Status:
COMPLETED
A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)
Lead Sponsor:
Sarepta Therapeutics, Inc.
Conditions:
Muscular Dystrophy, Duchenne
Eligibility:
MALE
4-7 years
Phase:
PHASE1
PHASE2
Brief Summary
The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD participants by measuring biological and clinical endpoints in three parts: two 48-week randomized, d...
Eligibility Criteria
Inclusion
- 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).
Exclusion
- 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.
- Other inclusion/exclusion criteria apply.
Key Trial Info
Start Date :
December 5 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 16 2023
Estimated Enrollment :
41 Patients enrolled
Trial Details
Trial ID
NCT03769116
Start Date
December 5 2018
End Date
August 16 2023
Last Update
November 14 2024
Active Locations (2)
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1
David Geffen School of Medicine at UCLA
Los Angeles, California, United States, 90095
2
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205