A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
NCT04626674 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 83
Last updated 2026-04-06
Summary
This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with DMD. The maximum participant duration for this study is 156 weeks.
Conditions
- Muscular Dystrophy, Duchenne
Interventions
- GENETIC
-
delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec
Sponsors & Collaborators
- collaborator INDUSTRY
-
Sarepta Therapeutics, Inc.
lead INDUSTRY
Principal Investigators
-
Medical Director · Sarepta Therapeutics, Inc.
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 2 Years
- Sex
- MALE
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2020-11-23
- Primary Completion
- 2027-12-31
- Completion
- 2028-02-29
- FDA Drug
- Yes
Countries
- United States
Study Locations
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