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)

Running, not enrolling · Phase 3 · Has a placebo group

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT05881408

Run by

Sarepta Therapeutics, Inc.

People needed

148

Starts

2023-05-31

Expected to finish

2028-06-30

Last updated by the study team

2026-05-22

Who can join

Age: any. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
• Cohort 1 only: Non-ambulatory per protocol-specified criteria.
• Cohort 2 only: Ambulatory per protocol-specified criteria and ≥8 to <18 years of age at the time of Screening.
• Ability to cooperate with motor assessment testing.
• Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
• Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) antibody titers are not elevated as per protocol-specified requirements.
• A pathogenic frameshift mutation or premature stop codon in the DMD gene, except for any deletion mutations in exon 8 and/or 9.

You may not qualify if…

• Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits.
• Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
• Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.
• Other inclusion or exclusion criteria could apply.

Where it is running

• Arkansas Children's Hospital — Little Rock, Arkansas, United States
• Lucile Packard Children's Hospital Stanford — Palo Alto, California, United States
• University of California at Davis Medical Center — Sacramento, California, United States
• Rady Children's Hospital-San Diego — San Diego, California, United States
• University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases — Gainesville, Florida, United States
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit — Baltimore, Maryland, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Washington University of St. Louis, St. Louis Children's Hospital — St Louis, Missouri, United States
• University of Rochester, Department of Neurology — Rochester, New York, United States
• Lenox Baker Children's Hospital (Duke University) — Durham, North Carolina, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Children's Hospital of the King's Daughters — Norfolk, Virginia, United States
• The Children's Hospital at Westmead — Westmead, New South Wales, Australia
• The Royal Children's Hospital — Parkville, Victoria, Australia
• Universitair Ziekenhuis Gent — Ghent, Oost-Vlaanderen, Belgium
• The Children's Hospital of Eastern Ontario — Ottawa, Ontario, Canada
• Research Institute McGill University Health Centre — Montreal, Quebec, Canada
• 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, Germany
• Universitatsklinikum Essen, Klinik fur Kinderheilkunde I, Abteilung Neuropadiatrie Essen — Essen, North Rhine-Westphalia, Germany
• 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

Full record on ClinicalTrials.gov

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