A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Completed · Phase 1, Phase 2 · NCT03375164
Conditions: Duchenne Muscular Dystrophy
At a glance
- Research payments reported, 2025
- $29,175
- U.S. investigators named
- 0
- Sites paid
- 0
- Planned or actual enrollment
- 4
About the trial
- Sponsor
- Sarepta Therapeutics, Inc.
- Tested
- delandistrogene moxeparvovec (genetic)
- Dates
- Started 2018-01-04; completion 2023-04-25
- Results posted
- Yes
This study was an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD. This study was originally designed to consist of 12 patients across 2 Cohorts. Cohort A would have included participants ages 3 months to 3 years, and Cohort B included participants ages 4 to 7 years old. No participants were enrolled in Cohort A.
From ClinicalTrials.gov, last updated 2024-11-14. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Sarepta Therapeutics, Inc. reported $29,175 in research payments for this study in 2025 (13 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
Sites paid to run it
- $29,175
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.