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)
Active, not recruiting · Phase 3 · NCT05881408
Conditions: Duchenne Muscular Dystrophy
At a glance
- Research payments reported, 2025
- $2.4M
- U.S. investigators named
- 15
- Sites paid
- 19
- Planned or actual enrollment
- 148
About the trial
- Sponsor
- Sarepta Therapeutics, Inc. (with Hoffmann-La Roche)
- Tested
- delandistrogene moxeparvovec (genetic), placebo (genetic)
- Dates
- Started 2023-05-31; completion 2028-06-30
- Results posted
- Not yet
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.
From ClinicalTrials.gov, last updated 2026-05-22. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Sarepta Therapeutics, Inc. reported $2.4M in research payments for this study in 2025 (924 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.
U.S. investigators named on this study
- Aravindhan Veerapandiyan, MD Child Neurology · Little Rock, AR
- Chamindra G. Laverty, M.D. Neurology · San Diego, CA
- Craig M. Mcdonald, M.D. Physical Medicine & Rehabilitation (PM&R) · Sacramento, CA
- Ana C. Tesi Rocha, MD Physical Medicine & Rehabilitation (PM&R) · Palo Alto, CA
- Carmen E. Leon Astudillo, M.D. Pediatric Pulmonology · Gainesville, FL
- Shant J. Manoushagian, M.D. Cardiology · Fort Myers, FL
- Jessica Nance, MD Child Neurology · Baltimore, MD
- Mai K. El Mallah, MD Pediatric Pulmonology · Durham, NC
- Emma Ciafaloni, MD Neurology · Rochester, NY
- Erika L. Finanger, M.D. Child Neurology · Portland, OR
- John F. Brandsema, MD Child Neurology · Philadelphia, PA
- Amy Waldman, MD Child Neurology · Philadelphia, PA
- Susan T. Iannaccone, MD Neurology · Dallas, TX
- Crystal M. Proud, M.D. Child Neurology · Norfolk, VA
- Matthew Harmelink, M.D. Child Neurology · Milwaukee, WI
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Children's Hospital of Philadelphia, PA $1.1M
- Children's Specialty Group, PLLC $245,658
- Strong Memorial Hospital, NY $211,259
- University of California Davis Medical Center, CA $185,409
- University of Florida $180,955
- Arkansas Childrens Research $157,962
- The Board of Trustees of the Leland Standford Junior $131,990
- Duke University $86,077
- Johns Hopkins University School of Medicine $47,971
- Childrens Hospital of Wisconsin $46,210
- Ohsu Hospital and Clinics, OR $24,950
- University of California San Diego Jacobs Medical Center $23,822
- Arkansas Childrens Hospital Research Institute $17,795
- The Regents of the University of California $8,088
- Stanford University $7,571
- The University of Texas Southwestern Medical Center $1,950
- Gi Reviewers LLC $1,500
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.