Ignite Creation Date:
2025-12-24 @ 11:53 PM
Ignite Modification Date:
2025-12-25 @ 9:48 PM
Study NCT ID:
NCT05967351
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2025-01-17
First Post:
2023-07-21
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020388', 'term': 'Muscular Dystrophy, Duchenne'}], 'ancestors': [{'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'OTHER', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 400}}, 'statusModule': {'overallStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2023-09-27', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-01', 'completionDateStruct': {'date': '2030-11-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-01-15', 'studyFirstSubmitDate': '2023-07-21', 'studyFirstSubmitQcDate': '2023-07-21', 'lastUpdatePostDateStruct': {'date': '2025-01-17', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-08-01', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2030-11-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)', 'timeFrame': 'Up to 5 years after dosing'}], 'secondaryOutcomes': [{'measure': 'Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec', 'timeFrame': 'Baseline, 5 years'}, {'measure': 'Change in Time to Rise From Floor From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec', 'timeFrame': 'Baseline, 5 years'}, {'measure': 'Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec', 'timeFrame': 'Baseline, 5 years'}, {'measure': 'Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec', 'timeFrame': 'Baseline, 5 years'}, {'measure': 'Change in PUL (Version 2.0) Domain Specific Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec', 'timeFrame': 'Baseline, 5 years'}, {'measure': 'Change in Forced Vital Capacity Percent (FVC%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec', 'timeFrame': 'Baseline, 5 years'}, {'measure': 'Change in Peak Expiratory Flow Percent (PEF%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec', 'timeFrame': 'Baseline, 5 years'}, {'measure': 'Change in Cardiac Magnetic Resonance Imaging (MRI) Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec', 'timeFrame': 'Baseline, 5 years'}, {'measure': 'Change in Musculoskeletal MRI Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec', 'timeFrame': 'Baseline, 5 years'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Duchenne Muscular Dystrophy', 'DMD', 'Pediatric', 'Long-term', 'Follow-up', 'Safety', 'Efficacy'], 'conditions': ['Duchenne Muscular Dystrophy']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study. No study drug will be administered as part of this study. Pre-infusion baseline will be defined as the timepoint just prior to infusion of delandistrogene moxeparvovec from a previous clinical study. Each participant will be followed for a minimum of 5 years post-infusion of delandistrogene moxeparvovec from a previous clinical study. The duration of participation in this study is dependent on the length of follow-up the participant completed post-infusion of delandistrogene moxeparvovec from a previous clinical study.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Received delandistrogene moxeparvovec for Duchenne muscular dystrophy in a previous clinical study.\n* Has (a) parent(s) or legal caregiver(s) or is ≥18 years of age and able to understand and comply with the study visit schedule and all other protocol requirements.\n\nExclusion Criteria:\n\n* Participant or family does not want to disclose participation with general practitioner/primary care physician and other medical providers.\n\nOther inclusion/exclusion criteria may apply.'}, 'identificationModule': {'nctId': 'NCT05967351', 'acronym': 'EXPEDITION', 'briefTitle': 'A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study', 'organization': {'class': 'INDUSTRY', 'fullName': 'Sarepta Therapeutics, Inc.'}, 'officialTitle': 'A Phase 3, Multinational, Long-term Follow-up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study', 'orgStudyIdInfo': {'id': 'SRP-9001-305'}, 'secondaryIdInfos': [{'id': '2023-505043-39-00', 'type': 'OTHER', 'domain': 'Clinical Trials Information System (CTIS)'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Delandistrogene Moxeparvovec', 'description': 'Participant received delandistrogene moxeparvovec in a previous clinical study.', 'interventionNames': ['Genetic: delandistrogene moxeparvovec']}], 'interventions': [{'name': 'delandistrogene moxeparvovec', 'type': 'GENETIC', 'otherNames': ['SRP-9001', 'delandistrogene moxeparvovec-rokl', 'ELEVIDYS'], 'description': 'No study drug will be administered as part of this study. Eligible participants who received treatment with delandistrogene moxeparvovec during a previous clinical study will be included.', 'armGroupLabels': ['Delandistrogene Moxeparvovec']}]}, 'contactsLocationsModule': {'locations': [{'zip': '72202', 'city': 'Little Rock', 'state': 'Arkansas', 'country': 'United States', 'facility': "Arkansas Children's Hospital", 'geoPoint': {'lat': 34.74648, 'lon': -92.28959}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'UCLA Medical Center', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'facility': "Lucile Packard Children's Hospital Stanford (LPCH)", 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '95817', 'city': 'Sacramento', 'state': 'California', 'country': 'United States', 'facility': 'University of California, Davis', 'geoPoint': {'lat': 38.58157, 'lon': -121.4944}}, {'zip': '92123', 'city': 'San Diego', 'state': 'California', 'country': 'United States', 'facility': "Rady Children's Hospital", 'geoPoint': {'lat': 32.71571, 'lon': -117.16472}}, {'zip': '80045-7106', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital of Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '32610', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Florida Clinical Research Center', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "Ann & Robert H. Lurie Children's Hospital of Chicago", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '52242', 'city': 'Iowa City', 'state': 'Iowa', 'country': 'United States', 'facility': 'University of Iowa ICTS Clinical Research Unit', 'geoPoint': {'lat': 41.66113, 'lon': -91.53017}}, {'zip': '21287', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'The Johns Hopkins Hospital, Rubenstein Child Health Bldg', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Boston Children's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University School of Medicine', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Columbia University Pediatric Neuromuscular Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '14642', 'city': 'Rochester', 'state': 'New York', 'country': 'United States', 'facility': 'University of Rochester Medical Center', 'geoPoint': {'lat': 43.15478, 'lon': -77.61556}}, {'zip': '27705', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': "Lenox Baker Children's Hospital (Duke University)", 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '43205', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': "Nationwide Children's Hospital", 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '97239', 'city': 'Portland', 'state': 'Oregon', 'country': 'United States', 'facility': 'Oregon Health and Science University', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Chiildren's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '75207', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': "Children's Health Specialty Center", 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '84132', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'University of Utah Health', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '23510', 'city': 'Norfolk', 'state': 'Virginia', 'country': 'United States', 'facility': "Children's Hospital of The King's Daughters", 'geoPoint': {'lat': 36.84681, 'lon': -76.28522}}, {'zip': '53226', 'city': 'Milwaukee', 'state': 'Wisconsin', 'country': 'United States', 'facility': "Children's Hospital Wisconsin", 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}, {'zip': '9000', 'city': 'Ghent', 'state': 'East Flanders', 'country': 'Belgium', 'facility': 'Universitair Ziekenhuis Gent, Neuromuscular Reference Centre (NMRC)', 'geoPoint': {'lat': 51.05, 'lon': 3.71667}}, {'zip': '45147', 'city': 'Essen', 'state': 'North Rhine-Westphalia', 'country': 'Germany', 'facility': 'Universitätsklinikum Essen - Klinik für Kinderheilkunde I', 'geoPoint': {'lat': 51.45657, 'lon': 7.01228}}, {'city': 'Hong Kong', 'country': 'Hong Kong', 'facility': "Hong Kong Children's Hospital", 'geoPoint': {'lat': 22.27832, 'lon': 114.17469}}, {'zip': '16147', 'city': 'Genova', 'country': 'Italy', 'facility': 'UOC Neurologia Pediatrica e Malattie Muscolari, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico', 'geoPoint': {'lat': 45.21604, 'lon': 11.87211}}, {'zip': '20122', 'city': 'Milan', 'country': 'Italy', 'facility': "UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico", 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'zip': '00168', 'city': 'Roma', 'country': 'Italy', 'facility': 'UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS, Universita Cattoclica del Sacro Cuore', 'geoPoint': {'lat': 44.99364, 'lon': 11.10642}}, {'zip': '650-0017', 'city': 'Kobe', 'state': 'Hyōgo', 'country': 'Japan', 'facility': 'Kobe University Hospital', 'geoPoint': {'lat': 34.6913, 'lon': 135.183}}, {'zip': '187-8551', 'city': 'Kodaira', 'state': 'Tokyo', 'country': 'Japan', 'facility': 'National Center of Neurology and Psychiatry', 'geoPoint': {'lat': 35.72603, 'lon': 139.48508}}, {'zip': '08950', 'city': 'Esplugues de Llobregat', 'state': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Sant Joan de Deu', 'geoPoint': {'lat': 41.37732, 'lon': 2.08809}}, {'zip': '46026', 'city': 'Valencia', 'country': 'Spain', 'facility': 'Hospital Universitario y Politecnico La fe. Neurology Department', 'geoPoint': {'lat': 39.47391, 'lon': -0.37966}}, {'zip': '807', 'city': 'Kaohsiung City', 'country': 'Taiwan', 'facility': 'Kaohsiung Medical University Chung-Ho Memorial Hospital', 'geoPoint': {'lat': 22.61626, 'lon': 120.31333}}, {'zip': '100225', 'city': 'Taipei', 'country': 'Taiwan', 'facility': 'National Taiwan University Hospital', 'geoPoint': {'lat': 25.05306, 'lon': 121.52639}}, {'zip': 'WC1N 1EJ', 'city': 'London', 'state': 'Greater London', 'country': 'United Kingdom', 'facility': 'Great Ormond Street Hospital For Children NHS Foundation Trust', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'OX3 9DU', 'city': 'Oxford', 'state': 'Oxfordshire', 'country': 'United Kingdom', 'facility': 'Oxford University Hospitals NHS Foundation Trust', 'geoPoint': {'lat': 51.75222, 'lon': -1.25596}}, {'zip': 'NE1 3BZ', 'city': 'Newcastle upon Tyne', 'country': 'United Kingdom', 'facility': 'Institute of Translational and Clinical Research', 'geoPoint': {'lat': 54.97328, 'lon': -1.61396}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Sarepta Therapeutics, Inc.', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Hoffmann-La Roche', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}