Lead Sponsor

Sarepta Therapeutics, Inc.

Age

4 - 7 years

Sex

MALE

Healthy Volunteers

No

•Established clinical diagnosis of DMD and documented dystrophin gene mutation of DMD phenotype.
•Indication of symptomatic muscular dystrophy by protocol-specified criteria.
•Ability to cooperate with motor assessment testing.
•Stable dose equivalent of oral corticosteroids for at least 12 weeks.
•A frameshift mutation contained between exons 18 and 58 (inclusive).

•Impaired cardiovascular function on echocardiogram.
•Prior or ongoing medical condition on physical examination, electrocardiogram, or laboratory findings that could adversely affect participant safety, compromise completion of follow-up, or impair assessment of study results.
•Exposure to another investigational drug or exon skipping medication within 6 months of screening.
•Exposure to an investigational or commercial gene therapy product.
•Abnormal liver or renal function by protocol-specified criteria.

Related studies

NCT05066633 · Muscular Dystrophy, Duchenne

NCT03992430 · Muscular Dystrophy, Duchenne

NCT07250737 · Muscular Disorders, Atrophic, Muscular Diseases, and more

NCT05429372 · Muscular Dystrophy, Duchenne

NCT03406780 · Muscular Dystrophies, Muscular Dystrophy, Duchenne, and more

A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)