A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
Condition(s) studied
Investigational drug(s) / intervention(s)
delandistrogene moxeparvovec: Single IV infusion of delandistrogene moxeparvovec
placebo: Single IV infusion of matching placebo
Study summary
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.
Eligibility
Primary outcome measure(s)
- Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72 — Baseline, Week 72
Trial sites (46)
| Facility | City | Region | Status |
|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | |
| Lucile Packard Children's Hospital Stanford | Palo Alto | California | |
| University of California at Davis Medical Center | Sacramento | California | |
| Rady Children's Hospital-San Diego | San Diego | California | |
| University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases | Gainesville | Florida | |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | |
| The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit | Baltimore | Maryland | |
| Boston Children's Hospital | Boston | Massachusetts | |
| Washington University of St. Louis, St. Louis Children's Hospital | St Louis | Missouri | |
| University of Rochester, Department of Neurology | Rochester | New York | |
| Lenox Baker Children's Hospital (Duke University) | Durham | North Carolina | |
| Nationwide Children's Hospital | Columbus | Ohio | |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | |
| The Children's Hospital at Westmead | Westmead | New South Wales | |
| The Royal Children's Hospital | Parkville | Victoria | |
| Universitair Ziekenhuis Gent | Ghent | Oost-Vlaanderen | |
| The Children's Hospital of Eastern Ontario | Ottawa | Ontario | |
| Research Institute McGill University Health Centre | Montreal | Quebec | |
| Centre Hospitalier Universitaire de Québec - Université Laval (pavillon Centre Hospitalier Universitaire Laval) | Québec | Canada | |
| LMU- Klinikum der Universitat Munchen, Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital, Abeteilung Neuropadiatrie, Campus Innenstadt | München | Bavaria | |
| Universitatsklinikum Essen, Klinik fur Kinderheilkunde I, Abteilung Neuropadiatrie Essen | Essen | North Rhine-Westphalia | |
| Universitatsklinikum Hamburg Eppendorf | Hamburg | Germany | |
| Hong Kong Children's Hospital | Hong Kong | Hong Kong | |
| Institute of Neruology, Schneider Children's Medical Center of Israel | Petah Tikva | Israel | |
| Tel Aviv Sourasky Medical Center | Tel Aviv | Israel | |
| U.O.S.D Centro Traslazionale di Miologia e Patologie Neurodegenerative, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico | Genova | Italy | |
| UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milan | Italy | |
| IRCCS Istituto Neurologico Carlo Besta Neurepsichiatria Infantile 2 - Epilettologia e Neurologia dello Sviluppo | Milan | Italy | |
| UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS | Roma | Italy | |
| National Hospital Organization Osaka Toneyama Medical Center | Toyonaka-shi | Osaka | |
| National Center of Neurology and Psychiatry | Kodaira | Tokyo | |
| Tokyo Women's Medical University Hospital | Shinjuku-ku | Tokyo | |
| Pusan National University Yangsan Hospital | Yangsan | Gyeongsangnam-do | |
| Seoul National University Hospital | Seoul | NAP | |
| Kyungpook National University Hospital | Daegu | South Korea | |
| Gangnam Severance Hospital, Yonsei University Health System | Seoul | South Korea | |
| Hospital Sant Joan de Deu | Esplugues de Llobregat | Barcelona | |
| Hospital Universitari Politecnic La Fe | Valencia | Spain | |
| Sahlgrenska Universitetssjukhuset | Gothenburg | Sweden |
+ 6 more sites — see the full list on the official registry below.
More Sarepta Therapeutics, Inc. trials in Sweden
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05881408 on ClinicalTrials.gov ↗ ← All trials in Sweden