": "M5742", + "name": "Central Nervous System Diseases", + "relevance": "LOW" + }, + { + "id": "M18879", + "name": "Motor Neuron Disease", + "relevance": "LOW" + }, + { + "id": "M4024", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + }, + { + "id": "M21558", + "name": "Neurodegenerative Diseases", + "relevance": "LOW" + }, + { + "id": "M12411", + "name": "Neuromuscular Diseases", + "relevance": "LOW" + }, + { + "id": "T5342", + "name": "Spinal Muscular Atrophy", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "T349", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + } + ], + "browseBranches": [ + { "abbrev": "BC23", "name": "Symptoms and General Pathology" }, + { "abbrev": "All", "name": "All Conditions" }, + { "abbrev": "BC10", "name": "Nervous System Diseases" }, + { "abbrev": "BC18", "name": "Nutritional and Metabolic Diseases" }, + { "abbrev": "Rare", "name": "Rare Diseases" } + ] + } + }, + "hasResults": false + }, + { + "protocolSection": { + "identificationModule": { + "nctId": "NCT03505099", + "orgStudyIdInfo": { "id": "AVXS-101-CL-304" }, + "secondaryIdInfos": [ + { "id": "2017-004087-35", "type": "EUDRACT_NUMBER" }, + { + "id": "JapicCTI-184203", + "type": "REGISTRY", + "domain": "JapicCTI" + }, + { + "id": "COAV101A12303", + "type": "OTHER", + "domain": "Novartis Pharmaceuticals" + } + ], + "organization": { "fullName": "Novartis", "class": "INDUSTRY" }, + "briefTitle": "Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2", + "officialTitle": "A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants With Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy With Multiple Copies of SMN2", + "acronym": "SPR1NT" + }, + "statusModule": { + "statusVerifiedDate": "2022-08", + "overallStatus": "COMPLETED", + "expandedAccessInfo": { "hasExpandedAccess": false }, + "startDateStruct": { "date": "2018-04-02", "type": "ACTUAL" }, + "primaryCompletionDateStruct": { + "date": "2021-06-15", + "type": "ACTUAL" + }, + "completionDateStruct": { "date": "2021-06-15", "type": "ACTUAL" }, + "studyFirstSubmitDate": "2018-04-13", + "studyFirstSubmitQcDate": "2018-04-13", + "studyFirstPostDateStruct": { + "date": "2018-04-23", + "type": "ACTUAL" + }, + "resultsFirstSubmitDate": "2021-12-14", + "resultsFirstSubmitQcDate": "2021-12-14", + "resultsFirstPostDateStruct": { + "date": "2022-01-11", + "type": "ACTUAL" + }, + "lastUpdateSubmitDate": "2022-08-12", + "lastUpdatePostDateStruct": { "date": "2022-09-07", "type": "ACTUAL" } + }, + "sponsorCollaboratorsModule": { + "responsibleParty": { "type": "SPONSOR" }, + "leadSponsor": { + "name": "Novartis Gene Therapies", + "class": "INDUSTRY" + }, + "collaborators": [ + { "name": "PRA Health Sciences", "class": "INDUSTRY" } + ] + }, + "oversightModule": { + "oversightHasDmc": true, + "isFdaRegulatedDrug": true, + "isFdaRegulatedDevice": false + }, + "descriptionModule": { + "briefSummary": "To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec-xioi in pre-symptomatic patients with SMA and 2 or 3 copies SMN2", + "detailedDescription": "Phase 3, open-label, single-arm study of a single, one-time dose of onasemnogene abeparvovec-xioi (gene replacement therapy) in patients with spinal muscular atrophy who meet enrollment criteria and are genetically defined by bi-allelic deletion of survival motor neuron 1 gene (SMN1) with 2 or 3 copies of survival motor neuron 2 gene (SMN2). Patients with SMN1 point mutations or the SMN2 gene modifier mutation (c.859G\\>C) may enroll but will not be included in the efficacy analysis sets.\n\nThe study includes a screening period, a gene replacement therapy period, and a follow-up period. During the screening period (Days -30 to -2), patients whose parent(s)/legal guardian(s) provide informed consent will undergo screening procedures to determine eligibility for study enrollment. Patients who meet the entry criteria will enter the in-patient gene replacement therapy period (Day -1 to Day 2). On Day -1, patients will be admitted to the hospital for pre-treatment baseline procedures. On Day 1, patients will receive a single, one-time intravenous (IV) infusion of onasemnogene abeparvovec-xioi, and will undergo in-patient safety monitoring for a minimum of 24 hours post infusion. Patients may be discharged 24 hours (48 hours in Japan) after the infusion, based on Investigator judgment. During the outpatient follow-up period (Days 3 to End of Study at 18 or 24 of age, dependent upon respective SMN2 copy number), patients will return at regularly scheduled intervals for efficacy and safety assessments until the End of Study when the patient reaches 18 months of age (SMN2 = 2) or 24 months of age (SMN2 = 3). After the End of Study visit, eligible patients will be invited to rollover into a long-term follow up study." + }, + "conditionsModule": { + "conditions": ["Spinal Muscular Atrophy"], + "keywords": ["gene therapy"] + }, + "designModule": { + "studyType": "INTERVENTIONAL", + "phases": ["PHASE3"], + "designInfo": { + "allocation": "NA", + "interventionModel": "SINGLE_GROUP", + "interventionModelDescription": "Open-label, single arm", + "primaryPurpose": "TREATMENT", + "maskingInfo": { "masking": "NONE" } + }, + "enrollmentInfo": { "count": 30, "type": "ACTUAL" } + }, + "armsInterventionsModule": { + "armGroups": [ + { + "label": "onasemnogene abeparvovec-xioi", + "type": "EXPERIMENTAL", + "description": "One-time intravenous infusion of onasemnogene abeparvovec-xioi at 1.1 X 10\\^14 vg/kg", + "interventionNames": ["Biological: onasemnogene abeparvovec-xioi"] + } + ], + "interventions": [ + { + "type": "BIOLOGICAL", + "name": "onasemnogene abeparvovec-xioi", + "description": "A non-replicating recombinant AAV9 containing the complimentary deoxyribonucleic acid (cDNA) of the human SMN gene under the control of the cytomegalovirus (CMV) enhancer/chicken-\u03b2-actin-hybrid promoter (CB). The AAV inverted terminal repeat (ITR) has been modified to promote intramolecular annealing of the transgene, thus forming a double-stranded transgene ready for transcription.", + "armGroupLabels": ["onasemnogene abeparvovec-xioi"], + "otherNames": ["Zolgensma"] + } + ] + }, + "outcomesModule": { + "primaryOutcomes": [ + { + "measure": "Cohort 1: Number of Participants Who Achieved Sitting Alone for at Least 30 Seconds", + "description": "Defined by the Bayley Scales of Infant and Toddler Development (BSID) Gross Motor (GM) subtest performance criteria number 26, confirmed by video recording, as a participant who sits for at least 30 seconds without assistance from another person or object. The participant was allowed to use their upper extremities.", + "timeFrame": "From Day 1 up to 18 months of age visit" + }, + { + "measure": "Cohort 2: Number of Participants Who Achieved Standing Alone for at Least 3 Seconds", + "description": "Defined by the BSID GM subtest performance criteria number 40, confirmed by video recording, as a participant who stands alone for at least 3 seconds unsupported.", + "timeFrame": "From Day 1 up to 24 months of age visit" + } + ], + "secondaryOutcomes": [ + { + "measure": "Cohort 1: Event-free Survival at 14 Months of Age", + "description": "Event-free survival at 14 months of age was defined as the number of participants who did not die, did not require permanent ventilation and did not withdraw from the study by 14 months of age.", + "timeFrame": "From Day 1 up to 14 months of age" + }, + { + "measure": "Cohort 1: Number of Participants Who Achieved the Ability to Maintain Weight at or Above the Third Percentile Without the Need for Non-Oral or Mechanical Feeding Support", + "description": "The ability to maintain weight at or above the third percentile without the need for non-oral or mechanical feeding support was defined by meeting the following criteria at each visit up to 18 months of age:\n\n* Did not receive nutrition through mechanical support (i.e., feeding tube)\n* Maintained weight (\u2265 third percentile for age and sex as defined by World Health Organization \\[WHO\\] guidelines) consistent with the participant's age at the assessment.", + "timeFrame": "From Day 1 up to 18 months of age" + }, + { + "measure": "Cohort 2: Number of Participants Who Achieved the Ability to Walk Alone", + "description": "Defined by the BSID GM subtest performance criteria number 43, confirmed by video recording, as a participant who takes 5 coordinated independent steps.", + "timeFrame": "From Day 1 up to 24 months of age visit" + } + ] + }, + "eligibilityModule": { + "eligibilityCriteria": "Inclusion Criteria:\n\n* Age \u22646 weeks (\u226442 days) at time of dose\n* Ability to tolerate thin liquids as demonstrated through a formal bedside swallowing test\n* Compound muscle action potential (CMAP) \u22652mV at Baseline; centralized review of CMAP data will be conducted\n* Gestational age of 35 to 42 weeks\n* Parent(s)/legal guardian(s) willing and able to complete the informed consent process and comply with study procedures and visit schedule\n\n * Patients with pre-symptomatic SMA Type 1 as determined by the following features:\n\n a. 2 copies of SMN2 (n \u226514)\n * Patients with pre-symptomatic SMA Type 2 as determined by the following features:\n\n 1. 3 copies of SMN2 (n \u226512)\n\nExclusion Criteria:\n\n* Weight at screening visit \\<2 kg\n* Hypoxemia (oxygen saturation \\<96% awake or asleep without any supplemental oxygen or respiratory support) at the screening visit or for altitudes \\>1000 m, oxygen saturation \\<92% awake or asleep without any supplemental oxygen or respiratory support at the screening visit\n* Any clinical signs or symptoms at screening or immediately prior to dosing that are, in the opinion of the Investigator, strongly suggestive of SMA\n* Tracheostomy or current prophylactic use or requirement of noninvasive ventilatory support at any time and for any duration prior to screening or during the screening period\n* Patients with signs of aspiration/inability to tolerate nonthickened liquids based on a formal swallowing test performed as part of screening or patients receiving any non-oral feeding method\n* Clinically significant abnormal laboratory values (gamma-glutamyl transferase \\[GGT\\], Alanine transaminase \\[ALT\\], and aspartate aminotransferase \\[AST\\], or total bilirubin \\> 2 \u00d7 the upper limit of normal \\[ULN\\], creatinine \u2265 1.0 mg/dL, hemoglobin \\[Hgb\\] \\< 8 or \\> 18 g/dL; white blood cell \\[WBC\\] \\> 20,000 per cmm) prior to gene replacement therapy. Patients with an elevated bilirubin level that is unequivocally the result of neonatal jaundice shall not be excluded\n* Treatment with an investigational or commercial product, including nusinersen, given for the treatment of SMA. This includes any history of gene therapy, prior antisense oligonucleotide treatment, or cell transplantation.\n* Patients whose weight-for-age is below the third percentile based on World Health Organization (WHO) Child Growth Standards\n* Biological mother with active viral infection as determined by screening laboratory samples (includes human immunodeficiency virus \\[HIV\\] or positive serology for hepatitis B or C)\n\n \u2022 Biological mothers with clinical suspicion of Zika virus that meet Centers for Disease Control and Prevention (CDC) Zika virus epidemiological criteria including history of residence in or travel to a geographic region with active Zika transmission at the time of travel will be tested for Zika virus RNA. Positive results warrant confirmed negative Zika virus RNA testing in the patient prior to enrollment.\n* Serious nonrespiratory tract illness requiring systemic treatment and/or hospitalization within 2 Weeks prior to screening\n* Upper or lower respiratory infection requiring medical attention, medical intervention, or increase in supportive care of any manner within 4 Weeks prior to dosing\n* Severe nonpulmonary/respiratory tract infection within 4 Weeks before administration of gene replacement therapy or concomitant illness that, in the opinion of the Investigator or Sponsor medical monitor, creates unnecessary risks for gene replacement therapy such as:\n\n * Major renal or hepatic impairment\n * Known seizure disorder\n * Diabetes mellitus\n * Idiopathic hypocalciuria\n * Symptomatic cardiomyopathy\n* Known allergy or hypersensitivity to prednisolone or other glucocorticosteroids or their excipients\n* Previous, planned or expected major surgical procedure including scoliosis repair surgery/procedure during the study assessment period\n* Concomitant use of any of the following: drugs for treatment of myopathy or neuropathy, agents used to treat diabetes mellitus, or ongoing immunosuppressive therapy, plasmapheresis, immunomodulators such as adalimumab, immunosuppressive therapy within 4 Weeks prior to gene replacement therapy\n* AntiAAV9 antibody titer \\>1:50 as determined by Enzyme-linked Immunosorbent Assay (ELISA) binding immunoassay\n\n \u2022 Should a potential patient demonstrate AntiAAV9 antibody titer \\>1:50, he or she may receive retesting inside the 30-Day screening period and will be eligible to participate if the AntiAAV9 antibody titer upon retesting is \u22641:50, provided the \\<6 Week age requirement at the time of dosing is still met\n* Biological mother involved with the care of the child refuses anti-AAV9 antibody testing prior to dosing", + "healthyVolunteers": false, + "sex": "ALL", + "maximumAge": "42 Days", + "stdAges": ["CHILD"] + }, + "contactsLocationsModule": { + "overallOfficials": [ + { + "name": "Novartis Pharmaceuticals", + "affiliation": "Novartis Pharmaceuticals", + "role": "STUDY_DIRECTOR" + } + ], + "locations": [ + { + "facility": "David Geffen School of Medicine at UCLA", + "city": "Los Angeles", + "state": "California", + "zip": "90095", + "country": "United States", + "geoPoint": { "lat": 34.05223, "lon": -118.24368 } + }, + { + "facility": "Children's Hospital Colorado", + "city": "Aurora", + "state": "Colorado", + "zip": "80045", + "country": "United States", + "geoPoint": { "lat": 39.72943, "lon": -104.83192 } + }, + { + "facility": "Nemours Children's Hospital", + "city": "Orlando", + "state": "Florida", + "zip": "32827", + "country": "United States", + "geoPoint": { "lat": 28.53834, "lon": -81.37924 } + }, + { + "facility": "Massachusetts General Hospital", + "city": "Boston", + "state": "Massachusetts", + "zip": "02114", + "country": "United States", + "geoPoint": { "lat": 42.35843, "lon": -71.05977 } + }, + { + "facility": "Helen DeVos Children's Hospital", + "city": "Grand Rapids", + "state": "Michigan", + "zip": "49503", + "country": "United States", + "geoPoint": { "lat": 42.96336, "lon": -85.66809 } + }, + { + "facility": "St. Louis Children's Hospital", + "city": "Saint Louis", + "state": "Missouri", + "zip": "63110", + "country": "United States", + "geoPoint": { "lat": 38.62727, "lon": -90.19789 } + }, + { + "facility": "Columbia University Medical Center", + "city": "New York", + "state": "New York", + "zip": "10032", + "country": "United States", + "geoPoint": { "lat": 40.71427, "lon": -74.00597 } + }, + { + "facility": "Nationwide Children's Hospital", + "city": "Columbus", + "state": "Ohio", + "zip": "43205", + "country": "United States", + "geoPoint": { "lat": 39.96118, "lon": -82.99879 } + }, + { + "facility": "Clinic for Special Children", + "city": "Strasburg", + "state": "Pennsylvania", + "zip": "17579", + "country": "United States", + "geoPoint": { "lat": 39.98316, "lon": -76.18412 } + }, + { + "facility": "Children's Medical Center Dallas", + "city": "Dallas", + "state": "Texas", + "zip": "75235", + "country": "United States", + "geoPoint": { "lat": 32.78306, "lon": -96.80667 } + }, + { + "facility": "University Hospital and UW Health Clinics", + "city": "Madison", + "state": "Wisconsin", + "zip": "53792", + "country": "United States", + "geoPoint": { "lat": 43.07305, "lon": -89.40123 } + }, + { + "facility": "Sydney Children's Hospital", + "city": "Randwick", + "state": "New South Wales", + "zip": "2145", + "country": "Australia", + "geoPoint": { "lat": -33.91439, "lon": 151.24895 } + }, + { + "facility": "Centre Hospitalier R\u00e9gional H\u00f4pital La Citadelle", + "city": "Li\u00e8ge", + "zip": "4000", + "country": "Belgium", + "geoPoint": { "lat": 50.63373, "lon": 5.56749 } + }, + { + "facility": "Canada Childrens Hospital of Eastern Ontario", + "city": "Ottawa", + "state": "Ontario", + "zip": "K1H8L1", + "country": "Canada", + "geoPoint": { "lat": 45.41117, "lon": -75.69812 } + }, + { + "facility": "Tokyo Women's Medical", + "city": "Tokyo", + "country": "Japan", + "geoPoint": { "lat": 35.6895, "lon": 139.69171 } + }, + { + "facility": "Great Ormond Street Hospital for Children NHS Foundation Trust", + "city": "London", + "zip": "WC1N 3JH", + "country": "United Kingdom", + "geoPoint": { "lat": 51.50853, "lon": -0.12574 } + } + ] + }, + "referencesModule": { + "references": [ + { + "pmid": "35715567", + "type": "DERIVED", + "citation": "Strauss KA, Farrar MA, Muntoni F, Saito K, Mendell JR, Servais L, McMillan HJ, Finkel RS, Swoboda KJ, Kwon JM, Zaidman CM, Chiriboga CA, Iannaccone ST, Krueger JM, Parsons JA, Shieh PB, Kavanagh S, Wigderson M, Tauscher-Wisniewski S, McGill BE, Macek TA. Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial. Nat Med. 2022 Jul;28(7):1390-1397. doi: 10.1038/s41591-022-01867-3. Epub 2022 Jun 17." + }, + { + "pmid": "35715566", + "type": "DERIVED", + "citation": "Strauss KA, Farrar MA, Muntoni F, Saito K, Mendell JR, Servais L, McMillan HJ, Finkel RS, Swoboda KJ, Kwon JM, Zaidman CM, Chiriboga CA, Iannaccone ST, Krueger JM, Parsons JA, Shieh PB, Kavanagh S, Tauscher-Wisniewski S, McGill BE, Macek TA. Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial. Nat Med. 2022 Jul;28(7):1381-1389. doi: 10.1038/s41591-022-01866-4. Epub 2022 Jun 17." + }, + { + "pmid": "34383289", + "type": "DERIVED", + "citation": "Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Oct;44(10):1109-1119. doi: 10.1007/s40264-021-01107-6. Epub 2021 Aug 12. Erratum In: Drug Saf. 2022 Feb;45(2):191-192. doi: 10.1007/s40264-021-01130-7." + } + ], + "seeAlsoLinks": [ + { + "label": "Novartis Clinical Trial Results", + "url": "https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=17902" + } + ] + }, + "ipdSharingStatementModule": { + "ipdSharing": "YES", + "description": "Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.\n\nThis trial data availability is according to the criteria and process described on https://www.clinicalstudydatarequest.com/.", + "url": "https://www.clinicalstudydatarequest.com/" + } + }, + "resultsSection": { + "participantFlowModule": { + "preAssignmentDetails": "A total of 44 participants were screened, of which 14 were screen failures and 30 were enrolled and received study drug. One participant had 4 copies of SMN2. This participant was excluded from all analysis populations and data is not reported due to privacy concerns.", + "recruitmentDetails": "A total of 30 participants took part in the trial at 16 sites in the United States, the United Kingdom, Belgium, Canada, Australia and Japan between April 2018 and June 2021.", + "groups": [ + { + "id": "FG000", + "title": "Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2", + "description": "Participants with bi-allelic deletions of survival of motor neuron 1 (SMN1) and 2 copies of SMN2 received a single dose of AVXS-101 administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 \u00d7 10\\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1-2 mg/kg/day from 1 day prior to AVXS-101 infusion until at least 30 days post-infusion at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "FG001", + "title": "Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2", + "description": "Participants with bi-allelic deletions of SMN1 and 3 copies of SMN2 received a single dose of AVXS-101 administered as an IV infusion over 60 minutes at a dose of 1.1 \u00d7 10\\^14 vg/kg on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1-2 mg/kg/day from 1 day prior to AVXS-101 infusion until at least 30 days post-infusion at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + } + ], + "periods": [ + { + "title": "Overall Study", + "milestones": [ + { + "type": "STARTED", + "achievements": [ + { "groupId": "FG000", "numSubjects": "14" }, + { "groupId": "FG001", "numSubjects": "15" } + ] + }, + { + "type": "Received AVXS-101", + "achievements": [ + { "groupId": "FG000", "numSubjects": "14" }, + { "groupId": "FG001", "numSubjects": "15" } + ] + }, + { + "type": "COMPLETED", + "achievements": [ + { "groupId": "FG000", "numSubjects": "14" }, + { "groupId": "FG001", "numSubjects": "15" } + ] + }, + { + "type": "NOT COMPLETED", + "achievements": [ + { "groupId": "FG000", "numSubjects": "0" }, + { "groupId": "FG001", "numSubjects": "0" } + ] + } + ] + } + ] + }, + "baselineCharacteristicsModule": { + "groups": [ + { + "id": "BG000", + "title": "Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2", + "description": "Participants with bi-allelic deletions of survival of motor neuron 1 (SMN1) and 2 copies of SMN2 received a single dose of AVXS-101 administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 \u00d7 10\\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1-2 mg/kg/day from 1 day prior to AVXS-101 infusion until at least 30 days post-infusion at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + }, + { + "id": "BG001", + "title": "Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2", + "description": "Participants with bi-allelic deletions of SMN1 and 3 copies of SMN2 received a single dose of AVXS-101 administered as an IV infusion over 60 minutes at a dose of 1.1 \u00d7 10\\^14 vg/kg on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1-2 mg/kg/day from 1 day prior to AVXS-101 infusion until at least 30 days post-infusion at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued" + }, + { + "id": "BG002", + "title": "Total", + "description": "Total of all reporting groups" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "BG000", "value": "14" }, + { "groupId": "BG001", "value": "15" }, + { "groupId": "BG002", "value": "29" } + ] + } + ], + "measures": [ + { + "title": "Age, Categorical", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "title": "<=18 years", + "measurements": [ + { "groupId": "BG000", "value": "14" }, + { "groupId": "BG001", "value": "15" }, + { "groupId": "BG002", "value": "29" } + ] + }, + { + "title": "Between 18 and 65 years", + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" } + ] + }, + { + "title": ">=65 years", + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" } + ] + } + ] + } + ] + }, + { + "title": "Sex: Female, Male", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "title": "Female", + "measurements": [ + { "groupId": "BG000", "value": "10" }, + { "groupId": "BG001", "value": "9" }, + { "groupId": "BG002", "value": "19" } + ] + }, + { + "title": "Male", + "measurements": [ + { "groupId": "BG000", "value": "4" }, + { "groupId": "BG001", "value": "6" }, + { "groupId": "BG002", "value": "10" } + ] + } + ] + } + ] + }, + { + "title": "Ethnicity (NIH/OMB)", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "title": "Hispanic or Latino", + "measurements": [ + { "groupId": "BG000", "value": "4" }, + { "groupId": "BG001", "value": "2" }, + { "groupId": "BG002", "value": "6" } + ] + }, + { + "title": "Not Hispanic or Latino", + "measurements": [ + { "groupId": "BG000", "value": "10" }, + { "groupId": "BG001", "value": "13" }, + { "groupId": "BG002", "value": "23" } + ] + }, + { + "title": "Unknown or Not Reported", + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" } + ] + } + ] + } + ] + }, + { + "title": "Race/Ethnicity, Customized", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "title": "Asian", + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "2" }, + { "groupId": "BG001", "value": "2" }, + { "groupId": "BG002", "value": "4" } + ] + } + ] + }, + { + "title": "American Indian or Alaska Native", + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "1" }, + { "groupId": "BG002", "value": "1" } + ] + } + ] + }, + { + "title": "Black or African American", + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "1" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "1" } + ] + } + ] + }, + { + "title": "White", + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "7" }, + { "groupId": "BG001", "value": "10" }, + { "groupId": "BG002", "value": "17" } + ] + } + ] + }, + { + "title": "Other", + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "4" }, + { "groupId": "BG001", "value": "2" }, + { "groupId": "BG002", "value": "6" } + ] + } + ] + } + ] + }, + { + "title": "SMN2 gene modifier mutation (c.859G>C) Present", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" } + ] + } + ] + } + ] + } + ] + }, + "outcomeMeasuresModule": { + "outcomeMeasures": [ + { + "type": "PRIMARY", + "title": "Cohort 1: Number of Participants Who Achieved Sitting Alone for at Least 30 Seconds", + "description": "Defined by the Bayley Scales of Infant and Toddler Development (BSID) Gross Motor (GM) subtest performance criteria number 26, confirmed by video recording, as a participant who sits for at least 30 seconds without assistance from another person or object. The participant was allowed to use their upper extremities.", + "populationDescription": "Intent-to-Treat (ITT) population (cohort 1) - All enrolled participants with bi-allelic SMN1 deletions and 2 copies of SMN2 without the SMN2 gene modifier mutation (c.859G\\>C) who received AVXS-101.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "From Day 1 up to 18 months of age visit", + "groups": [ + { + "id": "OG000", + "title": "Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2", + "description": "Participants with bi-allelic deletions of survival of motor neuron 1 (SMN1) and 2 copies of SMN2 received a single dose of AVXS-101 administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 \u00d7 10\\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1-2 mg/kg/day from 1 day prior to AVXS-101 infusion until at least 30 days post-infusion at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "14" }] + } + ], + "classes": [ + { + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "14" }] } + ] + } + ] + }, + { + "type": "PRIMARY", + "title": "Cohort 2: Number of Participants Who Achieved Standing Alone for at Least 3 Seconds", + "description": "Defined by the BSID GM subtest performance criteria number 40, confirmed by video recording, as a participant who stands alone for at least 3 seconds unsupported.", + "populationDescription": "ITT population (cohort 2) - All enrolled participants with bi-allelic SMN1 deletions and 3 copies of SMN2 without the SMN2 gene modifier mutation (c.859G\\>C) who received AVXS-101.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "From Day 1 up to 24 months of age visit", + "groups": [ + { + "id": "OG000", + "title": "Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2", + "description": "Participants with bi-allelic deletions of SMN1 and 3 copies of SMN2 received a single dose of AVXS-101 administered as an IV infusion over 60 minutes at a dose of 1.1 \u00d7 10\\^14 vg/kg on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1-2 mg/kg/day from 1 day prior to AVXS-101 infusion until at least 30 days post-infusion at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "15" }] + } + ], + "classes": [ + { + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "15" }] } + ] + } + ], + "analyses": [ + { + "groupIds": ["OG000"], + "nonInferiorityType": "SUPERIORITY", + "pValue": "<0.0001", + "statisticalMethod": "Fisher Exact", + "paramType": "Difference of Proportion", + "paramValue": "76.5", + "ciPctValue": "95", + "ciNumSides": "TWO_SIDED", + "ciLowerLimit": "50.95", + "ciUpperLimit": "92.21", + "otherAnalysisDescription": "Data for the current study were compared to historical control data (Finkel et al, 2014 - PubMed 25080519) where 19 out of 81 participants (23.46%) with 3 copies of SMN2 achieved standing alone for at least 3 seconds." + } + ] + }, + { + "type": "SECONDARY", + "title": "Cohort 1: Event-free Survival at 14 Months of Age", + "description": "Event-free survival at 14 months of age was defined as the number of participants who did not die, did not require permanent ventilation and did not withdraw from the study by 14 months of age.", + "populationDescription": "ITT population (cohort 1) - All enrolled participants with bi-allelic SMN1 deletions and 2 copies of SMN2 without the SMN2 gene modifier mutation (c.859G\\>C) who received AVXS-101.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "From Day 1 up to 14 months of age", + "groups": [ + { + "id": "OG000", + "title": "Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2", + "description": "Participants with bi-allelic deletions of survival of motor neuron 1 (SMN1) and 2 copies of SMN2 received a single dose of AVXS-101 administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 \u00d7 10\\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1-2 mg/kg/day from 1 day prior to AVXS-101 infusion until at least 30 days post-infusion at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "14" }] + } + ], + "classes": [ + { + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "14" }] } + ] + } + ], + "analyses": [ + { + "groupIds": ["OG000"], + "nonInferiorityType": "SUPERIORITY", + "pValue": "<0.0001", + "statisticalMethod": "Fisher Exact", + "paramType": "Difference of Proportion", + "paramValue": "73.9", + "ciPctValue": "95", + "ciNumSides": "TWO_SIDED", + "ciLowerLimit": "44.67", + "ciUpperLimit": "91.61", + "otherAnalysisDescription": "Data for the current study were compared to historical control data (Finkel et al 2014 - PubMed 25080519) where 6 out of 23 participants (26.09%) with 2 copies of SMN2 were alive and did not require permanent ventilation." + } + ] + }, + { + "type": "SECONDARY", + "title": "Cohort 1: Number of Participants Who Achieved the Ability to Maintain Weight at or Above the Third Percentile Without the Need for Non-Oral or Mechanical Feeding Support", + "description": "The ability to maintain weight at or above the third percentile without the need for non-oral or mechanical feeding support was defined by meeting the following criteria at each visit up to 18 months of age:\n\n* Did not receive nutrition through mechanical support (i.e., feeding tube)\n* Maintained weight (\u2265 third percentile for age and sex as defined by World Health Organization \\[WHO\\] guidelines) consistent with the participant's age at the assessment.", + "populationDescription": "ITT population (cohort 1) - All enrolled participants with bi-allelic SMN1 deletions and 2 copies of SMN2 without the SMN2 gene modifier mutation (c.859G\\>C) who received AVXS-101.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "From Day 1 up to 18 months of age", + "groups": [ + { + "id": "OG000", + "title": "Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2", + "description": "Participants with bi-allelic deletions of survival of motor neuron 1 (SMN1) and 2 copies of SMN2 received a single dose of AVXS-101 administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 \u00d7 10\\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1-2 mg/kg/day from 1 day prior to AVXS-101 infusion until at least 30 days post-infusion at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "14" }] + } + ], + "classes": [ + { + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "13" }] } + ] + } + ] + }, + { + "type": "SECONDARY", + "title": "Cohort 2: Number of Participants Who Achieved the Ability to Walk Alone", + "description": "Defined by the BSID GM subtest performance criteria number 43, confirmed by video recording, as a participant who takes 5 coordinated independent steps.", + "populationDescription": "ITT population (cohort 2) - All enrolled participants with bi-allelic SMN1 deletions and 3 copies of SMN2 without the SMN2 gene modifier mutation (c.859G\\>C) who received AVXS-101.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "From Day 1 up to 24 months of age visit", + "groups": [ + { + "id": "OG000", + "title": "Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2", + "description": "Participants with bi-allelic deletions of SMN1 and 3 copies of SMN2 received a single dose of AVXS-101 administered as an IV infusion over 60 minutes at a dose of 1.1 \u00d7 10\\^14 vg/kg on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1-2 mg/kg/day from 1 day prior to AVXS-101 infusion until at least 30 days post-infusion at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "15" }] + } + ], + "classes": [ + { + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "14" }] } + ] + } + ], + "analyses": [ + { + "groupIds": ["OG000"], + "nonInferiorityType": "SUPERIORITY", + "pValue": "<0.0001", + "statisticalMethod": "Fisher Exact", + "paramType": "Difference of Proportion", + "paramValue": "72.3", + "ciPctValue": "95", + "ciNumSides": "TWO_SIDED", + "ciLowerLimit": "44.90", + "ciUpperLimit": "90.11", + "otherAnalysisDescription": "Data for the current study were compared to historical control data (Finkel et al, 2014 - PubMed 25080519) where 17 out of 81 participants (20.99%) with 3 copies of SMN2 achieved the ability to walk alone." + } + ] + } + ] + }, + "adverseEventsModule": { + "frequencyThreshold": "0", + "timeFrame": "Cohort 1: Treatment-emergent adverse events (TEAEs) were collected from Day 1 up to the 18 months of age visit. Serious adverse events (SAEs) were collected from signing of informed consent to 30 days after the last study visit (up to a maximum of approximately 20 months). Cohort 2: TEAEs were collected from Day 1 up to the 24 months of age visit. SAEs were collected from signing of informed consent to 30 days after the last study visit (up to a maximum of approximately 26 months).", + "eventGroups": [ + { + "id": "EG000", + "title": "Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2", + "description": "Participants with bi-allelic deletions of survival of motor neuron 1 (SMN1) and 2 copies of SMN2 received a single dose of AVXS-101 administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 \u00d7 10\\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1-2 mg/kg/day from 1 day prior to AVXS-101 infusion until at least 30 days post-infusion at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued.", + "deathsNumAffected": 0, + "deathsNumAtRisk": 14, + "seriousNumAffected": 5, + "seriousNumAtRisk": 14, + "otherNumAffected": 14, + "otherNumAtRisk": 14 + }, + { + "id": "EG001", + "title": "Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2", + "description": "Participants with bi-allelic deletions of SMN1 and 3 copies of SMN2 received a single dose of AVXS-101 administered as an IV infusion over 60 minutes at a dose of 1.1 \u00d7 10\\^14 vg/kg on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1-2 mg/kg/day from 1 day prior to AVXS-101 infusion until at least 30 days post-infusion at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued.", + "deathsNumAffected": 0, + "deathsNumAtRisk": 15, + "seriousNumAffected": 3, + "seriousNumAtRisk": 15, + "otherNumAffected": 15, + "otherNumAtRisk": 15 + } + ], + "seriousEvents": [ + { + "term": "Middle ear effusion", + "organSystem": "Ear and labyrinth disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 14 + }, + { + "groupId": "EG001", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 15 + } + ] + }, + { + "term": "Inguinal hernia", + "organSystem": "Gastrointestinal disorders", + "sourceVocabulary": "MedDRA (23.0)", + "assessmentType": "SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 14 + }, + { + "groupId": "EG001", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 15 + } + ] + }, + { + "term": "Croup infectious", + 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Patients will be recruited in up to 3 centers in Brazil, over one year of recruitment.", + "samplingMethod": "NON_PROBABILITY_SAMPLE" + }, + "contactsLocationsModule": { + "centralContacts": [ + { + "name": "Novartis Pharmaceuticals", + "role": "CONTACT", + "phone": "+41613241111", + "email": "novartis.email@novartis.com" + }, + { "name": "Novartis Pharmaceuticals", "role": "CONTACT" } + ], + "overallOfficials": [ + { + "name": "Novartis Pharmaceuticals", + "affiliation": "Novartis Pharmaceuticals", + "role": "STUDY_DIRECTOR" + } + ], + "locations": [ + { + "facility": "Novartis Investigative Site", + "status": "RECRUITING", + "city": "Curitiba", + "state": "PR", + "zip": "81520-060", + "country": "Brazil", + "geoPoint": { "lat": -25.42778, "lon": -49.27306 } + }, + { + "facility": "Novartis Investigative Site", + "status": "RECRUITING", + "city": "Sao Paulo", + "state": "SP", + "zip": "05403-000", + "country": "Brazil", + "geoPoint": { "lat": -23.5475, "lon": -46.63611 } + } + ] + }, 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"relevance": "HIGH" + }, + { + "id": "M12090", + "name": "Muscular Atrophy", + "asFound": "Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12091", + "name": "Muscular Atrophy, Spinal", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M22519", + "name": "Pathological Conditions, Anatomical", + "relevance": "LOW" + }, + { + "id": "M22619", + "name": "Neuromuscular Manifestations", + "relevance": "LOW" + }, + { + "id": "M12404", + "name": "Neurologic Manifestations", + "relevance": "LOW" + }, + { + "id": "M15915", + "name": "Spinal Cord Diseases", + "relevance": "LOW" + }, + { + "id": "M5742", + "name": "Central Nervous System Diseases", + "relevance": "LOW" + }, + { + "id": "M18879", + "name": "Motor Neuron Disease", + "relevance": "LOW" + }, + { + "id": "M4024", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + }, + { + "id": "M21558", + "name": "Neurodegenerative Diseases", + "relevance": "LOW" + }, + { + "id": "M12411", + "name": "Neuromuscular Diseases", + "relevance": "LOW" + }, + { + "id": "T5342", + "name": "Spinal Muscular Atrophy", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "T349", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + } + ], + "browseBranches": [ + { "abbrev": "BC23", "name": "Symptoms and General Pathology" }, + { "abbrev": "All", "name": "All Conditions" }, + { "abbrev": "BC10", "name": "Nervous System Diseases" }, + { "abbrev": "BC18", "name": "Nutritional and Metabolic Diseases" }, + { "abbrev": "Rare", "name": "Rare Diseases" } + ] + } + }, + "hasResults": false + }, + { + "protocolSection": { + "identificationModule": { + "nctId": "NCT05089656", + "orgStudyIdInfo": { "id": "COAV101B12301" }, + "secondaryIdInfos": [ + { "id": "2021-003474-31", "type": "EUDRACT_NUMBER" } + ], + "organization": { "fullName": "Novartis", "class": "INDUSTRY" }, + "briefTitle": "Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)", + "officialTitle": "A Randomized, Sham-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Intrathecal OAV101 in Type 2 Spinal Muscular Atrophy (SMA) Patients Who Are \u2265 2 to < 18 Years of Age, Treatment Naive, Sitting, and Never Ambulatory", + "acronym": "STEER" + }, + "statusModule": { + "statusVerifiedDate": "2024-02", + "overallStatus": "ACTIVE_NOT_RECRUITING", + "expandedAccessInfo": { "hasExpandedAccess": false }, + "startDateStruct": { "date": "2022-02-01", "type": "ACTUAL" }, + "primaryCompletionDateStruct": { + "date": "2024-12-02", + "type": "ESTIMATED" + }, + "completionDateStruct": { "date": "2025-02-27", "type": "ESTIMATED" }, + "studyFirstSubmitDate": "2021-10-11", + "studyFirstSubmitQcDate": "2021-10-11", + "studyFirstPostDateStruct": { + "date": "2021-10-22", + "type": "ACTUAL" + }, + "lastUpdateSubmitDate": "2024-02-20", + "lastUpdatePostDateStruct": { "date": "2024-02-22", "type": "ACTUAL" } + }, + "sponsorCollaboratorsModule": { + "responsibleParty": { "type": "SPONSOR" }, + "leadSponsor": { + "name": "Novartis Pharmaceuticals", + "class": "INDUSTRY" + } + }, + "oversightModule": { + "oversightHasDmc": true, + "isFdaRegulatedDrug": true, + "isFdaRegulatedDevice": false + }, + "descriptionModule": { + "briefSummary": "To evaluate the efficacy, safety and tolerability of intrathecal (IT) OAV101 in treatment naive patients with Type 2 spinal muscular atrophy (SMA) who are \u2265 2 to \\< 18 years of age over a 15 month trial duration.", + "detailedDescription": "This is a multi-center, randomized, sham-controlled, double-blind study to investigate the safety, tolerability, and efficacy of IT OAV101 in sitting and never ambulatory SMA participants. The study will enroll treatment naive Type 2 SMA participants who are \u2265 2 years to \\< 18 years. The study consists of a Screening and a Baseline Period followed by a Treatment Period 1 and Follow-up Period 1 (total of 52 weeks) and a Treatment and Follow-up Period 2 (total of 12 weeks). The total trial duration period is 64 weeks.\n\nThe study will include a standard screening period that will last 45 to 60 days, during which eligibility will be assessed and baseline assessments will be performed prior to treatment. Participants who meet eligibility criteria at screening and baseline visits will be randomized in a 3:2 ratio to receive OAV101 by lumbar intrathecal injection or to receive a sham procedure.\n\nTreatment Period 1 consists of OAV101/sham administration with in-patient hospitalization on Study Day 1, Day 2 and Day 3 (optional). Treatment Period 1 is followed by a 52-week out-patient Follow-Up Period 1 for safety and efficacy assessments.\n\nAt the time point each participant completes Follow-up Period 1, those who are eligible will subsequently enter into Treatment Period 2. Entry into Treatment Period 2 will occur immediately after each participant completes their participation in Follow-up Period 1. In Treatment Period 2, eligible participants who received a sham procedure on Study Day 1 of Treatment Period 1 will be hospitalized to receive OAV101 and participants who received OAV101 on Study Day 1 of Treatment Period 1 will be hospitalized to receive a sham procedure on Week 52 +1 Day. A sham procedure is a skin prick in the lumbar region without any medication. In-patient observation will continue on Week 52 +2 Days and Week 52 +3 Days (optional). Treatment Period 2 is followed by a 12-week follow-up period for safety and efficacy assessments. Blinding is maintained for all patients during both Treatment Period 1 and 2. At the end of the study all participants will be eligible to enroll in a long-term follow-up study (15 years) to monitor long-term safety and efficacy.\n\nDuring the study, participants will complete visits as defined in the Schedule of Assessments. Prednisolone or placebo treatment will be given per study protocol.\n\nSafety monitoring will be performed as per study schedule and protocol requirements. Safety for the participants enrolled in the study will be evaluated by a designated group of unblinded study team members together with the Data Monitoring Committee (DMC) as described in the charter.\n\nThe primary analysis will be performed after all participants have completed Week 52 or discontinued prior to Week 52. A final analysis will be performed after all participants have completed Week 64 (or discontinued prior to Week 64)." + }, + "conditionsModule": { + "conditions": ["Type 2 Spinal Muscular Atrophy"], + "keywords": [ + "Zolgensma", + "OAV101", + "AVXS 101", + "gene therapy", + "Muscle atrophy", + "SBMA", + "spinal and bulbar muscular atrophy", + "spinal muscular atrophy", + "bulbar muscular atrophy", + "muscle function", + "myopathy", + "muscle wasting", + "atrophied muscle", + "loss of muscle strength", + "pediatric" + ] + }, + "designModule": { + "studyType": "INTERVENTIONAL", + "phases": ["PHASE3"], + "designInfo": { + "allocation": "RANDOMIZED", + "interventionModel": "CROSSOVER", + "interventionModelDescription": "A participant will receive a single, one-time dose of OAV101.", + "primaryPurpose": "TREATMENT", + "maskingInfo": { + "masking": "QUADRUPLE", + "whoMasked": [ + "PARTICIPANT", + "CARE_PROVIDER", + "INVESTIGATOR", + "OUTCOMES_ASSESSOR" + ] + } + }, + "enrollmentInfo": { "count": 125, "type": "ESTIMATED" } + }, + "armsInterventionsModule": { + "armGroups": [ + { + "label": "OAV101", + "type": "EXPERIMENTAL", + "description": "OAV101 administered as a single, one-time intrathecal dose of 1.2 x 10\\^14 vector genomes (vg).", + "interventionNames": ["Genetic: OAV101"] + }, + { + "label": "Sham control", + "type": "SHAM_COMPARATOR", + "description": "A skin prick in the lumbar region without any medication.", + "interventionNames": ["Procedure: Sham control"] + } + ], + "interventions": [ + { + "type": "GENETIC", + "name": "OAV101", + "description": "Gene therapy", + "armGroupLabels": ["OAV101"], + "otherNames": ["Zolgensma", "AVXS-101"] + }, + { + "type": "PROCEDURE", + "name": "Sham control", + "description": "The sham procedure will consist of a small needle prick on the lower back at the location where the LP injection is normally made. The needle will break the skin, but no needle insertion for lumbar puncture will occur.", + "armGroupLabels": ["Sham control"] + } + ] + }, + "outcomesModule": { + "primaryOutcomes": [ + { + "measure": "Change from baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) total score at the end of Follow-up Period 1 in treated patients compared to sham controls in the \u2265 2 to < 18 years age group", + "description": "The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability.", + "timeFrame": "Baseline up to 52 weeks" + } + ], + "secondaryOutcomes": [ + { + "measure": "Change from baseline in HFMSE total score at the end of Follow-up Period 1 in treated patients compared to sham controls in the \u2265 2 to < 5 years age group", + "description": "The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability.", + "timeFrame": "Baseline up to 52 weeks" + }, + { + "measure": "Change from baseline in Revised Upper Limb Module (RULM) total score at the end of Follow-up Period 1 in treated patients compared to sham controls in the \u2265 2 to < 18 years age group", + "description": "The RULM is a validated SMA specific assessment of motor performance in the upper limbs from childhood through adulthood in ambulatory and non-ambulatory individuals with SMA. The scale consists of 19 scorable items: 18 items scored on 0 (unable) to 2 (full achievement) scale, and one item that is scored from 0 (unable) to 1 (able). Total scores range from 0-37 points. Higher scores reflect higher level of motor ability.", + "timeFrame": "Baseline up to 52 weeks" + }, + { + "measure": "Change from baseline in the RULM total score at the end of Follow-up Period 1 in treated patients compared to sham controls in the \u2265 2 to < 5 years age group", + "description": "The RULM is a validated SMA specific assessment of motor performance in the upper limbs from childhood through adulthood in ambulatory and non-ambulatory individuals with SMA. The scale consists of 19 scorable items: 18 items scored on a 0 (unable) to 2 (full achievement) scale, and one item that is scored from 0 (unable) to 1 (able). Total scores range from 0-37 points. Higher scores reflect higher level of motor ability.", + "timeFrame": "Baseline up to 52 weeks" + }, + { + "measure": "Number of participants with treatment emergent Adverse Events and Serious Adverse Events", + "description": "An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign \\[including abnormal laboratory findings\\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study.\n\nA Treatment Emergent Adverse Event (TEAE) is defined as an event that emerges during treatment, having been absent pretreatment, or worsens relative to the pretreatment state.\n\nThe occurrence of AEs must be sought by non-directive questioning of the participant at each visit during the study. Adverse events also may be detected when they are volunteered by the participant during or between visits or through physical examination findings, laboratory test findings, or other assessments.", + "timeFrame": "Baseline up to 52 weeks" + }, + { + "measure": "Achievement of at least a 3-point improvement from baseline in HFMSE total score at the end of Follow-up Period 1 in the \u2265 2 to < 18 years age group", + "description": "The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability.", + "timeFrame": "Baseline up to 52 weeks" + }, + { + "measure": "Achievement of at least a 3-point improvement from baseline in HFMSE total score at the end of Follow-up Period 1 in the \u2265 2 to < 5 years age group", + "description": "The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability.", + "timeFrame": "Baseline up to 52 weeks" + }, + { + "measure": "Number of participants with adverse events of special interest (AESIs)", + "description": "An AESI is primarily defined by using standard Medical Dictionary for Regulatory Activities (MedDRA) queries, and identified as follows:\n\n* Hepatotoxicity\n* Thrombocytopenia\n* Cardiac adverse events\n* Dorsal Root Ganglia Toxicity\n* Thrombotic microangiopathy", + "timeFrame": "Baseline up to 52 weeks" + }, + { + "measure": "Number (and percentage) of patients with intracardiac thrombi", + "description": "Intracardiac thrombi is defined as the presence of thrombus on post-baseline echocardiograms", + "timeFrame": "Baseline up to 52 weeks" + }, + { + "measure": "Number(and percentage) of patients with low cardiac function", + "description": "Low cardiac function is defined as left ventricular ejection fraction \\<56% or left ventricular fractional shortening \\<28% on post-baseline echocardigrams", + "timeFrame": "Baseline up to 52 weeks" + } + ] + }, + "eligibilityModule": { + "eligibilityCriteria": "Key Inclusion criteria:\n\n* Diagnostic confirmation during screening period of 5q SMA\n* The patient must be treatment naive (historical or current use) for all SMN-targeting therapies (e.g., risdiplam (Evrysdi) and nusinersen (Spinraza)).\n* Onset of clinical signs and symptoms at \u2265 6 months of age\n* A complete Hammersmith Functional Motor Scale - Expanded (HFMSE) assessment during the screening period for trial eligibility\n* Able to sit independently at screening, but has never had the ability to walk independently.\n\nKey Exclusion criteria:\n\n* Anti-adeno-associated virus serotype 9 (AAV9) antibody titer reported as elevated (reference to \\> 1:50 or validated result consistent with being elevated) at screening as determined by sponsor designated lab.\n* Infectious process (e.g. viral, bacterial) or febrile illness prior to start of screening, and up to OAV101 treatment or sham procedure\n* Hepatic dysfunction (i.e. alanine aminotransferase (ALT), total bilirubin, gamma-glutamyl transferase (GGT) or glutamate dehydrogenase (GLDH), \\> upper limit of normal (ULN).\n* Requiring invasive ventilation, awake noninvasive ventilation for \\> 6 hours during a 24-hour period, noninvasive ventilation for \\> 12 hours during a 24-hour period or requiring tracheostomy\n* Complications at screening that would interfere with motor efficacy assessments including but not limited to, severe contractures or Cobb angle \\> 40 in a sitting position\n* Surgery for scoliosis or hip fixation in the 12 months prior to Screening or planned within the next 64 weeks\n* Clinically significant sensory abnormalities in the neurological examination at Screening", + "healthyVolunteers": false, + "sex": "ALL", + "minimumAge": "2 Years", + "maximumAge": "17 Years", + "stdAges": ["CHILD"] + }, + "contactsLocationsModule": { + "locations": [ + { + "facility": "Connecticut Children's Medical Center", + "city": "Farmington", + "state": "Connecticut", + "zip": "06032", + "country": "United States", + "geoPoint": { "lat": 41.71982, "lon": -72.83204 } + }, + { + "facility": "Ann & Robert H. 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These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.\n\nThis trial data availability is according to the criteria and process described on https://www.clinicalstudydatarequest.com/.", + "url": "https://www.clinicalstudydatarequest.com/" + } + }, + "derivedSection": { + "miscInfoModule": { + "versionHolder": "2024-12-04", + "removedCountries": ["Greece"] + }, + "conditionBrowseModule": { + "meshes": [ + { "id": "D009133", "term": "Muscular Atrophy" }, + { "id": "D009134", "term": "Muscular Atrophy, Spinal" }, + { "id": "D001284", "term": "Atrophy" } + ], + "ancestors": [ + { "id": "D020763", "term": "Pathological Conditions, Anatomical" }, + { "id": "D020879", "term": "Neuromuscular Manifestations" }, + { "id": "D009461", "term": "Neurologic Manifestations" }, + { "id": "D009422", "term": "Nervous System Diseases" }, + { "id": "D013118", "term": "Spinal Cord Diseases" }, + { "id": "D002493", "term": "Central Nervous System Diseases" }, + { "id": "D016472", "term": "Motor Neuron Disease" }, + { "id": "D019636", "term": "Neurodegenerative Diseases" }, + { "id": "D009468", "term": "Neuromuscular Diseases" } + ], + "browseLeaves": [ + { "id": "M21265", "name": "Wasting Syndrome", "relevance": "LOW" }, + { "id": "M5363", "name": "Cachexia", "relevance": "LOW" }, + { + "id": "M4589", + "name": "Atrophy", + "asFound": "Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12090", + "name": "Muscular Atrophy", + "asFound": "Muscular Atrophy", + "relevance": "HIGH" + }, + { "id": "M12092", "name": "Muscular Diseases", "relevance": "LOW" }, + { + "id": "M12091", + "name": "Muscular Atrophy, Spinal", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M28216", + "name": "Bulbo-Spinal Atrophy, X-Linked", + "relevance": "LOW" + }, + { + "id": "M22519", + "name": "Pathological Conditions, Anatomical", + "relevance": "LOW" + }, + { + "id": "M22619", + "name": "Neuromuscular Manifestations", + "relevance": "LOW" + }, + { + "id": "M12404", + "name": "Neurologic Manifestations", + "relevance": "LOW" + }, + { + "id": "M15915", + "name": "Spinal Cord Diseases", + "relevance": "LOW" + }, + { + "id": "M5742", + "name": "Central Nervous System Diseases", + "relevance": "LOW" + }, + { + "id": "M18879", + "name": "Motor Neuron Disease", + "relevance": "LOW" + }, + { + "id": "M4024", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + }, + { + "id": "M21558", + "name": "Neurodegenerative Diseases", + "relevance": "LOW" + }, + { + "id": "M12411", + "name": "Neuromuscular Diseases", + "relevance": "LOW" + }, + { "id": "T3215", "name": "Kennedy Disease", "relevance": "LOW" }, + { + "id": "T5342", + "name": "Spinal Muscular Atrophy", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "T349", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + } + ], + "browseBranches": [ + { "abbrev": "BC18", "name": "Nutritional and Metabolic Diseases" }, + { "abbrev": "All", "name": "All Conditions" }, + { "abbrev": "BC23", "name": "Symptoms and General Pathology" }, + { "abbrev": "BC10", "name": "Nervous System Diseases" }, + { "abbrev": "BC05", "name": "Musculoskeletal Diseases" }, + { + "abbrev": "BC16", + "name": "Diseases and Abnormalities at or Before Birth" + }, + { "abbrev": "Rare", "name": "Rare Diseases" } + ] + } + }, + "hasResults": false + }, + { + "protocolSection": { + "identificationModule": { + "nctId": "NCT02122952", + "orgStudyIdInfo": { "id": "AVXS-101-CL-101" }, + "secondaryIdInfos": [ + { + "id": "COAV101A12101", + "type": "OTHER", + "domain": "Novartis Pharmaceuticals" + } + ], + "organization": { "fullName": "Novartis", "class": "INDUSTRY" }, + "briefTitle": "Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1", + "officialTitle": "Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101" + }, + "statusModule": { + "statusVerifiedDate": "2022-08", + "overallStatus": "COMPLETED", + "expandedAccessInfo": { "hasExpandedAccess": false }, + "startDateStruct": { "date": "2014-05-05", "type": "ACTUAL" }, + "primaryCompletionDateStruct": { + "date": "2017-12-15", + "type": "ACTUAL" + }, + "completionDateStruct": { "date": "2017-12-15", "type": "ACTUAL" }, + "studyFirstSubmitDate": "2014-04-23", + "studyFirstSubmitQcDate": "2014-04-23", + "studyFirstPostDateStruct": { + "date": "2014-04-25", + "type": "ESTIMATED" + }, + "resultsFirstSubmitDate": "2018-07-20", + "resultsFirstSubmitQcDate": "2019-02-08", + "resultsFirstPostDateStruct": { + "date": "2019-05-10", + "type": "ACTUAL" + }, + "lastUpdateSubmitDate": "2022-08-30", + "lastUpdatePostDateStruct": { "date": "2022-09-15", "type": "ACTUAL" } + }, + "sponsorCollaboratorsModule": { + "responsibleParty": { "type": "SPONSOR" }, + "leadSponsor": { + "name": "Novartis Gene Therapies", + "class": "INDUSTRY" + } + }, + "oversightModule": { "oversightHasDmc": true }, + "descriptionModule": { + "briefSummary": "The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of AVXS-101 as a treatment of spinal muscular atrophy Type 1 (SMN1).", + "detailedDescription": "The study will evaluate safety and efficacy of gene therapy in spinal muscular atrophy Type 1 (SMA1) patients. SMA is caused by low levels of the survival motor neuron (SMN) protein, and affects all muscles in the body. There is no effective treatment for SMA and current drug therapy has been unsuccessful in stabilizing or reversing this disease. Only supportive care is currently possible.\n\nOpen-label, dose-escalation clinical trial of AVXS-101 injected intravenously through a peripheral limb vein. Short-term safety will be evaluated over a two year period. Patients will be tested at baseline and return for follow up visits on days 7, 14, 21, 30, followed by once every month through 12 months post dose, and then every three months through two (2) years post infusion. Unscheduled visits may occur if the PI determines that they are necessary.\n\nThe primary analysis for efficacy will be assessed when all patients reach 13.6 months of age (a database lock will be performed at the time point at which all patients reach 13.6 months of age). A follow-up safety analysis will be completed at the time point at which the last patient reaches 24 months post-dose.\n\nUpon completion of the 2-year study period, patients will be monitored annually as per standard of care for up to 15 years." + }, + "conditionsModule": { + "conditions": ["Spinal Muscular Atrophy 1"], + "keywords": [ + "Gene Transfer", + "Gene Therapy", + "Adeno-associated virus", + "Survival Motor Neuron", + "SMN", + "AAV9" + ] + }, + "designModule": { + "studyType": "INTERVENTIONAL", + "phases": ["PHASE1"], + "designInfo": { + "allocation": "NON_RANDOMIZED", + "interventionModel": "SINGLE_GROUP", + "primaryPurpose": "TREATMENT", + "maskingInfo": { "masking": "NONE" } + }, + "enrollmentInfo": { "count": 15, "type": "ACTUAL" } + }, + "armsInterventionsModule": { + "armGroups": [ + { + "label": "Cohort 1", + "type": "EXPERIMENTAL", + "description": "6.7 X 10\\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3)", + "interventionNames": ["Biological: AVXS-101"] + }, + { + "label": "Cohort 2", + "type": "EXPERIMENTAL", + "description": "2.0 X 10\\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12)", + "interventionNames": ["Biological: AVXS-101"] + } + ], + "interventions": [ + { + "type": "BIOLOGICAL", + "name": "AVXS-101", + "description": "Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-\u03b2-actin promoter", + "armGroupLabels": ["Cohort 1", "Cohort 2"], + "otherNames": ["Zolgensma"] + } + ] + }, + "outcomesModule": { + "primaryOutcomes": [ + { + "measure": "Number of Participants That Experienced One Grade III or Higher Unanticipated, Treatment-related Toxicity That Presents With Clinical Symptoms and Requires Medical Treatment", + "timeFrame": "2 years" + } + ], + "secondaryOutcomes": [ + { + "measure": "Number of Participants Who Experienced Permanent Ventilation or Death", + "description": "Permanent ventilation was defined as the requirement of \u2265 16-hour respiratory assistance, including non-invasive ventilatory support, per day continuously for \u2265 2 weeks in the absence of an acute reversible illness, excluding perioperative ventilation.", + "timeFrame": "Up to 13.6 months of age" + }, + { + "measure": "Percent Change From Baseline in Mean Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score", + "description": "Score ranges from 0 to 64, where 64 is the maximum possible score. A higher score is indicative of higher/better motor function. CHOP-INTEND assessments were discontinued once patients achieved higher functioning status, so the number of available data points decreased over time.", + "timeFrame": "Baseline to 24 months post-dose" + }, + { + "measure": "Number of Participants With Assessed Improvement in Motor Function", + "description": "Improvement in motor function was determined by achievement of developmental milestones, specifically achievement of ability to sit unassisted for at least 30 seconds, determined by physical therapist and confirmed by an independent central video reviewer. Achievement of functional independent sitting was defined as the ability to maintain a sitting position independently for at least 30 seconds as confirmed per video evaluation by an expert central reviewer based on videos taken either at scheduled visits or provided by the parent/legal guardian.", + "timeFrame": "24 months post-dose" + } + ] + }, + "eligibilityModule": { + "eligibilityCriteria": "Inclusion Criteria:\n\n* Six or nine months of age and younger (depending on cohort) on day of vector infusion with Type 1 SMA as defined by the following features:\n\n * Diagnosis of SMA based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and 2 copies of SMN2.\n * Onset of disease at birth up to 6 months of age.\n * Hypotonia by clinical evaluation with delay in motor skills, poor head control, round shoulder posture and hypermobility of joints.\n\nExclusion Criteria:\n\n* Active viral infection (includes HIV or serology positive for hepatitis B or C)\n* Use of invasive ventilatory support (tracheotomy with positive pressure)\\* or pulse oximetry \\<95% saturation.\n* Patients may be put on non-invasive ventilator support (BiPAP) for less than 16 hours a day at the discretion of their physician or research staff.\n* Concomitant illness that in the opinion of the PI creates unnecessary risks for gene transfer\n* Concomitant use of any of the following drugs: drugs for treatment of myopathy or neuropathy, agents used to treat diabetes mellitus, or ongoing immunosuppressive therapy or immunosuppressive therapy within 3 months of starting the trial (e.g. corticosteroids, cyclosporine, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin, rituximab)\n* Patients with Anti-AAV9 antibody titers \\>1:50 as determined by ELISA binding immunoassay.\n* Abnormal laboratory values considered clinically significant (GGT \\> 3XULN, bilirubin \u2265 3.0 mg/dL , creatinine \u2265 1.8 mg/dL, Hgb \\< 8 or \\> 18 g/Dl; WBC \\> 20,000 per cmm) Participation in a recent SMA treatment clinical trial that in the opinion of the PI creates unnecessary risks for gene transfer.\n* Family does not want to disclose patient's study participation with primary care physician and other medical providers.\n* Patient with signs of aspiration based on a swallowing test and unwilling to use an alternative method to oral feeding.\n* Patients with a single base substitution in SMN2 (c.859G\\>C in exon 7) will be excluded based on predicted mild phenotype.", + "healthyVolunteers": false, + "sex": "ALL", + "maximumAge": "6 Months", + "stdAges": ["CHILD"] + }, + "contactsLocationsModule": { + "overallOfficials": [ + { + "name": "Jerry R Mendell, MD", + "affiliation": "The Research Institute at Nationwide Children's Hospital", + "role": "PRINCIPAL_INVESTIGATOR" + } + ], + "locations": [ + { + "facility": "Nationwide Children's Hospital", + "city": "Columbus", + "state": "Ohio", + "zip": "43205", + "country": "United States", + "geoPoint": { "lat": 39.96118, "lon": -82.99879 } + } + ] + }, + "referencesModule": { + "references": [ + { + "pmid": "21811247", + "type": "BACKGROUND", + "citation": "Bevan AK, Duque S, Foust KD, Morales PR, Braun L, Schmelzer L, Chan CM, McCrate M, Chicoine LG, Coley BD, Porensky PN, Kolb SJ, Mendell JR, Burghes AH, Kaspar BK. Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders. Mol Ther. 2011 Nov;19(11):1971-80. doi: 10.1038/mt.2011.157. Epub 2011 Aug 2." + }, + { + "pmid": "20639395", + "type": "BACKGROUND", + "citation": "Bevan AK, Hutchinson KR, Foust KD, Braun L, McGovern VL, Schmelzer L, Ward JG, Petruska JC, Lucchesi PA, Burghes AH, Kaspar BK. Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery. Hum Mol Genet. 2010 Oct 15;19(20):3895-905. doi: 10.1093/hmg/ddq300. Epub 2010 Jul 16." + }, + { + "pmid": "20190738", + "type": "BACKGROUND", + "citation": "Foust KD, Wang X, McGovern VL, Braun L, Bevan AK, Haidet AM, Le TT, Morales PR, Rich MM, Burghes AH, Kaspar BK. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat Biotechnol. 2010 Mar;28(3):271-4. doi: 10.1038/nbt.1610. Epub 2010 Feb 28.", + "retractions": [ + { + "pmid": "36203015", + "source": "Nat Biotechnol. 2022 Nov;40(11):1692" + } + ] + }, + { + "pmid": "19098898", + "type": "BACKGROUND", + "citation": "Foust KD, Nurre E, Montgomery CL, Hernandez A, Chan CM, Kaspar BK. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol. 2009 Jan;27(1):59-65. doi: 10.1038/nbt.1515. Epub 2008 Dec 21." + }, + { + "pmid": "34383289", + "type": "DERIVED", + "citation": "Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Oct;44(10):1109-1119. doi: 10.1007/s40264-021-01107-6. Epub 2021 Aug 12. Erratum In: Drug Saf. 2022 Feb;45(2):191-192. doi: 10.1007/s40264-021-01130-7." + }, + { + "pmid": "31277975", + "type": "DERIVED", + "citation": "Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019 Sep;98:39-45. doi: 10.1016/j.pediatrneurol.2019.05.005. Epub 2019 May 13." + }, + { + "pmid": "30879249", + "type": "DERIVED", + "citation": "Dabbous O, Maru B, Jansen JP, Lorenzi M, Cloutier M, Guerin A, Pivneva I, Wu EQ, Arjunji R, Feltner D, Sproule DM. Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1. Adv Ther. 2019 May;36(5):1164-1176. doi: 10.1007/s12325-019-00923-8. Epub 2019 Mar 16." + }, + { + "pmid": "30548438", + "type": "DERIVED", + "citation": "Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbous O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb;54(2):179-185. doi: 10.1002/ppul.24203. Epub 2018 Dec 12." + }, + { + "pmid": "29091557", + "type": "DERIVED", + "citation": "Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Wells C, Cardenas JA, Heitzer MD, Kaspar A, Corcoran S, Braun L, Likhite S, Miranda C, Meyer K, Foust KD, Burghes AHM, Kaspar BK. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy. N Engl J Med. 2017 Nov 2;377(18):1713-1722. doi: 10.1056/NEJMoa1706198." + } + ], + "seeAlsoLinks": [ + { + "label": "Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital", + "url": "http://www.nationwidechildrens.org/center-for-gene-therapy" + }, + { "label": "AveXis, Inc", "url": "http://avexis.com/" }, + { + "label": "Novartis Clinical Trial Results", + "url": "https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=17849" + } + ] + } + }, + "resultsSection": { + "participantFlowModule": { + "preAssignmentDetails": "30 day screening period prior to enrollment and dosing.", + "recruitmentDetails": "Recruitment period May 2014 - Dec 2015", + "groups": [ + { + "id": "FG000", + "title": "Cohort 1", + "description": "6.7 X 10\\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3)\n\nAVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-\u03b2-actin promoter" + }, + { + "id": "FG001", + "title": "Cohort 2", + "description": "2.0 X 10\\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12)\n\nAVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-\u03b2-actin promoter" + } + ], + "periods": [ + { + "title": "Overall Study", + "milestones": [ + { + "type": "STARTED", + "achievements": [ + { "groupId": "FG000", "numSubjects": "3" }, + { "groupId": "FG001", "numSubjects": "12" } + ] + }, + { + "type": "COMPLETED", + "achievements": [ + { "groupId": "FG000", "numSubjects": "3" }, + { "groupId": "FG001", "numSubjects": "12" } + ] + }, + { + "type": "NOT COMPLETED", + "achievements": [ + { "groupId": "FG000", "numSubjects": "0" }, + { "groupId": "FG001", "numSubjects": "0" } + ] + } + ] + } + ] + }, + "baselineCharacteristicsModule": { + "groups": [ + { + "id": "BG000", + "title": "Cohort 1", + "description": "6.7 X 10\\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3)\n\nAVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-\u03b2-actin promoter" + }, + { + "id": "BG001", + "title": "Cohort 2", + "description": "2.0 X 10\\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12)\n\nAVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-\u03b2-actin promoter" + }, + { + "id": "BG002", + "title": "Total", + "description": "Total of all reporting groups" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "BG000", "value": "3" }, + { "groupId": "BG001", "value": "12" }, + { "groupId": "BG002", "value": "15" } + ] + } + ], + "measures": [ + { + "title": "Age, Categorical", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "title": "<=18 years", + "measurements": [ + { "groupId": "BG000", "value": "3" }, + { "groupId": "BG001", "value": "12" }, + { "groupId": "BG002", "value": "15" } + ] + }, + { + "title": "Between 18 and 65 years", + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" } + ] + }, + { + "title": ">=65 years", + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" } + ] + } + ] + } + ] + }, + { + "title": "Age, Continuous", + "paramType": "MEAN", + "dispersionType": "STANDARD_DEVIATION", + "unitOfMeasure": "months", + "classes": [ + { + "categories": [ + { + "measurements": [ + { + "groupId": "BG000", + "value": "6.33", + "spread": "0.751" + }, + { + "groupId": "BG001", + "value": "3.42", + "spread": "2.063" + }, + { + "groupId": "BG002", + "value": "4.00", + "spread": "2.209" + } + ] + } + ] + } + ] + }, + { + "title": "Sex: Female, Male", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "title": "Female", + "measurements": [ + { "groupId": "BG000", "value": "2" }, + { "groupId": "BG001", "value": "7" }, + { "groupId": "BG002", "value": "9" } + ] + }, + { + "title": "Male", + "measurements": [ + { "groupId": "BG000", "value": "1" }, + { "groupId": "BG001", "value": "5" }, + { "groupId": "BG002", "value": "6" } + ] + } + ] + } + ] + }, + { + "title": "Race/Ethnicity, Customized", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "title": "Not Hispanic or Latino", + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "3" }, + { "groupId": "BG001", "value": "10" }, + { "groupId": "BG002", "value": "13" } + ] + } + ] + }, + { + "title": "Hispanic or Latino", + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "2" }, + { "groupId": "BG002", "value": "2" } + ] + } + ] + } + ] + }, + { + "title": "Region of Enrollment", + "paramType": "NUMBER", + "unitOfMeasure": "participants", + "classes": [ + { + "title": "United States", + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "3" }, + { "groupId": "BG001", "value": "12" }, + { "groupId": "BG002", "value": "15" } + ] + } + ] + } + ] + }, + { + "title": "SMN2 copy number = 2", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "3" }, + { "groupId": "BG001", "value": "12" }, + { "groupId": "BG002", "value": "15" } + ] + } + ] + } + ] + }, + { + "title": "bi-allelic deletions of SMN1", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "3" }, + { "groupId": "BG001", "value": "12" }, + { "groupId": "BG002", "value": "15" } + ] + } + ] + } + ] + }, + { + "title": "Exon 7 gene modifier mutation", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "0" }, + { "groupId": "BG001", "value": "0" }, + { "groupId": "BG002", "value": "0" } + ] + } + ] + } + ] + } + ] + }, + "outcomeMeasuresModule": { + "outcomeMeasures": [ + { + "type": "PRIMARY", + "title": "Number of Participants That Experienced One Grade III or Higher Unanticipated, Treatment-related Toxicity That Presents With Clinical Symptoms and Requires Medical Treatment", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "2 years", + "groups": [ + { + "id": "OG000", + "title": "Cohort 1", + "description": "6.7 X 10\\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3)\n\nAVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-\u03b2-actin promoter" + }, + { + "id": "OG001", + "title": "Cohort 2", + "description": "2.0 X 10\\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12)\n\nAVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-\u03b2-actin promoter" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "3" }, + { "groupId": "OG001", "value": "12" } + ] + } + ], + "classes": [ + { + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "1" }, + { "groupId": "OG001", "value": "3" } + ] + } + ] + } + ] + }, + { + "type": "SECONDARY", + "title": "Number of Participants Who Experienced Permanent Ventilation or Death", + "description": "Permanent ventilation was defined as the requirement of \u2265 16-hour respiratory assistance, including non-invasive ventilatory support, per day continuously for \u2265 2 weeks in the absence of an acute reversible illness, excluding perioperative ventilation.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "Up to 13.6 months of age", + "groups": [ + { + "id": "OG000", + "title": "Cohort 1", + "description": "6.7 X 10\\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3)\n\nAVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-\u03b2-actin promoter" + }, + { + "id": "OG001", + "title": "Cohort 2", + "description": "2.0 X 10\\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12)\n\nAVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-\u03b2-actin promoter" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "3" }, + { "groupId": "OG001", "value": "12" } + ] + } + ], + "classes": [ + { + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "0" } + ] + } + ] + } + ] + }, + { + "type": "SECONDARY", + "title": "Percent Change From Baseline in Mean Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score", + "description": "Score ranges from 0 to 64, where 64 is the maximum possible score. A higher score is indicative of higher/better motor function. CHOP-INTEND assessments were discontinued once patients achieved higher functioning status, so the number of available data points decreased over time.", + "populationDescription": "Includes all treated participants with non-missing data. For a major, systemic, and externally imposed limitation of movement that prevented accurate assessment of multiple items, the total score was regarded as missing. Also, CHOP-INTEND assessments were discontinued once participants achieved higher functioning status (2 consecutive scores \u226562).", + "reportingStatus": "POSTED", + "paramType": "MEAN", + "dispersionType": "Standard Deviation", + "unitOfMeasure": "Percentage Change", + "timeFrame": "Baseline to 24 months post-dose", + "groups": [ + { + "id": "OG000", + "title": "Cohort 1", + "description": "6.7 X 10\\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3)\n\nAVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-\u03b2-actin promoter" + }, + { + "id": "OG001", + "title": "Cohort 2", + "description": "2.0 X 10\\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12)\n\nAVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-\u03b2-actin promoter" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "6" } + ] + } + ], + "classes": [ + { + "categories": [ + { + "measurements": [ + { + "groupId": "OG001", + "value": "30.7", + "spread": "145.61" + } + ] + } + ] + } + ] + }, + { + "type": "SECONDARY", + "title": "Number of Participants With Assessed Improvement in Motor Function", + "description": "Improvement in motor function was determined by achievement of developmental milestones, specifically achievement of ability to sit unassisted for at least 30 seconds, determined by physical therapist and confirmed by an independent central video reviewer. Achievement of functional independent sitting was defined as the ability to maintain a sitting position independently for at least 30 seconds as confirmed per video evaluation by an expert central reviewer based on videos taken either at scheduled visits or provided by the parent/legal guardian.", + "populationDescription": "full analysis set- all treated patients", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "24 months post-dose", + "groups": [ + { + "id": "OG000", + "title": "Cohort 1", + "description": "6.7 X 10\\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3)\n\nAVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-\u03b2-actin promoter" + }, + { + "id": "OG001", + "title": "Cohort 2", + "description": "2.0 X 10\\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12)\n\nAVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-\u03b2-actin promoter" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [ + { "groupId": "OG000", "value": "3" }, + { "groupId": "OG001", "value": "12" } + ] + } + ], + "classes": [ + { + "categories": [ + { + "measurements": [ + { "groupId": "OG000", "value": "0" }, + { "groupId": "OG001", "value": "9" } + ] + } + ] + } + ], + "analyses": [ + { + "groupIds": ["OG000", "OG001"], + "groupDescription": "Data for the current study were compared to historical control data (Pediatric Neuromuscular Clinical Research \\[PNCR\\], Finkel et al 2014 - PubMed 25080519) where 0 participants were able to sit independently.", + "nonInferiorityType": "SUPERIORITY", + "pValue": "<0.001", + "statisticalMethod": "One-sided Exact Binomial Test" + } + ] + } + ] + }, + "adverseEventsModule": { + "frequencyThreshold": "0", + "timeFrame": "Adverse events were collected from the single dose of study treatment until 24 months post dose", + "eventGroups": [ + { + "id": "EG000", + "title": "Cohort 1", + "description": "6.7 X 10\\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3)\n\nAVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-\u03b2-actin promoter", + "deathsNumAffected": 0, + "deathsNumAtRisk": 3, + "seriousNumAffected": 3, + "seriousNumAtRisk": 3, + "otherNumAffected": 3, + "otherNumAtRisk": 3 + }, + { + "id": "EG001", + "title": "Cohort 2", + "description": "2.0 X 10\\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12)\n\nAVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-\u03b2-actin promoter", + "deathsNumAffected": 0, + "deathsNumAtRisk": 12, + "seriousNumAffected": 10, + "seriousNumAtRisk": 12, + "otherNumAffected": 12, + "otherNumAtRisk": 12 + } + ], + "seriousEvents": [ + { + "term": "pneumonia", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 10, + "numAffected": 7, + "numAtRisk": 12 + } + ] + }, + { + "term": "rhinovirus infection", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 5, + "numAffected": 2, + "numAtRisk": 12 + } + ] + }, + { + "term": "enterovirus infection", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 3, + "numAffected": 2, + "numAtRisk": 12 + } + ] + }, + { + "term": "parainfluenzae virus infection", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 2, + "numAffected": 2, + "numAtRisk": 12 + } + ] + }, + { + "term": "respiratory syncytial virus bronchiolitis", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 2, + "numAffected": 2, + "numAtRisk": 12 + } + ] + }, + { + "term": "upper respiratory tract infection", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 3, + "numAffected": 3, + "numAtRisk": 12 + } + ] + }, + { + "term": "adenovirus infection", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 2, + "numAffected": 2, + "numAtRisk": 12 + } + ] + }, + { + "term": "human rhinovirus test positive", + "organSystem": "Investigations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 2, + "numAffected": 2, + "numAtRisk": 12 + } + ] + }, + { + "term": "lower respiratory tract infection", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 2, + "numAffected": 1, + "numAtRisk": 12 + } + ] + }, + { + "term": "penumonia aspiration", + "organSystem": "Respiratory, thoracic and mediastinal disorders", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 2, + "numAffected": 2, + "numAtRisk": 12 + } + ] + }, + { + "term": "postoperative wound infection", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 2, + "numAffected": 1, + "numAtRisk": 12 + } + ] + }, + { + "term": "respiratory distress", + "organSystem": "Respiratory, thoracic and mediastinal disorders", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 2, + "numAffected": 2, + "numAtRisk": 12 + } + ] + }, + { + "term": "transaminases increased", + "organSystem": "Investigations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 12 + } + ] + }, + { + "term": "atelectasis", + "organSystem": "Respiratory, thoracic and mediastinal disorders", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 12 + } + ] + }, + { + "term": "bronchitis", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 12 + } + ] + }, + { + "term": "dehydration", + "organSystem": "Metabolism and nutrition disorders", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 12 + } + ] + }, + { + "term": "enterovirus test positive", + "organSystem": "Investigations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 12 + } + ] + }, + { + "term": "femur fracture", + "organSystem": "Injury, poisoning and procedural complications", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 12 + } + ] + }, + { + "term": "gastroenteritis", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 12 + } + ] + }, + { + "term": "gastroenteritis viral", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 12 + } + ] + }, + { + "term": "influenza", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 12 + } + ] + }, + { + "term": "norovirus test positive", + "organSystem": "Investigations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 12 + } + ] + }, + { + "term": "oxygen saturation decreased", + "organSystem": "Investigations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 12 + } + ] + }, + { + "term": "pneumonia parainfluenzae viral", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 12 + } + ] + }, + { + "term": "pneumonia viral", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 12 + } + ] + }, + { + "term": "post procedural hemorrhage", + "organSystem": "Injury, poisoning and procedural complications", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 12 + } + ] + }, + { + "term": "respiratory failure", + "organSystem": "Respiratory, thoracic and mediastinal disorders", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 12 + } + ] + }, + { + "term": "tachycardia", + "organSystem": "Cardiac disorders", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 12 + } + ] + }, + { + "term": "viral upper respiratory tract infection", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 12 + } + ] + }, + { + "term": "pneumonia respiratory syncytial viral", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 2, + "numAffected": 2, + "numAtRisk": 12 + } + ] + } + ], + "otherEvents": [ + { + "term": "constipation", + "organSystem": "Gastrointestinal disorders", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 8, + "numAffected": 6, + "numAtRisk": 12 + } + ] + }, + { + "term": "diarrhea", + "organSystem": "Gastrointestinal disorders", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 3, + "numAffected": 3, + "numAtRisk": 12 + } + ] + }, + { + "term": "gastroesophageal reflux disease", + "organSystem": "Gastrointestinal disorders", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 6, + "numAffected": 5, + "numAtRisk": 12 + } + ] + }, + { + "term": "vomiting", + "organSystem": "Gastrointestinal disorders", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 0, + "numAffected": 0, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 11, + "numAffected": 8, + "numAtRisk": 12 + } + ] + }, + { + "term": "pyrexia", + "organSystem": "General disorders", + "sourceVocabulary": "MedDRA 20.0", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 3 + }, + { + "groupId": "EG001", + "numEvents": 12, + "numAffected": 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"browseLeaves": [ + { "id": "M17522", "name": "Virus Diseases", "relevance": "LOW" }, + { + "id": "M4589", + "name": "Atrophy", + "asFound": "Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12090", + "name": "Muscular Atrophy", + "asFound": "Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12091", + "name": "Muscular Atrophy, Spinal", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M17637", + "name": "Spinal Muscular Atrophies of Childhood", + "asFound": "Spinal Muscular Atrophy 1", + "relevance": "HIGH" + }, + { + "id": "M22519", + "name": "Pathological Conditions, Anatomical", + "relevance": "LOW" + }, + { + "id": "M22619", + "name": "Neuromuscular Manifestations", + "relevance": "LOW" + }, + { + "id": "M12404", + "name": "Neurologic Manifestations", + "relevance": "LOW" + }, + { + "id": "M15915", + "name": "Spinal Cord Diseases", + "relevance": "LOW" + }, + { + "id": "M5742", + "name": "Central Nervous System Diseases", + "relevance": "LOW" + }, + { + "id": "M18879", + "name": "Motor Neuron Disease", + "relevance": "LOW" + }, + { + "id": "M4024", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + }, + { + "id": "M21558", + "name": "Neurodegenerative Diseases", + "relevance": "LOW" + }, + { + "id": "M12411", + "name": "Neuromuscular Diseases", + "relevance": "LOW" + }, + { + "id": "M22092", + "name": "Heredodegenerative Disorders, Nervous System", + "relevance": "LOW" + }, + { + "id": "M23686", + "name": "Genetic Diseases, Inborn", + "relevance": "LOW" + }, + { + "id": "T5342", + "name": "Spinal Muscular Atrophy", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "T5343", + "name": "Spinal Muscular Atrophy 1", + "asFound": "Spinal Muscular Atrophy 1", + "relevance": "HIGH" + }, + { + "id": "T349", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + } + ], + "browseBranches": [ + { "abbrev": "BC01", "name": "Infections" }, + { "abbrev": "All", "name": "All Conditions" }, + { "abbrev": "BC23", "name": "Symptoms and General Pathology" }, + { "abbrev": "BC10", "name": "Nervous System Diseases" }, + { + "abbrev": "BC16", + "name": "Diseases and Abnormalities at or Before Birth" + }, + { "abbrev": "BC18", "name": "Nutritional and Metabolic Diseases" }, + { "abbrev": "Rare", "name": "Rare Diseases" } + ] + } + }, + "hasResults": true + }, + { + "protocolSection": { + "identificationModule": { + "nctId": "NCT05073133", + "orgStudyIdInfo": { "id": "COAV101A1IC01" }, + "secondaryIdInfos": [ + { "id": "2023-000864-67", "type": "EUDRACT_NUMBER" } + ], + "organization": { "fullName": "Novartis", "class": "INDUSTRY" }, + "briefTitle": "Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)", + "officialTitle": "A Phase IV Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the saFEty, toLerability and effIcacy of Gene Replacement Therapy With intravenousOAV101(AVXS101) in Pediatric Patients From Latin America With Spinal Muscular Atrophy (SMA) - OFELIA", + "acronym": "OFELIA" + }, + "statusModule": { + "statusVerifiedDate": "2024-10", + "overallStatus": "COMPLETED", + "expandedAccessInfo": { "hasExpandedAccess": false }, + "startDateStruct": { "date": "2021-11-04", "type": "ACTUAL" }, + "primaryCompletionDateStruct": { + "date": "2023-08-08", + "type": "ACTUAL" + }, + "completionDateStruct": { "date": "2023-08-08", "type": "ACTUAL" }, + "studyFirstSubmitDate": "2021-09-20", + "studyFirstSubmitQcDate": "2021-09-28", + "studyFirstPostDateStruct": { + "date": "2021-10-11", + "type": "ACTUAL" + }, + "resultsFirstSubmitDate": "2023-12-15", + "resultsFirstSubmitQcDate": "2023-12-15", + "resultsFirstPostDateStruct": { + "date": "2024-05-31", + "type": "ACTUAL" + }, + "lastUpdateSubmitDate": "2024-10-07", + "lastUpdatePostDateStruct": { "date": "2024-10-09", "type": "ACTUAL" } + }, + "sponsorCollaboratorsModule": { + "responsibleParty": { "type": "SPONSOR" }, + "leadSponsor": { + "name": "Novartis Pharmaceuticals", + "class": "INDUSTRY" + } + }, + "oversightModule": { + "oversightHasDmc": true, + "isFdaRegulatedDrug": false, + "isFdaRegulatedDevice": false + }, + "descriptionModule": { + "briefSummary": "This was a phase IV Open-label, single-arm, single-dose, multicenter study, to evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene \u2264 24 months and weighing \u2264 17 kg, over a 18-month period post infusion.", + "detailedDescription": "This is an open-label, single arm, multi-center study to evaluate the safety, tolerability, and efficacy of IV OAV101 in symptomatic SMA pediatric participants. The study enrolled participants \u2264 24 months old that weigh \u2264 17 kg. Participants who met eligibility criteria at Screening and Baseline visits received a single dose of IV OAV101 t the approved dose of 1.1e14 vg/kg and were followed for 18 months. The study included a 20-day screening period in which there were 2 Screening visits, during which, eligibility was assessed (Screening 1), weight was collected for dose calculation (Screening 2), and baseline assessments were performed prior to treatment.\n\nOn Day -1, participants were admitted to the hospital for pre-treatment baseline procedures including prednisolone treatment per study protocol. On Day 1, participants received a single IV infusion of OAV101. Participants were discharged 12-48 hours after the infusion, based on Investigator judgment. Safety monitoring was performed on an ongoing basis per protocol requirement and was evaluated by the clinical safety team as well as DMC (Data monitoring committee)." + }, + "conditionsModule": { + "conditions": ["Muscular Atrophy, Spinal"], + "keywords": [ + "Zolgensma", + "OAV101", + "AVXS 101", + "gene therapy", + "Muscle atrophy", + "SBMA", + "spinal and bulbar muscular atrophy", + "spinal muscular atrophy", + "bulbar muscular atrophy", + "muscle function", + "myopathy", + "muscle wasting", + "atrophied muscle", + "loss of muscle strength" + ] + }, + "designModule": { + "studyType": "INTERVENTIONAL", + "phases": ["PHASE4"], + "designInfo": { + "allocation": "NA", + "interventionModel": "SINGLE_GROUP", + "interventionModelDescription": "OAV101 will be administered as a single IV infusion at 1.1e14 vg/kg over approximately 60 minutes", + "primaryPurpose": "TREATMENT", + "maskingInfo": { "masking": "NONE" } + }, + "enrollmentInfo": { "count": 16, "type": "ACTUAL" } + }, + "armsInterventionsModule": { + "armGroups": [ + { + "label": "OAV101", + "type": "EXPERIMENTAL", + "description": "A single IV infusion at 1.1e14 vector genome (vg)/kg over approximately 60 minutes", + "interventionNames": ["Genetic: OAV101"] + } + ], + "interventions": [ + { + "type": "GENETIC", + "name": "OAV101", + "description": "A single Gene Therapy IV infusion at 1.1e14 vector genome (vg)/kg over approximately 60 minutes", + "armGroupLabels": ["OAV101"], + "otherNames": ["AVXS-101, Zolgensma"] + } + ] + }, + "outcomesModule": { + "primaryOutcomes": [ + { + "measure": "Number of Participants With Treatment Emergent AEs and SAEs", + "description": "An AE is any untoward medical occurrence (eg any unfavorable and unintended sign \\[including abnormal laboratory findings\\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study. Therefore, an AE may or may not be temporally or causally associated with the use of a medicinal (investigational) product.\n\nChanges from baseline in vital signs, cardiac safety assessments, and clinical laboratory results are reported as Adverse Events if clinically significant and as applicable, per investigator assessment.", + "timeFrame": "Up to Month 18" + }, + { + "measure": "Evaluation of Important Identified and Important Potential Risks - Treatment-emergent Adverse Events of Special Interest", + "description": "An AE is any untoward medical occurrence (eg any unfavorable and unintended sign \\[including abnormal laboratory findings\\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study. Therefore, an AE may or may not be temporally or causally associated with the use of a medicinal (investigational) product.\n\nAdverse events of special interest (AESI) are defined by the important identified risk and important potential risk: Hepatotoxicity, Thrombocytopenia, Cardiac adverse events, Sensory abnormalities suggestive of ganglionopathy, and Thrombotic microangiopathy. These were assessed by the investigator.", + "timeFrame": "Up to Month 18" + } + ], + "secondaryOutcomes": [ + { + "measure": "Number of Participants Who Achieve Development Motor Milestones According to the World Health Organization-Multicentre Growth Reference Study (WHO-MGRS)", + "description": "The World Health Organization-Multicentre Growth Reference Study (WHO-MGRS) was used to measure developmental motor milestones. This was assessed via the milestone checklist. The 6 developmental milestones are: sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone. A yes response indicates that the patient reached a particular development milestone.", + "timeFrame": "Baseline (Screening), and at Weeks 26, 52 and 78" + } + ] + }, + "eligibilityModule": { + "eligibilityCriteria": "Inclusion Criteria:\n\n1. Written informed consent/assent obtained prior to any assessment performed\n2. Symptomatic SMA diagnosis based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and any copy of SMN2 gene.\n3. Age \u2264 24 months of age at time of treatment\n\n3. Weight \u226417 kg at the time of Screening Period 4. Na\u00efve to treatment or have discontinued an approved drug/therapy 5. Up-to date on recommended childhood vaccinations and RSV prophylaxis with palivizumab (also known as Synagis), per local standard of care\n\nKey Exclusion Criteria:\n\n1. Previous use of OAV101 or any AAV9 gene therapy\n2. Participant with history of aspiration pneumonia or signs of aspiration (eg, coughing or sputtering of food) within 4 weeks prior to Screening\n3. Participant dependent on gastrostomy feeding tube for 100% of nutritional intake.\n4. Anti-AAV9 antibody titer \\> 1:50 as determined by ligand binding immunoassay at the time of screening\n5. Inability to take corticosteroids\n6. Concomitant use of immunosuppressive therapy, plasmapheresis, immunomodulators such as adalimumab, or immunosuppressive therapy within 3 months prior to gene replacement therapy (eg, cyclosporine, tacrolimus, methotrexate, rituximab cyclophosphamide, IV immunoglobulin)\n7. Hepatic dysfunction (i.e. AST, ALT, bilirubin, GGT or GLDH, \u2265 ULN; CTCAE \u2265 1) at Screening (with the exception of isolated AST elevation: in the absence of other liver laboratory abnormalities, isolated AST elevation is not considered exclusionary)\n8. Previously treated with nusinersen (Spinraza\u00ae) within 4 months prior to Screening\n9. Previously treated with risdiplam (EvrysdiTM) within 15 days prior to Screening (washout period of at least 5 half-lives before Screening)", + "healthyVolunteers": false, + "sex": "ALL", + "maximumAge": "24 Months", + "stdAges": ["CHILD"] + }, + "contactsLocationsModule": { + "locations": [ + { + "facility": "Novartis Investigative Site", + "city": "Caba", + "state": "Buenos Aires", + "zip": "C1181ACH", + "country": "Argentina", + "geoPoint": { "lat": -34.61315, "lon": -58.37723 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Buenos Aires", + "zip": "C1245AAM", + "country": "Argentina", + "geoPoint": { "lat": -34.61315, "lon": -58.37723 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Belo Horizonte", + "state": "MG", + "zip": "30130-000", + "country": "Brazil", + "geoPoint": { "lat": -19.92083, "lon": -43.93778 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Porto Alegre", + "state": "RS", + "zip": "90035-903", + "country": "Brazil", + "geoPoint": { "lat": -30.03306, "lon": -51.23 } + }, + { + "facility": "Novartis Investigative Site", + "city": "Sao Paulo", + "state": "SP", + "zip": "05403-000", + "country": "Brazil", + "geoPoint": { "lat": -23.5475, "lon": -46.63611 } + } + ] + }, + "referencesModule": { + "seeAlsoLinks": [ + { + "label": "A Plain Language Trial Summary is available on www.novctrd.com", + "url": "https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=1876" + } + ] + }, + "ipdSharingStatementModule": { + "ipdSharing": "YES", + "description": "Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on https://www.clinicalstudydatarequest.com/.", + "url": "https://www.clinicalstudydatarequest.com/" + } + }, + "resultsSection": { + "participantFlowModule": { + "preAssignmentDetails": "On Day -1, participants were admitted to the hospital for pre-treatment baseline procedures including prednisolone treatment per study protocol.", + "recruitmentDetails": "16 participants were enrolled into the study, at five sites from Brazil (three sites) and Argentina (two sites). Six participants were from Argentina and 10 from Brazil.", + "groups": [ + { + "id": "FG000", + "title": "OAV101", + "description": "A single IV infusion at 1.1e14 vg/kg over approximately 60 minutes" + } + ], + "periods": [ + { + "title": "Overall Study", + "milestones": [ + { + "type": "STARTED", + "achievements": [{ "groupId": "FG000", "numSubjects": "16" }] + }, + { + "type": "COMPLETED", + "achievements": [{ "groupId": "FG000", "numSubjects": "14" }] + }, + { + "type": "NOT COMPLETED", + "achievements": [{ "groupId": "FG000", "numSubjects": "2" }] + } + ], + "dropWithdraws": [ + { + "type": "Death", + "reasons": [{ "groupId": "FG000", "numSubjects": "2" }] + } + ] + } + ] + }, + "baselineCharacteristicsModule": { + "groups": [ + { + "id": "BG000", + "title": "OAV101", + "description": "A single IV infusion at 1.1e14 vg/kg over approximately 60 minutes" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "BG000", "value": "16" }] + } + ], + "measures": [ + { + "title": "Age, Continuous", + "paramType": "MEAN", + "dispersionType": "STANDARD_DEVIATION", + "unitOfMeasure": "months", + "classes": [ + { + "title": "Age at dosing", + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "BG000", "value": "16" }] + } + ], + "categories": [ + { + "measurements": [ + { + "groupId": "BG000", + "value": "15.79", + "spread": "5.89" + } + ] + } + ] + } + ] + }, + { + "title": "Sex: Female, Male", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "BG000", "value": "16" }] + } + ], + "categories": [ + { + "title": "Female", + "measurements": [{ "groupId": "BG000", "value": "11" }] + }, + { + "title": "Male", + "measurements": [{ "groupId": "BG000", "value": "5" }] + } + ] + } + ] + }, + { + "title": "Race and Ethnicity Not Collected", + "populationDescription": "Race and Ethnicity were not collected from any participant.", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "BG000", "value": "0" }] + } + ] + } + ] + } + ] + }, + "outcomeMeasuresModule": { + "outcomeMeasures": [ + { + "type": "PRIMARY", + "title": "Number of Participants With Treatment Emergent AEs and SAEs", + "description": "An AE is any untoward medical occurrence (eg any unfavorable and unintended sign \\[including abnormal laboratory findings\\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study. Therefore, an AE may or may not be temporally or causally associated with the use of a medicinal (investigational) product.\n\nChanges from baseline in vital signs, cardiac safety assessments, and clinical laboratory results are reported as Adverse Events if clinically significant and as applicable, per investigator assessment.", + "populationDescription": "Full analysis set (FAS) - all treated patients.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "Up to Month 18", + "groups": [ + { + "id": "OG000", + "title": "OAV101", + "description": "A single IV infusion at 1.1e14 vg/kg over approximately 60 minutes" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "16" }] + } + ], + "classes": [ + { + "title": "Any treatment-emergent adverse events", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "16" }] } + ] + }, + { + "title": "Any treatment-emergent adverse events related to OAV101", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "11" }] } + ] + }, + { + "title": "Any serious treatment-emergent adverse events", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "11" }] } + ] + }, + { + "title": "Serious treatment-emergent adverse events related to OAV101", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "3" }] } + ] + }, + { + "title": "Treatment-emergent adverse events leading to study discontinuation", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "0" }] } + ] + }, + { + "title": "Treatment-emergent adverse events leading to death", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "2" }] } + ] + }, + { + "title": "Treatment-emergent adverse events of special interest", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "12" }] } + ] + } + ] + }, + { + "type": "PRIMARY", + "title": "Evaluation of Important Identified and Important Potential Risks - Treatment-emergent Adverse Events of Special Interest", + "description": "An AE is any untoward medical occurrence (eg any unfavorable and unintended sign \\[including abnormal laboratory findings\\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study. Therefore, an AE may or may not be temporally or causally associated with the use of a medicinal (investigational) product.\n\nAdverse events of special interest (AESI) are defined by the important identified risk and important potential risk: Hepatotoxicity, Thrombocytopenia, Cardiac adverse events, Sensory abnormalities suggestive of ganglionopathy, and Thrombotic microangiopathy. These were assessed by the investigator.", + "populationDescription": "Full analysis set (FAS) - all treated patients.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "Up to Month 18", + "groups": [ + { + "id": "OG000", + "title": "OAV101", + "description": "A single IV infusion at 1.1e14 vg/kg over approximately 60 minutes" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "16" }] + } + ], + "classes": [ + { + "title": "Risk name: Hepatotoxicity", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "11" }] } + ] + }, + { + "title": "-Preferred term: Aspartate aminotransferase increased", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "5" }] } + ] + }, + { + "title": "-Preferred term: Alanine aminotransferase increased", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "5" }] } + ] + }, + { + "title": "-Preferred term: Blood alkaline phosphatase increased", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "2" }] } + ] + }, + { + "title": "-Preferred term: Bilirubin conjugated increased", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "2" }] } + ] + }, + { + "title": "-Preferred term: Gamma-glutamyltransferase increased", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "5" }] } + ] + }, + { + "title": "-Preferred term: Hepatic enzyme increased", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "3" }] } + ] + }, + { + "title": "-Preferred term: Hepatic failure", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "1" }] } + ] + }, + { + "title": "-Preferred term: Transaminases increased", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "2" }] } + ] + }, + { + "title": "Risk name: Thrombocytopenia", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "5" }] } + ] + }, + { + "title": "-Preferred term: Platelet count decreased", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "4" }] } + ] + }, + { + "title": "-Preferred term: Thrombocytopenia", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "1" }] } + ] + }, + { + "title": "Risk name: Thrombotic microangiopathy", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "2" }] } + ] + }, + { + "title": "-Preferred term: Thrombotic microangiopathy", + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "2" }] } + ] + } + ] + }, + { + "type": "SECONDARY", + "title": "Number of Participants Who Achieve Development Motor Milestones According to the World Health Organization-Multicentre Growth Reference Study (WHO-MGRS)", + "description": "The World Health Organization-Multicentre Growth Reference Study (WHO-MGRS) was used to measure developmental motor milestones. This was assessed via the milestone checklist. The 6 developmental milestones are: sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone. A yes response indicates that the patient reached a particular development milestone.", + "populationDescription": "Full analysis set (FAS) - all treated patients with evaluable assessments at the respective assessment time points.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "Baseline (Screening), and at Weeks 26, 52 and 78", + "groups": [ + { + "id": "OG000", + "title": "OAV101", + "description": "A single IV infusion at 1.1e14 vg/kg over approximately 60 minutes" + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "16" }] + } + ], + "classes": [ + { + "title": "Screening - Sitting without support", + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "16" }] + } + ], + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "6" }] } + ] + }, + { + "title": "Screening - Hands-and-knees crawling", + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "16" }] + } + ], + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "2" }] } + ] + }, + { + "title": "Screening - Standing with assistance", + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "16" }] + } + ], + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "1" }] } + ] + }, + { + "title": "Screening - Walking with assistance", + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "16" }] + } + ], + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "0" }] } + ] + }, + { + "title": "Screening - Standing alone", + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "16" }] + } + ], + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "0" }] } + ] + }, + { + "title": "Screening - Walking alone", + "denoms": [ + { + "units": "Participants", 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Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial." + }, + "pointOfContact": { + "title": "Study Director", + "organization": "Novartis Pharmaceuticals", + "email": "Novartis.email@Novartis.com", + "phone": "+ 1 862 778 8300" + } + } + }, + "documentSection": { + "largeDocumentModule": { + "largeDocs": [ + { + "typeAbbrev": "Prot", + "hasProtocol": true, + "hasSap": false, + "hasIcf": false, + "label": "Study Protocol", + "date": "2022-03-24", + "uploadDate": "2023-12-15T11:35", + "filename": "Prot_000.pdf", + "size": 903613 + }, + { + "typeAbbrev": "SAP", + "hasProtocol": false, + "hasSap": true, + "hasIcf": false, + "label": "Statistical Analysis Plan", + "date": "2023-07-20", + "uploadDate": "2023-12-15T11:35", + "filename": "SAP_001.pdf", + "size": 620723 + } + ] + } + }, + "derivedSection": { + "miscInfoModule": { "versionHolder": "2024-12-04" }, + "conditionBrowseModule": { + "meshes": [ + 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"Muscular Atrophy", + "asFound": "Muscular Atrophy", + "relevance": "HIGH" + }, + { "id": "M12092", "name": "Muscular Diseases", "relevance": "LOW" }, + { + "id": "M12091", + "name": "Muscular Atrophy, Spinal", + "asFound": "Muscular Atrophy, Spinal", + "relevance": "HIGH" + }, + { + "id": "M28216", + "name": "Bulbo-Spinal Atrophy, X-Linked", + "relevance": "LOW" + }, + { + "id": "M22519", + "name": "Pathological Conditions, Anatomical", + "relevance": "LOW" + }, + { + "id": "M22619", + "name": "Neuromuscular Manifestations", + "relevance": "LOW" + }, + { + "id": "M12404", + "name": "Neurologic Manifestations", + "relevance": "LOW" + }, + { + "id": "M15915", + "name": "Spinal Cord Diseases", + "relevance": "LOW" + }, + { + "id": "M5742", + "name": "Central Nervous System Diseases", + "relevance": "LOW" + }, + { + "id": "M18879", + "name": "Motor Neuron Disease", + "relevance": "LOW" + }, + { + "id": "M4024", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + }, + { + "id": "M21558", + "name": "Neurodegenerative Diseases", + "relevance": "LOW" + }, + { + "id": "M12411", + "name": "Neuromuscular Diseases", + "relevance": "LOW" + }, + { "id": "T3215", "name": "Kennedy Disease", "relevance": "LOW" }, + { + "id": "T5342", + "name": "Spinal Muscular Atrophy", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "T349", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + } + ], + "browseBranches": [ + { "abbrev": "BC18", "name": "Nutritional and Metabolic Diseases" }, + { "abbrev": "All", "name": "All Conditions" }, + { "abbrev": "BC23", "name": "Symptoms and General Pathology" }, + { "abbrev": "BC10", "name": "Nervous System Diseases" }, + { "abbrev": "BC05", "name": "Musculoskeletal Diseases" }, + { + "abbrev": "BC16", + "name": "Diseases and Abnormalities at or Before Birth" + }, + { "abbrev": "Rare", "name": "Rare Diseases" } + ] + } + }, + "hasResults": true + }, + { + "protocolSection": { + "identificationModule": { + "nctId": "NCT05575011", + "orgStudyIdInfo": { "id": "277HV101" }, + "secondaryIdInfos": [ + { "id": "2022-000956-12", "type": "EUDRACT_NUMBER" } + ], + "organization": { "fullName": "Biogen", "class": "INDUSTRY" }, + "briefTitle": "A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec", + "officialTitle": "A Randomized, Blinded, Placebo-Controlled, Phase 1 Single Ascending Dose Study in Healthy Adult Male Volunteers and an Open-Label Multiple Ascending Dose Study in Pediatric SMA Participants Previously Treated With Onasemnogene Abeparvovec (Zolgensma\u2122) to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115" + }, + "statusModule": { + "statusVerifiedDate": "2024-07", + "overallStatus": "RECRUITING", + "expandedAccessInfo": { "hasExpandedAccess": false }, + "startDateStruct": { "date": "2022-10-10", "type": "ACTUAL" }, + "primaryCompletionDateStruct": { + "date": "2027-09-01", + "type": "ESTIMATED" + }, + "completionDateStruct": { "date": "2027-09-01", "type": "ESTIMATED" }, + "studyFirstSubmitDate": "2022-10-07", + "studyFirstSubmitQcDate": "2022-10-07", + "studyFirstPostDateStruct": { + "date": "2022-10-12", + "type": "ACTUAL" + }, + "lastUpdateSubmitDate": "2024-07-17", + "lastUpdatePostDateStruct": { "date": "2024-07-19", "type": "ACTUAL" } + }, + "sponsorCollaboratorsModule": { + "responsibleParty": { "type": "SPONSOR" }, + "leadSponsor": { "name": "Biogen", "class": "INDUSTRY" } + }, + "oversightModule": { + "oversightHasDmc": true, + "isFdaRegulatedDrug": true, + "isFdaRegulatedDevice": false + }, + "descriptionModule": { + "briefSummary": "In this study, researchers will learn about a study drug called BIIB115 in healthy male volunteers and in participants with spinal muscular atrophy (SMA). This study will focus on children with SMA.\n\nThe main objective of the study is to learn more about the safety of BIIB115 and how participants respond to different doses of BIIB115. The main question researchers want to answer is:\n\nHow many participants have adverse events and serious adverse events during the study? Adverse events are unwanted health problems that may or may not be caused by the study drug.\n\nResearchers will also learn more about how the body processes BIIB115. They will do this by measuring the levels of BIIB115 in both the blood and the cerebrospinal fluid, also known as the CSF. This is the fluid around the brain and spinal cord.\n\nThe study will be split into 2 parts - Part A and Part B.\n\nDuring Part A:\n\n* After screening, healthy volunteers will be randomly placed into 1 of 4 groups to receive either BIIB115 or a placebo. A placebo looks like the study drug but contains no real medicine.\n* Participants will receive a single dose of either BIIB115 or the placebo as an injection directly into the spinal canal on Day 1.\n* Neither the researchers nor the participants will know if the participants will receive BIIB115 or the placebo.\n* The treatment and follow up period will last for 13 months.\n* Participants will have up to 6 clinic visits and 4 telephone calls.\n\nDuring Part B:\n\n* In Part B, children with SMA will receive BIIB115. Both researchers and participants will know they are receiving BIIB115.\n* Participants will receive 2 total doses of BIIB115 given at 2 different times.\n* The treatment and follow up period will last for 25 months.\n* Participants will have up to 14 clinic visits and 6 telephone calls.\n\nIn both parts, participants will stay in the clinic for 24 hours after each dose for so that researchers can check on their health and any medical problems they might have." + }, + "conditionsModule": { + "conditions": ["Healthy Volunteer", "Muscular Atrophy, Spinal"] + }, + "designModule": { + "studyType": "INTERVENTIONAL", + "phases": ["PHASE1"], + "designInfo": { + "allocation": "RANDOMIZED", + "interventionModel": "SEQUENTIAL", + "interventionModelDescription": "Sequential for Parts A and B, parallel for Cohorts 1-4 within Part A, parallel for Cohorts 5-6 within Part B", + "primaryPurpose": "TREATMENT", + "maskingInfo": { + "masking": "QUADRUPLE", + "maskingDescription": "Blinded for Part A and open-label for Part B of the study.", + "whoMasked": [ + "PARTICIPANT", + "CARE_PROVIDER", + "INVESTIGATOR", + "OUTCOMES_ASSESSOR" + ] + } + }, + "enrollmentInfo": { "count": 62, "type": "ESTIMATED" } + }, + "armsInterventionsModule": { + "armGroups": [ + { + "label": "Part A: Cohort 1: BIIB115 Dose 1", + "type": "EXPERIMENTAL", + "description": "Participants will receive a single dose of BIIB115, Dose 1, via IT bolus injection, on Day 1.", + "interventionNames": ["Drug: BIIB115"] + }, + { + "label": "Part A: Cohort 2: BIIB115 Dose 2", + "type": "EXPERIMENTAL", + "description": "Participants will receive a single dose of BIIB115, Dose 2, via IT bolus injection, on Day 1.", + "interventionNames": ["Drug: BIIB115"] + }, + { + "label": "Part A: Cohort 3: BIIB115 Dose 3", + "type": "EXPERIMENTAL", + "description": "Participants will receive a single dose of BIIB115, Dose 3, via IT bolus injection, on Day 1.", + "interventionNames": ["Drug: BIIB115"] + }, + { + "label": "Part A: Cohort 4: BIIB115 Dose 4", + "type": "EXPERIMENTAL", + "description": "Participants will receive a single dose of BIIB115, Dose 4, via IT bolus injection, on Day 1.", + "interventionNames": ["Drug: BIIB115"] + }, + { + "label": "Part A: Cohorts 1-4: BIIB115-Matching Placebo", + "type": "PLACEBO_COMPARATOR", + "description": "Participants will receive a single dose of BIIB115-matching placebo, via IT bolus injection, on Day 1.", + "interventionNames": ["Drug: BIIB115-Matching Placebo"] + }, + { + "label": "Part B: Cohort 5: BIIB115 Dose 3", + "type": "EXPERIMENTAL", + "description": "Pediatric SMA participants previously treated with onasemnogene abeparvovec will receive two doses of BIIB115, Dose 3, via IT bolus injection at two separate time points.", + "interventionNames": ["Drug: BIIB115"] + }, + { + "label": "Part B: Cohort 6: BIIB115 Dose 4", + "type": "EXPERIMENTAL", + "description": "Pediatric SMA participants previously treated with onasemnogene abeparvovec will receive two doses of BIIB115, Dose 4, via IT bolus injectionat two separate time points.", + "interventionNames": ["Drug: BIIB115"] + } + ], + "interventions": [ + { + "type": "DRUG", + "name": "BIIB115", + "description": "Administered as specified in the treatment arm", + "armGroupLabels": [ + "Part A: Cohort 1: BIIB115 Dose 1", + "Part A: Cohort 2: BIIB115 Dose 2", + "Part A: Cohort 3: BIIB115 Dose 3", + "Part A: Cohort 4: BIIB115 Dose 4", + "Part B: Cohort 5: BIIB115 Dose 3", + "Part B: Cohort 6: BIIB115 Dose 4" + ] + }, + { + "type": "DRUG", + "name": "BIIB115-Matching Placebo", + "description": "Administered as specified in the treatment arm", + "armGroupLabels": [ + "Part A: Cohorts 1-4: BIIB115-Matching Placebo" + ] + } + ] + }, + "outcomesModule": { + "primaryOutcomes": [ + { + "measure": "Parts A and B: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)", + "description": "An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.\n\nA serious adverse event (SAE) is any untoward medical occurrence that at any dose results in death, in the view of the investigator, places the participant at immediate risk of death (a life-threatening event), requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect or is a medically important event.", + "timeFrame": "Part A: Up to Day 393, Part B: Up to Day 720" + } + ], + "secondaryOutcomes": [ + { + "measure": "Parts A and B: Concentration of BIIB115 in Cerebral Spinal Fluid (CSF)", + "timeFrame": "Part A: Day 1 to Day 180, Part B: Days 1 and 360" + }, + { + "measure": "Part A: Terminal Elimination Half-Life (t\u00bd) of BIIB115 in CSF", + "timeFrame": "Day 1 to Day 180" + }, + { + "measure": "Parts A and B: Concentration of BIIB115 in Serum", + "timeFrame": "Part A: Day 1 to Day 180, Part B: Day 1 to 720" + }, + { + "measure": "Parts A and B: Terminal Elimination Half-Life (t\u00bd) of BIIB115 in Serum", + "timeFrame": "Part A: Day 1 to Day 180, Part B: Day 1 to Day 720" + }, + { + "measure": "Parts A and B: Area Under the Concentration-Time Curve from Time 0 to Last Measurable Concentration (AUC0-last) of BIIB115 in Serum", + "timeFrame": "Part A: Day 1 to Day 180, Part B: Day 1 to Day 720" + }, + { + "measure": "Parts A and B: Area Under the Concentration-Time Curve from Time 0 to Infinity (AUCinf) of BIIB115 in Serum", + "timeFrame": "Part A: Day 1 to Day 180, Part B: Day 1 to Day 720" + }, + { + "measure": "Parts A and B: Maximum Observed Concentration (Cmax) of BIIB115 in Serum", + "timeFrame": "Part A: Day 1 to Day 180, Part B: Day 1 to Day 720" + }, + { + "measure": "Parts A and B: Time to Reach Maximum Observed Concentration (Tmax) of BIIB115 in Serum", + "timeFrame": "Part A: Day 1 to Day 180, Part B: Day 1 to Day 720" + } + ] + }, + "eligibilityModule": { + "eligibilityCriteria": "Key Inclusion Criteria:\n\nPart A:\n\n* Male healthy participants aged 18 to 55 years, inclusive\n* Have a body mass index of 18 to 30 kilograms per meter square (kg/m\\^2), inclusive\n* Must be in good health as determined by the investigator, based on medical history and screening evaluations\n\nPart B:\n\n* Age 0.5 to 12 years old, inclusive, at the time of informed consent\n* Weight \u22657 kg at the time of informed consent\n* Genetic diagnosis of SMA (5q SMA homozygous survival motor neuron 1 (SMN1) gene deletion or mutation or compound heterozygous mutation)\n* Survival motor neuron 2 (SMN2) copy number \u22651\n* Must have received intravenous (IV) onasemnogene abeparvovec per the approved label or per guidelines including the steroid regimen and monitoring specified therein\n* Treatment with onasemnogene abeparvovec \u2265180 days prior to first BIIB115 dose\n* Potential for improvement due to suboptimal clinical status secondary to SMA, as determined by the Investigator\n\nKey Exclusion Criteria:\n\nPart A:\n\n* Any reason, anatomical or otherwise (including abnormal hematology/coagulation), that presents increase of risk of complication from multiple lumbar puncture (LP) procedures required for dosing and CSF collection, per the investigator discretion\n* History of any clinically significant cardiac, endocrine, gastrointestinal, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, or renal disease, or other major disease, as determined by the Investigator\n* Chronic, recurrent, or serious infection, as determined by the investigator, within 90 days prior to screening or between screening and Day -1\n* Current enrollment or a plan to enroll in any interventional clinical study of a drug, biologic, or device, in which an investigational treatment or approved therapy for investigational use is administered within 3 months (or 5 half-lives of the agent, whichever is longer) prior to randomization\n\nPart B:\n\n* Severe or serious AEs related to onasemnogene abeparvovec therapy that are ongoing during Screening\n* Interval of \\<180 days between onasemnogene abeparvovec therapy and first BIIB115 dose\n* Ongoing steroid treatment following onasemnogene abeparvovec at time of screening\n* History of drug induced liver injury or liver failure per Hy's law definition\n* History of thrombotic micrangiopathy\n* Treatment with any SMN2-splicing modifier (nusinersen or risdiplam) after receiving onasemnogene abeparvovec. Treatment with nusinersen \\<12 months from the first dose of BIIB115.\n* Any reason, anatomical or otherwise (including abnormal hematology/coagulation), that presents increase of risk of complication from the LP procedures, CSF circulation, or safety assessments, including a history of hydrocephalus or implanted shunt for CSF drainage.\n* Permanent ventilation, defined as tracheostomy or \u226516 hours ventilation /day continuously for \\>21 days in the absence of an acute reversible event\n\nNOTE: Other protocol defined Inclusion/Exclusion criteria may apply.", + "healthyVolunteers": true, + "sex": "ALL", + "minimumAge": "6 Months", + "maximumAge": "55 Years", + "stdAges": ["CHILD", "ADULT"] + }, + "contactsLocationsModule": { + "centralContacts": [ + { + "name": "US Biogen Clinical Trial Center", + "role": "CONTACT", + "phone": "866-633-4636", + "email": "clinicaltrials@biogen.com" + }, + { + "name": "Global Biogen Clinical Trial Center", + "role": "CONTACT", + "email": "clinicaltrials@biogen.com" + } + ], + "overallOfficials": [ + { + "name": "Medical Director", + "affiliation": "Biogen", + "role": "STUDY_DIRECTOR" + } + ], + "locations": [ + { + "facility": "Universitair Ziekenhuis Gent", + "status": "RECRUITING", + "city": "Gent", + "zip": "9000", + "country": "Belgium", + "contacts": [ + { + "role": "CONTACT", + "phone": "3293321954", + "email": "elke.devos@uzgent.be" + }, + { + "name": "Nicolas Deconinck", + "role": "PRINCIPAL_INVESTIGATOR" + } + ], + "geoPoint": { "lat": 51.05, "lon": 3.71667 } + }, + { + "facility": "Children's Hospital of Eastern Ontario", + "status": "RECRUITING", + "city": "Ontario", + "zip": "K1H 8L1", + "country": "Canada", + "contacts": [ + { + "role": "CONTACT", + "phone": "(613) 737-7600 x6051", + "email": "hmcmillan@cheo.on.ca" + }, + { "name": "Hugh McMillan", "role": "PRINCIPAL_INVESTIGATOR" } + ] + }, + { + "facility": "H\u00f4pital Armand Trousseau", + "status": "RECRUITING", + "city": "Paris", + "zip": "75012", + "country": "France", + "contacts": [ + { + "role": "CONTACT", + "phone": "33171738393", + "email": "a.seferian@institut-myologie.org" + }, + { "name": "Andreea Sefarian", "role": "PRINCIPAL_INVESTIGATOR" } + ], + "geoPoint": { "lat": 48.85341, "lon": 2.3488 } + }, + { + "facility": "Universitatsklinikum Essen", + "status": "RECRUITING", + "city": "Essen", + "zip": "45147", + "country": "Germany", + "contacts": [ + { + "role": "CONTACT", + "phone": "+492017232508", + "email": "heike.koelbel@uk-essen.de" + }, + { "name": "Heike Koelbel", "role": "PRINCIPAL_INVESTIGATOR" } + ], + "geoPoint": { "lat": 51.45657, "lon": 7.01228 } + }, + { + "facility": "Universitaetsklinikum Freiburg", + "status": "RECRUITING", + "city": "Freiburg", + "zip": "79106", + "country": "Germany", + "contacts": [ + { + "role": "CONTACT", + "phone": "+49 761 27042999", + "email": "astrid.pechmann@uniklinik-freiburg.de" + }, + { "name": "Astrid Pechmann", "role": "PRINCIPAL_INVESTIGATOR" } + ], + "geoPoint": { "lat": 47.9959, "lon": 7.85222 } + }, + { + "facility": "Universitaetsklinikum Heidelberg", + "status": "RECRUITING", + "city": "Heidelberg", + "zip": "69120", + "country": "Germany", + "contacts": [ + { + "role": "CONTACT", + "phone": "496221564002", + "email": "Andreas.Ziegler@med.uni-heidelberg.de" + }, + { "name": "Andreas Ziegler", "role": "PRINCIPAL_INVESTIGATOR" } + ], + "geoPoint": { "lat": 49.40768, "lon": 8.69079 } + }, + { + "facility": "Schneider Children's Medical Center", + "status": "RECRUITING", + "city": "Petach-Tikvah", + "zip": "4920235", + "country": "Israel", + "contacts": [ + { + "role": "CONTACT", + "phone": "972-3-9253874", + "email": "sharonah@clalit.org.il" + }, + { "name": "Sharon Aharoni", "role": "PRINCIPAL_INVESTIGATOR" } + ], + "geoPoint": { "lat": 32.08707, "lon": 34.88747 } + }, + { + "facility": "Fondazione Serena Onlus - Centro Clinico Nemo", + "status": "RECRUITING", + "city": "Milano", + "zip": "20162", + "country": "Italy", + "contacts": [ + { + "role": "CONTACT", + "phone": "390291433264", + "email": "valeria.sansone@centrocliniconemo.it" + }, + { "name": "Valeria Sansone", "role": "PRINCIPAL_INVESTIGATOR" } + ], + "geoPoint": { "lat": 45.46427, "lon": 9.18951 } + }, + { + "facility": "Pediatric Neurology Unit, Catholic University", + "status": "RECRUITING", + "city": "Rome", + "zip": "00168", + "country": "Italy", + "contacts": [ + { + "role": "CONTACT", + "phone": "390630155600", + "email": "marika.pane@policlinicogemelli.it" + }, + { "name": "Marika Pane", "role": "PRINCIPAL_INVESTIGATOR" } + ], + "geoPoint": { "lat": 41.89193, "lon": 12.51133 } + }, + { + "facility": "Kyungpook National University Hospital", + "status": "RECRUITING", + "city": "Daegu", + "zip": "700-721", + "country": "Korea, Republic of", + "geoPoint": { "lat": 35.87028, "lon": 128.59111 } + }, + { + "facility": "Seoul National University Hospital", + "status": "RECRUITING", + "city": "Seoul", + "zip": "03080", + "country": "Korea, Republic of", + "geoPoint": { "lat": 37.566, "lon": 126.9784 } + }, + { + "facility": "Centre For Human Drug Research", + "status": "ACTIVE_NOT_RECRUITING", + "city": "Leiden", + "zip": "2333", + "country": "Netherlands", + "geoPoint": { "lat": 52.15833, "lon": 4.49306 } + }, + { + "facility": "UMC Utrecht", + "status": "RECRUITING", + "city": "Utrecht", + "zip": "3584 CX", + "country": "Netherlands", + "contacts": [ + { "role": "CONTACT", "phone": "088-7550646" }, + { "name": "Ludo Van der Pol", "role": "PRINCIPAL_INVESTIGATOR" } + ], + "geoPoint": { "lat": 52.09083, "lon": 5.12222 } + }, + { + "facility": "Instytut Centrum Zdrowia Matki Polki Dept of Neurology", + "status": "RECRUITING", + "city": "Lodz", + "zip": "93-338", + "country": "Poland", + "contacts": [ + { + "role": "CONTACT", + "phone": "48606989185", + "email": "l.przyslo@gmail.com" + }, + { "name": "Lukasz Przyslo", "role": "PRINCIPAL_INVESTIGATOR" } + ], + "geoPoint": { "lat": 51.75, "lon": 19.46667 } + }, + { + "facility": "Instytut \"Pomnik - Centrum Zdrowia Dziecka", + "status": "RECRUITING", + "city": "Warsaw", + "zip": "04-730", + "country": "Poland", + "contacts": [ + { + "role": "CONTACT", + "phone": "48228151603", + "email": "k.kotulska@ipczd.pl" + }, + { + "name": "Katarzyna Kotulska-Jozwiak", + "role": "PRINCIPAL_INVESTIGATOR" + } + ], + "geoPoint": { "lat": 52.22977, "lon": 21.01178 } + }, + { + "facility": "PRATIA S.A. MTZ Clinical Research Powered by Pratia", + "status": "ACTIVE_NOT_RECRUITING", + "city": "Warszawa", + "zip": "02-172", + "country": "Poland", + "geoPoint": { "lat": 52.22977, "lon": 21.01178 } + }, + { + "facility": "Great Ormond Street Hospital for Children", + "status": "RECRUITING", + "city": "Bloomsbury", + "zip": "WC1N 3JH", + "country": "United Kingdom", + "contacts": [ + { + "role": "CONTACT", + "phone": "02077626932", + "email": "Mariacristina.scoto@gosh.nhs.uk" + }, + { + "name": "Mariacristina Scoto", + "role": "PRINCIPAL_INVESTIGATOR" + } + ], + "geoPoint": { "lat": 51.52228, "lon": -0.12252 } + }, + { + "facility": "Sheffield Childrens Hospital", + "status": "RECRUITING", + "city": "Sheffield", + "zip": "S10 2TH", + "country": "United Kingdom", + "contacts": [ + { + "role": "CONTACT", + "phone": "1143053951", + "email": "min.ong@nhs.net" + }, + { "name": "Min Ong", "role": "PRINCIPAL_INVESTIGATOR" } + ], + "geoPoint": { "lat": 53.38297, "lon": -1.4659 } + } + ] + }, + "ipdSharingStatementModule": { + "ipdSharing": "YES", + "description": "In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/", + "url": "https://vivli.org/" + } + }, + "derivedSection": { + "miscInfoModule": { "versionHolder": "2024-12-04" }, + "conditionBrowseModule": { + "meshes": [ + { "id": "D009133", "term": "Muscular Atrophy" }, + { "id": "D009134", "term": "Muscular Atrophy, Spinal" }, + { "id": "D001284", "term": "Atrophy" } + ], + "ancestors": [ + { "id": "D020763", "term": "Pathological Conditions, Anatomical" }, + { "id": "D020879", "term": "Neuromuscular Manifestations" }, + { "id": "D009461", "term": "Neurologic Manifestations" }, + { "id": "D009422", "term": "Nervous System Diseases" }, + { "id": "D013118", "term": "Spinal Cord Diseases" }, + { "id": "D002493", "term": "Central Nervous System Diseases" }, + { "id": "D016472", "term": "Motor Neuron Disease" }, + { "id": "D019636", "term": "Neurodegenerative Diseases" }, + { "id": "D009468", "term": "Neuromuscular Diseases" } + ], + "browseLeaves": [ + { + "id": "M4589", + "name": "Atrophy", + "asFound": "Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12090", + "name": "Muscular Atrophy", + "asFound": "Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12091", + "name": "Muscular Atrophy, Spinal", + "asFound": "Muscular Atrophy, Spinal", + "relevance": "HIGH" + }, + { + "id": "M22519", + "name": "Pathological Conditions, Anatomical", + "relevance": "LOW" + }, + { + "id": "M22619", + "name": "Neuromuscular Manifestations", + "relevance": "LOW" + }, + { + "id": "M12404", + "name": "Neurologic Manifestations", + "relevance": "LOW" + }, + { + "id": "M15915", + "name": "Spinal Cord Diseases", + "relevance": "LOW" + }, + { + "id": "M5742", + "name": "Central Nervous System Diseases", + "relevance": "LOW" + }, + { + "id": "M18879", + "name": "Motor Neuron Disease", + "relevance": "LOW" + }, + { + "id": "M4024", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + }, + { + "id": "M21558", + "name": "Neurodegenerative Diseases", + "relevance": "LOW" + }, + { + "id": "M12411", + "name": "Neuromuscular Diseases", + "relevance": "LOW" + }, + { + "id": "T5342", + "name": "Spinal Muscular Atrophy", + "asFound": "Muscular Atrophy, Spinal", + "relevance": "HIGH" + }, + { + "id": "T349", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + } + ], + "browseBranches": [ + { "abbrev": "BC23", "name": "Symptoms and General Pathology" }, + { "abbrev": "All", "name": "All Conditions" }, + { "abbrev": "BC10", "name": "Nervous System Diseases" }, + { "abbrev": "BC18", "name": "Nutritional and Metabolic Diseases" }, + { "abbrev": "Rare", "name": "Rare Diseases" } + ] + } + }, + "hasResults": false + }, + { + "protocolSection": { + "identificationModule": { + "nctId": "NCT03306277", + "orgStudyIdInfo": { "id": "AVXS-101-CL-303" }, + "secondaryIdInfos": [ + { "id": "2020-000095-38", "type": "EUDRACT_NUMBER" }, + { + "id": "COAV101A12302", + "type": "OTHER", + "domain": "Novartis Pharmaceuticals" + } + ], + "organization": { "fullName": "Novartis", "class": "INDUSTRY" }, + "briefTitle": "Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1", + "officialTitle": "Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion", + "acronym": "STR1VE" + }, + "statusModule": { + "statusVerifiedDate": "2022-08", + "overallStatus": "COMPLETED", + "expandedAccessInfo": { "hasExpandedAccess": false }, + "startDateStruct": { "date": "2017-10-24", "type": "ACTUAL" }, + "primaryCompletionDateStruct": { + "date": "2019-11-12", + "type": "ACTUAL" + }, + "completionDateStruct": { "date": "2019-11-12", "type": "ACTUAL" }, + "studyFirstSubmitDate": "2017-10-02", + "studyFirstSubmitQcDate": "2017-10-04", + "studyFirstPostDateStruct": { + "date": "2017-10-11", + "type": "ACTUAL" + }, + "resultsFirstSubmitDate": "2020-06-25", + "resultsFirstSubmitQcDate": "2020-06-25", + "resultsFirstPostDateStruct": { + "date": "2020-07-16", + "type": "ACTUAL" + }, + "lastUpdateSubmitDate": "2022-08-12", + "lastUpdatePostDateStruct": { "date": "2022-08-16", "type": "ACTUAL" } + }, + "sponsorCollaboratorsModule": { + "responsibleParty": { "type": "SPONSOR" }, + "leadSponsor": { + "name": "Novartis Gene Therapies", + "class": "INDUSTRY" + } + }, + "oversightModule": { + "oversightHasDmc": true, + "isFdaRegulatedDrug": true, + "isFdaRegulatedDevice": false + }, + "descriptionModule": { + "briefSummary": "Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.", + "detailedDescription": "Phase 3, open-label, single-arm, single-dose, study of onasemnogene abeparvovec-xioi (gene replacement therapy) in participants with spinal muscular atrophy (SMA) Type 1 who meet enrollment criteria and are genetically defined by nonfunctional survival motor neuron 1 gene (SMN1) with 1 or 2 copies of survival motor neuron 2 gene (SMN2). Fifteen (15) participants \\< 6 months (\\< 180 days) of age at the time of gene replacement therapy (Day 1) will be enrolled." + }, + "conditionsModule": { + "conditions": ["SMA - Spinal Muscular Atrophy", "Gene Therapy"] + }, + "designModule": { + "studyType": "INTERVENTIONAL", + "phases": ["PHASE3"], + "designInfo": { + "allocation": "NA", + "interventionModel": "SINGLE_GROUP", + "interventionModelDescription": "One-time intravenous administration of onasemnogene abeparvovec-xioi.", + "primaryPurpose": "TREATMENT", + "maskingInfo": { "masking": "NONE" } + }, + "enrollmentInfo": { "count": 22, "type": "ACTUAL" } + }, + "armsInterventionsModule": { + "armGroups": [ + { + "label": "Onasemnogene Abeparvovec-xioi", + "type": "EXPERIMENTAL", + "description": "One-time Intravenous administration of onasemnogene abeparvovec-xioi at the therapeutic dose.", + "interventionNames": ["Biological: Onasemnogene Abeparvovec-xioi"] + } + ], + "interventions": [ + { + "type": "BIOLOGICAL", + "name": "Onasemnogene Abeparvovec-xioi", + "description": "Non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the complimentary deoxyribonucleic acid (cDNA) of the human SMN gene under the control of the cytomegalovirus (CMV) enhancer/chicken-\u03b2-actin-hybrid promoter (CB). The AAV inverted terminal repeat (ITR) has been modified to promote intramolecular annealing of the transgene, thus forming a double-stranded transgene ready for transcription.", + "armGroupLabels": ["Onasemnogene Abeparvovec-xioi"], + "otherNames": ["Zolgensma"] + } + ] + }, + "outcomesModule": { + "primaryOutcomes": [ + { + "measure": "Achievement of Independent Sitting for at Least 30 Seconds", + "description": "Independent sitting is defined as sitting up straight with head erect for at least 30 seconds.\n\nThis endpoint is a co-primary endpoint. The two co-primary efficacy endpoints were assessed in sequence: The endpoint of functional independent sitting was assessed first and, only when this assessment met statistical significance, was the endpoint of event-free survival assessed.", + "timeFrame": "Up to 18 months" + }, + { + "measure": "Event-free Survival", + "description": "Survival is defined by the avoidance of combined endpoint of either death or permanent ventilation, which is defined by tracheostomy or by the requirement of \u2265 16 hours of respiratory assistance per day for \u2265 14 consecutive days in the absence of an acute reversible illness, excluding perioperative ventilation. Permanent ventilation is considered a surrogate for death. An acute reversible illness is defined as any condition other than SMA that results in increased medical intervention.\n\nThe endpoint is a co-primary endpoint. The two co-primary efficacy endpoints were assessed in sequence: The endpoint of functional independent sitting was assessed first and, only when this assessment met statistical significance was the survival endpoint assessed.", + "timeFrame": "14 months" + } + ], + "secondaryOutcomes": [ + { + "measure": "Ability to Thrive", + "description": "Ability to thrive is defined as achieving all of the following at 18 months of age:\n\n* does not receive nutrition through mechanical support or other non-oral method\n* ability to tolerate thin liquids as demonstrated through a formal swallowing test\n* maintains weight\n\nThis is a co-secondary endpoint. The two co-secondary endpoints were assessed in sequence: The endpoint of ability to thrive was assessed first and, only when this assessment met statistical significance was the endpoint of ventilatory support independence assessed.", + "timeFrame": "18 months" + }, + { + "measure": "Ventilatory Support Independence", + "description": "Ventilatory support independence is defined as requiring no daily ventilator support/usage at 18 months of age, excluding acute reversible illness and perioperative ventilation, through assessment of actual usage data captured from the device (Phillips Trilogy BiPAP device). This endpoint is derived solely from the Phillips Trilogy BiPAP device.\n\nThis is a co-secondary endpoint. The two co-secondary endpoints were assessed in sequence: The endpoint of ability to thrive was assessed first and, only when this assessment met statistical significance was the endpoint of ventilatory support independence assessed.", + "timeFrame": "Up to 18 months" + } + ] + }, + "eligibilityModule": { + "eligibilityCriteria": "Inclusion Criteria:\n\n* Participants with SMA Type 1 as determined by the following features: a. Diagnosis of SMA based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and 1 or 2 copies of SMN2 (inclusive of the known SMN2 gene modifier mutation (c.859G\\>C))2\n* The first 3 participants enrolled must meet the criteria for the Intent-To-Treat Population\n* Participants must be \\< 6 months (\\< 180 days) of age at the time of onasemnogene abeparvovec-xioi infusion\n* Participants must have a swallowing evaluation test performed prior to administration of gene replacement therapy\n* Up-to-date on childhood vaccinations. Seasonal vaccinations that include palivizumab prophylaxis (also known as Synagis) to prevent respiratory syncytial virus (RSV) infections are also recommended in accordance with American Academy of Pediatrics\n* Parent(s)/legal guardian(s) willing and able to complete the informed consent process and comply with study procedures and visit schedule\n\nExclusion Criteria:\n\n* Previous, planned or expected scoliosis repair surgery/procedure during the study assessment period\n* Pulse oximetry \\< 96% saturation at screening while the participant is awake or asleep without any supplemental oxygen or respiratory support, or for altitudes \\> 1000 m, oxygen saturation \\< 92% awake or asleep without any supplemental oxygen or respiratory support Pulse oximetry saturation may decrease to \\< 96% after screening provided that the saturation does not decrease by \u2265 4 percentage points\n* Tracheostomy or current use or requirement of non-invasive ventilatory support averaging \u2265 6 hours daily over the 7 days prior to the screening visit; or \u2265 6 hours/day on average during the screening period or requiring ventilatory support while awake over the 7 days prior to screening or at any point during the screening period prior to dosing\n* Participants with signs of aspiration/inability to tolerate non-thickened- liquids based on a formal swallowing test performed as part of screening. Participants with a gastrostomy tube who pass the swallowing test will be allowed to enroll in the study\n* Participants whose weight-for-age is below the third percentile based on World Health Organization (WHO) Child Growth Standards\n* Active viral infection (includes human immunodeficiency virus \\[HIV\\] or positive serology for hepatitis B or C, or Zika virus)\n* Serious non-respiratory tract illness requiring systemic treatment and/or hospitalization within 2 weeks prior to screening\n* Upper or lower respiratory infection requiring medical attention, medical intervention, or increase in supportive care of any manner within 4 weeks prior to screening\n* Severe non-pulmonary/respiratory tract infection within 4 weeks before administration of gene replacement therapy or concomitant illness that creates unnecessary risks for gene replacement therapy such as: a. Major renal or hepatic impairment b. Known seizure disorder c. Diabetes mellitus d. Idiopathic hypocalcuria e. Symptomatic cardiomyopathy\n* Known allergy or hypersensitivity to prednisolone or other glucocorticosteroids or their excipients\n* Concomitant use of any of the following: drugs for treatment of myopathy or neuropathy, agents used to treat diabetes mellitus, or ongoing immunosuppressive therapy, plasmapheresis, immunomodulators such as adalimumab, immunosuppressive therapy within 3 months prior to gene replacement therapy\n* Anti-adeno-associated virus serotype 9 (AAV9) antibody titer \\> 1:50 as determined by Enzyme-linked Immunosorbent Assay (ELISA) binding immunoassay. Should a potential participant demonstrate Anti-AAV9 antibody titer \\> 1:50, he or she may receive retesting within 30 days of the screening period and will be eligible to participate if the Anti-AAV9 antibody titer upon retesting is \u2264 1:50\n* Clinically significant abnormal laboratory values (gamma glutamyl- transpeptidase \\[GGT\\], ALT, and AST \\> 3 \u00d7 ULN, bilirubin \u2265 3.0 mg/dL, creatinine \u2265 1.0 mg/dL, hemoglobin \\[Hgb\\] \\< 8 or \\> 18 g/dL; white blood cell \\[WBC\\] \\> 20,000 per cmm) prior to gene replacement therapy\n* Participation in recent SMA treatment clinical study (with the exception of observational Cohort studies or non-interventional studies) or receipt of an investigational or commercial compound, product, or therapy administered with the intent to treat SMA at any time prior to screening for this study. Oral \u03b2-agonists must be discontinued at least 30 days before gene therapy dosing. Inhaled albuterol specifically prescribed for the purposes of respiratory (bronchodilator) management is acceptable and not a contraindication at any time prior to screening for this study\n* Expectation of major surgical procedures during the study assessment period\n* Parent(s)/legal guardian(s) unable or unwilling to comply with study procedures or inability to travel for repeat visits\n* Parent(s)/legal guardian(s) unwilling to keep study results/observations confidential or to refrain from posting confidential study results/observations on social media sites\n* Parent(s)/legal guardian(s) refuses to sign consent form\n* Gestational age at birth \\< 35 weeks (245 days)", + "healthyVolunteers": false, + "sex": "ALL", + "maximumAge": "180 Days", + "stdAges": ["CHILD"] + }, + "contactsLocationsModule": { + "locations": [ + { + "facility": "David Geffen School of Medicine at UCLA", + "city": "Los Angeles", + "state": "California", + "zip": "90095", + "country": "United States", + "geoPoint": { "lat": 34.05223, "lon": -118.24368 } + }, + { + "facility": "Stanford University", + "city": "Stanford", + "state": "California", + "zip": "94305", + "country": "United States", + "geoPoint": { "lat": 37.42411, "lon": -122.16608 } + }, + { + "facility": "Children's Hospital Colorado", + "city": "Aurora", + "state": "Colorado", + "zip": "80045", + "country": "United States", + "geoPoint": { "lat": 39.72943, "lon": -104.83192 } + }, + { + "facility": "Nemours Children's Hospital", + "city": "Orlando", + "state": "Florida", + "zip": "32827", + "country": "United States", + "geoPoint": { "lat": 28.53834, "lon": -81.37924 } + }, + { + "facility": "Ann and Robert H Lurie Children's Hospital", + "city": "Chicago", + "state": "Illinois", + "zip": "60611", + "country": "United States", + "geoPoint": { "lat": 41.85003, "lon": -87.65005 } + }, + { + "facility": "Johns Hopkins Pediatric Neurology", + "city": "Baltimore", + "state": "Maryland", + "zip": "21287", + "country": "United States", + "geoPoint": { "lat": 39.29038, "lon": -76.61219 } + }, + { + "facility": "Boston Children's Hospital", + "city": "Boston", + "state": "Massachusetts", + "zip": "02115", + "country": "United States", + "geoPoint": { "lat": 42.35843, "lon": -71.05977 } + }, + { + "facility": "Washington Unviersity School of Medicine", + "city": "Saint Louis", + "state": "Missouri", + "zip": "63110", + "country": "United States", + "geoPoint": { "lat": 38.62727, "lon": -90.19789 } + }, + { + "facility": "Columbia University", + "city": "New York", + "state": "New York", + "zip": "10032", + "country": "United States", + "geoPoint": { "lat": 40.71427, "lon": -74.00597 } + }, + { + "facility": "Duke University", + "city": "Durham", + "state": "North Carolina", + "zip": "27713", + "country": "United States", + "geoPoint": { "lat": 35.99403, "lon": -78.89862 } + }, + { + "facility": "Nationwide Children's Hospital", + "city": "Columbus", + "state": "Ohio", + "zip": "43205", + "country": "United States", + "geoPoint": { "lat": 39.96118, "lon": -82.99879 } + }, + { + "facility": "Oregon Health and Science University", + "city": "Portland", + "state": "Oregon", + "zip": "97239", + "country": "United States", + "geoPoint": { "lat": 45.52345, "lon": -122.67621 } + }, + { + "facility": "Children's Hospital of Philadelphia", + "city": "Philadelphia", + "state": "Pennsylvania", + "zip": "19104", + "country": "United States", + "geoPoint": { "lat": 39.95233, "lon": -75.16379 } + }, + { + "facility": "University of Texas Southwestern Medical Center", + "city": "Dallas", + "state": "Texas", + "zip": "75235", + "country": "United States", + "geoPoint": { "lat": 32.78306, "lon": -96.80667 } + }, + { + "facility": "University of Utah", + "city": "Salt Lake City", + "state": "Utah", + "zip": "84112", + "country": "United States", + "geoPoint": { "lat": 40.76078, "lon": -111.89105 } + }, + { + "facility": "University of Wisconsin (Madison)", + "city": "Madison", + "state": "Wisconsin", + "zip": "53792", + "country": "United States", + "geoPoint": { "lat": 43.07305, "lon": -89.40123 } + } + ] + }, + "referencesModule": { + "references": [ + { + "pmid": "34383289", + "type": "DERIVED", + "citation": "Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Oct;44(10):1109-1119. doi: 10.1007/s40264-021-01107-6. Epub 2021 Aug 12. Erratum In: Drug Saf. 2022 Feb;45(2):191-192. doi: 10.1007/s40264-021-01130-7." + }, + { + "pmid": "33743238", + "type": "DERIVED", + "citation": "Day JW, Finkel RS, Chiriboga CA, Connolly AM, Crawford TO, Darras BT, Iannaccone ST, Kuntz NL, Pena LDM, Shieh PB, Smith EC, Kwon JM, Zaidman CM, Schultz M, Feltner DE, Tauscher-Wisniewski S, Ouyang H, Chand DH, Sproule DM, Macek TA, Mendell JR. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial. Lancet Neurol. 2021 Apr;20(4):284-293. doi: 10.1016/S1474-4422(21)00001-6. Epub 2021 Mar 17." + } + ], + "seeAlsoLinks": [ + { "label": "sponsor company website", "url": "http://AveXis.com" }, + { + "label": "Novartis Results Record", + "url": "https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=17657" + } + ] + }, + "ipdSharingStatementModule": { + "ipdSharing": "YES", + "description": "Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.\n\nThis trial data availability is according to the criteria and process described on https://www.clinicalstudydatarequest.com/.", + "url": "https://www.clinicalstudydatarequest.com/." + } + }, + "resultsSection": { + "participantFlowModule": { + "preAssignmentDetails": "A screening period of up to 30 days occurred before treatment.", + "recruitmentDetails": "22 participants were recruited across 16 study centers in the United States.", + "groups": [ + { + "id": "FG000", + "title": "Onasemnogene Abeparvovec-xioi", + "description": "One-time Intravenous administration of onasemnogene abeparvovec-xioi at the therapeutic dose.\n\nOnasemnogene Abeparvovec-xioi: Non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the complimentary deoxyribonucleic acid (cDNA) of the human SMN gene under the control of the cytomegalovirus (CMV) enhancer/chicken-\u03b2-actin-hybrid promoter (CB). The AAV inverted terminal repeat (ITR) has been modified to promote intramolecular annealing of the transgene, thus forming a double-stranded transgene ready for transcription." + } + ], + "periods": [ + { + "title": "Overall Study", + "milestones": [ + { + "type": "STARTED", + "achievements": [{ "groupId": "FG000", "numSubjects": "22" }] + }, + { + "type": "COMPLETED", + "achievements": [{ "groupId": "FG000", "numSubjects": "19" }] + }, + { + "type": "NOT COMPLETED", + "achievements": [{ "groupId": "FG000", "numSubjects": "3" }] + } + ], + "dropWithdraws": [ + { + "type": "Death", + "reasons": [{ "groupId": "FG000", "numSubjects": "1" }] + }, + { + "type": "Adverse Event", + "reasons": [{ "groupId": "FG000", "numSubjects": "1" }] + }, + { + "type": "Withdrawal by Subject", + "reasons": [{ "groupId": "FG000", "numSubjects": "1" }] + } + ] + } + ] + }, + "baselineCharacteristicsModule": { + "groups": [ + { + "id": "BG000", + "title": "Onasemnogene Abeparvovec-xioi", + "description": "One-time Intravenous administration of onasemnogene abeparvovec-xioi at the therapeutic dose.\n\nOnasemnogene Abeparvovec-xioi: Non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the complimentary deoxyribonucleic acid (cDNA) of the human SMN gene under the control of the cytomegalovirus (CMV) enhancer/chicken-\u03b2-actin-hybrid promoter (CB). The AAV inverted terminal repeat (ITR) has been modified to promote intramolecular annealing of the transgene, thus forming a double-stranded transgene ready for transcription." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "BG000", "value": "22" }] + } + ], + "measures": [ + { + "title": "Age, Categorical", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "title": "<=18 years", + "measurements": [{ "groupId": "BG000", "value": "22" }] + }, + { + "title": "Between 18 and 65 years", + "measurements": [{ "groupId": "BG000", "value": "0" }] + }, + { + "title": ">=65 years", + "measurements": [{ "groupId": "BG000", "value": "0" }] + } + ] + } + ] + }, + { + "title": "Age, Continuous", + "paramType": "MEAN", + "dispersionType": "STANDARD_DEVIATION", + "unitOfMeasure": "months", + "classes": [ + { + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "3.7", "spread": "1.6" } + ] + } + ] + } + ] + }, + { + "title": "Sex: Female, Male", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "title": "Female", + "measurements": [{ "groupId": "BG000", "value": "12" }] + }, + { + "title": "Male", + "measurements": [{ "groupId": "BG000", "value": "10" }] + } + ] + } + ] + }, + { + "title": "Ethnicity (NIH/OMB)", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "title": "Hispanic or Latino", + "measurements": [{ "groupId": "BG000", "value": "4" }] + }, + { + "title": "Not Hispanic or Latino", + "measurements": [{ "groupId": "BG000", "value": "18" }] + }, + { + "title": "Unknown or Not Reported", + "measurements": [{ "groupId": "BG000", "value": "0" }] + } + ] + } + ] + }, + { + "title": "Race (NIH/OMB)", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "classes": [ + { + "categories": [ + { + "title": "American Indian or Alaska Native", + "measurements": [{ "groupId": "BG000", "value": "0" }] + }, + { + "title": "Asian", + "measurements": [{ "groupId": "BG000", "value": "2" }] + }, + { + "title": "Native Hawaiian or Other Pacific Islander", + "measurements": [{ "groupId": "BG000", "value": "0" }] + }, + { + "title": "Black or African American", + "measurements": [{ "groupId": "BG000", "value": "3" }] + }, + { + "title": "White", + "measurements": [{ "groupId": "BG000", "value": "11" }] + }, + { + "title": "More than one race", + "measurements": [{ "groupId": "BG000", "value": "0" }] + }, + { + "title": "Unknown or Not Reported", + "measurements": [{ "groupId": "BG000", "value": "6" }] + } + ] + } + ] + }, + { + "title": "Region of Enrollment", + "paramType": "NUMBER", + "unitOfMeasure": "participants", + "classes": [ + { + "title": "United States", + "categories": [ + { "measurements": [{ "groupId": "BG000", "value": "22" }] } + ] + } + ] + }, + { + "title": "Patient reported hospitalizations", + "paramType": "NUMBER", + "unitOfMeasure": "participants", + "classes": [ + { + "title": "Yes", + "categories": [ + { "measurements": [{ "groupId": "BG000", "value": "17" }] } + ] + }, + { + "title": "No", + "categories": [ + { "measurements": [{ "groupId": "BG000", "value": "5" }] } + ] + } + ] + }, + { + "title": "Weight at baseline", + "paramType": "MEAN", + "dispersionType": "STANDARD_DEVIATION", + "unitOfMeasure": "kg", + "classes": [ + { + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "5.8", "spread": "1.1" } + ] + } + ] + } + ] + }, + { + "title": "Height/length at baseline", + "paramType": "MEAN", + "dispersionType": "STANDARD_DEVIATION", + "unitOfMeasure": "cm", + "classes": [ + { + "categories": [ + { + "measurements": [ + { "groupId": "BG000", "value": "61.3", "spread": "4.3" } + ] + } + ] + } + ] + } + ] + }, + "outcomeMeasuresModule": { + "outcomeMeasures": [ + { + "type": "PRIMARY", + "title": "Achievement of Independent Sitting for at Least 30 Seconds", + "description": "Independent sitting is defined as sitting up straight with head erect for at least 30 seconds.\n\nThis endpoint is a co-primary endpoint. The two co-primary efficacy endpoints were assessed in sequence: The endpoint of functional independent sitting was assessed first and, only when this assessment met statistical significance, was the endpoint of event-free survival assessed.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "Up to 18 months", + "groups": [ + { + "id": "OG000", + "title": "Onasemnogene Abeparvovec-xioi", + "description": "One-time Intravenous administration of onasemnogene abeparvovec-xioi at the therapeutic dose.\n\nOnasemnogene Abeparvovec-xioi: Non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the complimentary deoxyribonucleic acid (cDNA) of the human SMN gene under the control of the cytomegalovirus (CMV) enhancer/chicken-\u03b2-actin-hybrid promoter (CB). The AAV inverted terminal repeat (ITR) has been modified to promote intramolecular annealing of the transgene, thus forming a double-stranded transgene ready for transcription." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "22" }] + } + ], + "classes": [ + { + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "13" }] } + ] + } + ], + "analyses": [ + { + "groupIds": ["OG000"], + "groupDescription": "This comparison is made to an assumed rate of zero 0 (or as low as 0.1%). By definition, children with spinal muscular atrophy Type 1 are never able to sit independently.", + "nonInferiorityType": "SUPERIORITY", + "pValue": "<0.0001", + "statisticalMethod": "One-sided Exact Binomial Test" + } + ] + }, + { + "type": "PRIMARY", + "title": "Event-free Survival", + "description": "Survival is defined by the avoidance of combined endpoint of either death or permanent ventilation, which is defined by tracheostomy or by the requirement of \u2265 16 hours of respiratory assistance per day for \u2265 14 consecutive days in the absence of an acute reversible illness, excluding perioperative ventilation. Permanent ventilation is considered a surrogate for death. An acute reversible illness is defined as any condition other than SMA that results in increased medical intervention.\n\nThe endpoint is a co-primary endpoint. The two co-primary efficacy endpoints were assessed in sequence: The endpoint of functional independent sitting was assessed first and, only when this assessment met statistical significance was the survival endpoint assessed.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "14 months", + "groups": [ + { + "id": "OG000", + "title": "Onasemnogene Abeparvovec-xioi", + "description": "One-time Intravenous administration of onasemnogene abeparvovec-xioi at the therapeutic dose.\n\nOnasemnogene Abeparvovec-xioi: Non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the complimentary deoxyribonucleic acid (cDNA) of the human SMN gene under the control of the cytomegalovirus (CMV) enhancer/chicken-\u03b2-actin-hybrid promoter (CB). The AAV inverted terminal repeat (ITR) has been modified to promote intramolecular annealing of the transgene, thus forming a double-stranded transgene ready for transcription." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "22" }] + } + ], + "classes": [ + { + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "20" }] } + ] + } + ], + "analyses": [ + { + "groupIds": ["OG000"], + "groupDescription": "This comparison is made against the results from the age and gender-matched control participants selected from existing natural history data sets (PNCR) \\[Neurol. 2014; 83(9):810-817\\].", + "nonInferiorityType": "SUPERIORITY", + "pValue": "<0.0001", + "statisticalMethod": "Fisher Exact", + "otherAnalysisDescription": "Data for the current study were compared to historical control data (Finkel et al,2014 - PubMed 25080519) where event-free survival was 6 out of 23 participants (26.1%) at 14 months of age." + } + ] + }, + { + "type": "SECONDARY", + "title": "Ability to Thrive", + "description": "Ability to thrive is defined as achieving all of the following at 18 months of age:\n\n* does not receive nutrition through mechanical support or other non-oral method\n* ability to tolerate thin liquids as demonstrated through a formal swallowing test\n* maintains weight\n\nThis is a co-secondary endpoint. The two co-secondary endpoints were assessed in sequence: The endpoint of ability to thrive was assessed first and, only when this assessment met statistical significance was the endpoint of ventilatory support independence assessed.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "18 months", + "groups": [ + { + "id": "OG000", + "title": "Onasemnogene Abeparvovec-xioi", + "description": "One-time Intravenous administration of onasemnogene abeparvovec-xioi at the therapeutic dose.\n\nOnasemnogene Abeparvovec-xioi: Non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the complimentary deoxyribonucleic acid (cDNA) of the human SMN gene under the control of the cytomegalovirus (CMV) enhancer/chicken-\u03b2-actin-hybrid promoter (CB). The AAV inverted terminal repeat (ITR) has been modified to promote intramolecular annealing of the transgene, thus forming a double-stranded transgene ready for transcription." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "22" }] + } + ], + "classes": [ + { + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "9" }] } + ] + } + ] + }, + { + "type": "SECONDARY", + "title": "Ventilatory Support Independence", + "description": "Ventilatory support independence is defined as requiring no daily ventilator support/usage at 18 months of age, excluding acute reversible illness and perioperative ventilation, through assessment of actual usage data captured from the device (Phillips Trilogy BiPAP device). This endpoint is derived solely from the Phillips Trilogy BiPAP device.\n\nThis is a co-secondary endpoint. The two co-secondary endpoints were assessed in sequence: The endpoint of ability to thrive was assessed first and, only when this assessment met statistical significance was the endpoint of ventilatory support independence assessed.", + "reportingStatus": "POSTED", + "paramType": "COUNT_OF_PARTICIPANTS", + "unitOfMeasure": "Participants", + "timeFrame": "Up to 18 months", + "groups": [ + { + "id": "OG000", + "title": "Onasemnogene Abeparvovec-xioi", + "description": "One-time Intravenous administration of onasemnogene abeparvovec-xioi at the therapeutic dose.\n\nOnasemnogene Abeparvovec-xioi: Non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the complimentary deoxyribonucleic acid (cDNA) of the human SMN gene under the control of the cytomegalovirus (CMV) enhancer/chicken-\u03b2-actin-hybrid promoter (CB). The AAV inverted terminal repeat (ITR) has been modified to promote intramolecular annealing of the transgene, thus forming a double-stranded transgene ready for transcription." + } + ], + "denoms": [ + { + "units": "Participants", + "counts": [{ "groupId": "OG000", "value": "22" }] + } + ], + "classes": [ + { + "categories": [ + { "measurements": [{ "groupId": "OG000", "value": "18" }] } + ] + } + ] + } + ] + }, + "adverseEventsModule": { + "frequencyThreshold": "5", + "timeFrame": "Up to 18 months", + "eventGroups": [ + { + "id": "EG000", + "title": "Onasemnogene Abeparvovec-xioi", + "description": "One-time Intravenous administration of onasemnogene abeparvovec-xioi at the therapeutic dose.\n\nOnasemnogene Abeparvovec-xioi: Non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the complimentary deoxyribonucleic acid (cDNA) of the human SMN gene under the control of the cytomegalovirus (CMV) enhancer/chicken-\u03b2-actin-hybrid promoter (CB). The AAV inverted terminal repeat (ITR) has been modified to promote intramolecular annealing of the transgene, thus forming a double-stranded transgene ready for transcription.", + "deathsNumAffected": 1, + "deathsNumAtRisk": 22, + "seriousNumAffected": 10, + "seriousNumAtRisk": 22, + "otherNumAffected": 22, + "otherNumAtRisk": 22 + } + ], + "seriousEvents": [ + { + "term": "Alanine aminotransferase increased", + "organSystem": "Investigations", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Aspartate aminotransferase increased", + "organSystem": "Investigations", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Human metapneumovirus test positive", + "organSystem": "Investigations", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Transaminases increased", + "organSystem": "Investigations", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Cyanosis", + "organSystem": "Cardiac disorders", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Respiratory distress", + "organSystem": "Respiratory, thoracic and mediastinal disorders", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 7, + "numAffected": 4, + "numAtRisk": 22 + } + ] + }, + { + "term": "Respiratory failure", + "organSystem": "Respiratory, thoracic and mediastinal disorders", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 2, + "numAffected": 2, + "numAtRisk": 22 + } + ] + }, + { + "term": "Acute respiratory failure", + "organSystem": "Respiratory, thoracic and mediastinal disorders", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Atelectasis", + "organSystem": "Respiratory, thoracic and mediastinal disorders", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 2, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Pneumonia aspiration", + "organSystem": "Respiratory, thoracic and mediastinal disorders", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Respiratory arrest", + "organSystem": "Respiratory, thoracic and mediastinal disorders", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Hydrocephalus", + "organSystem": "Nervous system disorders", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Dysphagia", + "organSystem": "Gastrointestinal disorders", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Device malfunction", + "organSystem": "Product Issues", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Abnormal weight gain", + "organSystem": "Metabolism and nutrition disorders", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Failure to thrive", + "organSystem": "Metabolism and nutrition disorders", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Feeding disorder", + "organSystem": "Metabolism and nutrition disorders", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Bronchiolitis", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 2, + "numAffected": 2, + "numAtRisk": 22 + } + ] + }, + { + "term": "Pneumonia", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 2, + "numAffected": 2, + "numAtRisk": 22 + } + ] + }, + { + "term": "Respiratory syncytial virus bronchiolitis", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 2, + "numAffected": 2, + "numAtRisk": 22 + } + ] + }, + { + "term": "Bacterial tracheitis", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Device related infection", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Pneumonia bacterial", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Rhinovirus infection", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 2, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Sepsis", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + }, + { + "term": "Upper respiratory tract infection", + "organSystem": "Infections and infestations", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 1, + "numAffected": 1, + "numAtRisk": 22 + } + ] + } + ], + "otherEvents": [ + { + "term": "Diastolic hypertension", + "organSystem": "Vascular disorders", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 2, + "numAffected": 2, + "numAtRisk": 22 + } + ] + }, + { + "term": "Pyrexia", + "organSystem": "General disorders", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 26, + "numAffected": 12, + "numAtRisk": 22 + } + ] + }, + { + "term": "Use of accessory respiratory muscles", + "organSystem": "Respiratory, thoracic and mediastinal disorders", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 7, + "numAffected": 5, + "numAtRisk": 22 + } + ] + }, + { + "term": "Contusion", + "organSystem": "Injury, poisoning and procedural complications", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 4, + "numAffected": 4, + "numAtRisk": 22 + } + ] + }, + { + "term": "Arthropod bite", + "organSystem": "Injury, poisoning and procedural complications", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 7, + "numAffected": 3, + "numAtRisk": 22 + } + ] + }, + { + "term": "Aspartate aminotransferase increased", + "organSystem": "Investigations", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 9, + "numAffected": 6, + "numAtRisk": 22 + } + ] + }, + { + "term": "Alanine aminotransferase increased", + "organSystem": "Investigations", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": "NON_SYSTEMATIC_ASSESSMENT", + "stats": [ + { + "groupId": "EG000", + "numEvents": 8, + "numAffected": 5, + "numAtRisk": 22 + } + ] + }, + { + "term": "Blood creatine phosphokinase MB increased", + "organSystem": "Investigations", + "sourceVocabulary": "MedDRA (20.1)", + "assessmentType": 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}, + "derivedSection": { + "miscInfoModule": { "versionHolder": "2024-12-04" }, + "conditionBrowseModule": { + "meshes": [ + { "id": "D009133", "term": "Muscular Atrophy" }, + { "id": "D009134", "term": "Muscular Atrophy, Spinal" }, + { "id": "D001284", "term": "Atrophy" } + ], + "ancestors": [ + { "id": "D020763", "term": "Pathological Conditions, Anatomical" }, + { "id": "D020879", "term": "Neuromuscular Manifestations" }, + { "id": "D009461", "term": "Neurologic Manifestations" }, + { "id": "D009422", "term": "Nervous System Diseases" }, + { "id": "D013118", "term": "Spinal Cord Diseases" }, + { "id": "D002493", "term": "Central Nervous System Diseases" }, + { "id": "D016472", "term": "Motor Neuron Disease" }, + { "id": "D019636", "term": "Neurodegenerative Diseases" }, + { "id": "D009468", "term": "Neuromuscular Diseases" } + ], + "browseLeaves": [ + { + "id": "M4589", + "name": "Atrophy", + "asFound": "Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12090", + "name": "Muscular 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Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101", + "acronym": "START" + }, + "statusModule": { + "statusVerifiedDate": "2024-02", + "overallStatus": "ACTIVE_NOT_RECRUITING", + "expandedAccessInfo": { "hasExpandedAccess": false }, + "startDateStruct": { "date": "2017-09-21", "type": "ACTUAL" }, + "primaryCompletionDateStruct": { + "date": "2030-12-02", + "type": "ESTIMATED" + }, + "completionDateStruct": { "date": "2030-12-02", "type": "ESTIMATED" }, + "studyFirstSubmitDate": "2018-01-30", + "studyFirstSubmitQcDate": "2018-02-02", + "studyFirstPostDateStruct": { + "date": "2018-02-05", + "type": "ACTUAL" + }, + "lastUpdateSubmitDate": "2024-02-27", + "lastUpdatePostDateStruct": { "date": "2024-02-29", "type": "ACTUAL" } + }, + "sponsorCollaboratorsModule": { + "responsibleParty": { "type": "SPONSOR" }, + "leadSponsor": { + "name": "Novartis Gene Therapies", + "class": "INDUSTRY" + } + }, + "oversightModule": { + "oversightHasDmc": true, + "isFdaRegulatedDrug": true, + "isFdaRegulatedDevice": false + }, + "descriptionModule": { + "briefSummary": "This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi. Patients will roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years.", + "detailedDescription": "This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi. Patients will roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years. The last visit of the parent study or early discontinuation from the parent study may serve as the visit at which the informed consent form process is conducted for the AVXS 101-LT-001 long term follow-up safety study. Patients will return annually for follow up study visits for five (5) years, and then will be contacted via phone annually for ten (10) years. Additionally, patient record transfers from their local physician and/or neurologist will be requested in conjunction with the annual study visits and phone contacts for review by the investigator.\n\nIf the patient is unable to return to the original investigative site, the sponsor will arrange with the patients' local established physician to serve as an additional investigator to conduct the required assessments.\n\nThis clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.)." + }, + "conditionsModule": { "conditions": ["Spinal Muscular Atrophy 1"] }, + "designModule": { + "studyType": "OBSERVATIONAL", + "patientRegistry": false, + "designInfo": { + "observationalModel": "OTHER", + "timePerspective": "PROSPECTIVE" + }, + "enrollmentInfo": { "count": 13, "type": "ACTUAL" } + }, + "armsInterventionsModule": { + "interventions": [ + { + "type": "BIOLOGICAL", + "name": "Onasemnogene Abeparvovec-xioi", + "description": "Patients received treatment with onasemnogene abeparvovec-xioi in the parent study, AVXS-101-CL-101", + "otherNames": ["Zolgensma"] + } + ] + }, + "outcomesModule": { + "primaryOutcomes": [ + { + "measure": "Long-Term Safety", + "description": "The primary objective is to collect long term safety data of patients with SMA Type 1 who were treated with onasemnogene abeparvovec-xioi in the AVXS-101-CL-101 gene replacement therapy clinical trial by assessing incidence of SAEs and Adverse Events of Special Interest", + "timeFrame": "15 years" + } + ] + }, + "eligibilityModule": { + "eligibilityCriteria": "Inclusion Criteria:\n\n1. Patient who received onasemnogene abeparvovec-xioi in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1.\n2. Parent/legal guardian willing and able to complete the informed consent process, comply with study procedures and visit schedule.\n\nExclusion Criteria:\n\n1. Parent/legal guardian unable or unwilling to participate in the long term follow up safety study.", + "healthyVolunteers": false, + "sex": "ALL", + "stdAges": ["CHILD", "ADULT", "OLDER_ADULT"], + "studyPopulation": "Patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi will roll over from the previous study into the AVXS-101-LT-001 study for continuous safety monitoring for up to 15 years.", + "samplingMethod": "NON_PROBABILITY_SAMPLE" + }, + "contactsLocationsModule": { + "locations": [ + { + "facility": "Nationwide Children's Hospital", + "city": "Columbus", + "state": "Ohio", + "zip": "43205", + "country": "United States", + "geoPoint": { "lat": 39.96118, "lon": -82.99879 } + } + ] + }, + "referencesModule": { + "references": [ + { + "pmid": "34383289", + "type": "DERIVED", + "citation": "Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Oct;44(10):1109-1119. doi: 10.1007/s40264-021-01107-6. Epub 2021 Aug 12. Erratum In: Drug Saf. 2022 Feb;45(2):191-192. doi: 10.1007/s40264-021-01130-7." + }, + { + "pmid": "33999158", + "type": "DERIVED", + "citation": "Mendell JR, Al-Zaidy SA, Lehman KJ, McColly M, Lowes LP, Alfano LN, Reash NF, Iammarino MA, Church KR, Kleyn A, Meriggioli MN, Shell R. Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy. JAMA Neurol. 2021 Jul 1;78(7):834-841. doi: 10.1001/jamaneurol.2021.1272." + } + ] + }, + "ipdSharingStatementModule": { "ipdSharing": "NO" } + }, + "derivedSection": { + "miscInfoModule": { "versionHolder": "2024-12-04" }, + "conditionBrowseModule": { + "meshes": [ + { "id": "D009133", "term": "Muscular Atrophy" }, + { "id": "D009134", "term": "Muscular Atrophy, Spinal" }, + { + "id": "D014897", + "term": "Spinal Muscular Atrophies of Childhood" + }, + { "id": "D001284", "term": "Atrophy" } + ], + "ancestors": [ + { "id": "D020763", "term": "Pathological Conditions, Anatomical" }, + { "id": "D020879", "term": "Neuromuscular Manifestations" }, + { "id": "D009461", "term": "Neurologic Manifestations" }, + { "id": "D009422", "term": "Nervous System Diseases" }, + { "id": "D013118", "term": "Spinal Cord Diseases" }, + { "id": "D002493", "term": "Central Nervous System Diseases" }, + { "id": "D016472", "term": "Motor Neuron Disease" }, + { "id": "D019636", "term": "Neurodegenerative Diseases" }, + { "id": "D009468", "term": "Neuromuscular Diseases" }, + { + "id": "D020271", + "term": "Heredodegenerative Disorders, Nervous System" + }, + { "id": "D030342", "term": "Genetic Diseases, Inborn" } + ], + "browseLeaves": [ + { + "id": "M4589", + "name": "Atrophy", + "asFound": "Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12090", + "name": "Muscular Atrophy", + "asFound": "Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M12091", + "name": "Muscular Atrophy, Spinal", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "M17637", + "name": "Spinal Muscular Atrophies of Childhood", + "asFound": "Spinal Muscular Atrophy 1", + "relevance": "HIGH" + }, + { + "id": "M22519", + "name": "Pathological Conditions, Anatomical", + "relevance": "LOW" + }, + { + "id": "M22619", + "name": "Neuromuscular Manifestations", + "relevance": "LOW" + }, + { + "id": "M12404", + "name": "Neurologic Manifestations", + "relevance": "LOW" + }, + { + "id": "M15915", + "name": "Spinal Cord Diseases", + "relevance": "LOW" + }, + { + "id": "M5742", + "name": "Central Nervous System Diseases", + "relevance": "LOW" + }, + { + "id": "M18879", + "name": "Motor Neuron Disease", + "relevance": "LOW" + }, + { + "id": "M4024", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + }, + { + "id": "M21558", + "name": "Neurodegenerative Diseases", + "relevance": "LOW" + }, + { + "id": "M12411", + "name": "Neuromuscular Diseases", + "relevance": "LOW" + }, + { + "id": "M22092", + "name": "Heredodegenerative Disorders, Nervous System", + "relevance": "LOW" + }, + { + "id": "M23686", + "name": "Genetic Diseases, Inborn", + "relevance": "LOW" + }, + { + "id": "T5342", + "name": "Spinal Muscular Atrophy", + "asFound": "Spinal Muscular Atrophy", + "relevance": "HIGH" + }, + { + "id": "T5343", + "name": "Spinal Muscular Atrophy 1", + "asFound": "Spinal Muscular Atrophy 1", + "relevance": "HIGH" + }, + { + "id": "T349", + "name": "Amyotrophic Lateral Sclerosis", + "relevance": "LOW" + } + ], + "browseBranches": [ + { "abbrev": "BC23", "name": "Symptoms and General Pathology" }, + { "abbrev": "All", "name": "All Conditions" }, + { "abbrev": "BC10", "name": "Nervous System Diseases" }, + { + "abbrev": "BC16", + "name": "Diseases and Abnormalities at or Before Birth" + }, + { "abbrev": "BC18", "name": "Nutritional and Metabolic Diseases" }, + { "abbrev": "Rare", "name": "Rare Diseases" } + ] + } + }, + "hasResults": false + } + ] +} diff --git a/tests/test_fetch_clinical_trials.py b/tests/test_fetch_clinical_trials.py index eece6a8..bf7896f 100644 --- a/tests/test_fetch_clinical_trials.py +++ b/tests/test_fetch_clinical_trials.py @@ -47,3 +47,16 @@ def test_fetch_clinical_trials(fixtures_dir: Path): assert example.derived.conditions[0] == MeshConcept( id="D009133", term="Muscular Atrophy" ) + + with ( + requests_mock.Mocker() as m, + ( + fixtures_dir / "fetch_clinical_trial_zolgensma_parsing_error.json" + ).open() as json_response, + ): + m.get( + "https://clinicaltrials.gov/api/v2/studies?query.intr=zolgensma", + text=json_response.read(), + ) + # check that parsing errors are skipped + results = get_clinical_trials(drug_name="zolgensma", skip_parsing_failures=True) From 6b1ff7693a4058c1cd113b508e1deb5dcefc0ea1 Mon Sep 17 00:00:00 2001 From: James Stevenson Date: Mon, 16 Dec 2024 09:09:53 -0500 Subject: [PATCH 2/2] add docstring --- src/regbot/fetch/clinical_trials.py | 8 +++++++- 1 file changed, 7 insertions(+), 1 deletion(-) diff --git a/src/regbot/fetch/clinical_trials.py b/src/regbot/fetch/clinical_trials.py index bf0114e..81ae87a 100644 --- a/src/regbot/fetch/clinical_trials.py +++ b/src/regbot/fetch/clinical_trials.py @@ -889,6 +889,13 @@ def _format_study(study_input: dict) -> Study: def _get_id(study_response: dict, url: str, i: int) -> str | None: + """Extract NCT ID from study response + + :param study_response: a single study response object + :param url: URL used to issue request + :param i: index of individual study within response to that URL + :return: NCT ID if available (should be, but we're being careful) + """ study_id = ( study_response.get("protocolSection", {}) .get("identificationModule", {}) @@ -900,7 +907,6 @@ def _get_id(study_response: dict, url: str, i: int) -> str | None: i, url, ) - study_id = "" return study_id