A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Completed · Phase 3 · NCT05096221
Conditions: Duchenne Muscular Dystrophy
At a glance
- Research payments reported, 2025
- $662,570
- U.S. investigators named
- 21
- Sites paid
- 27
- Planned or actual enrollment
- 126
About the trial
- Sponsor
- Sarepta Therapeutics, Inc. (with Hoffmann-La Roche)
- Tested
- delandistrogene moxeparvovec (genetic), placebo (genetic)
- Dates
- Started 2021-10-27; completion 2024-10-25
- Results posted
- Yes
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.
From ClinicalTrials.gov, last updated 2025-07-08. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Sarepta Therapeutics, Inc. reported $662,570 in research payments for this study in 2025 (331 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
- Perry B. Shieh, MD Neurology · Los Angeles, CA
- Susan Apkon, MD Physical Medicine & Rehabilitation (PM&R) · Aurora, CO
- Carmen E. Leon Astudillo, M.D. Pediatric Pulmonology · Gainesville, FL
- Katherine D. Mathews, MD Physical Medicine & Rehabilitation (PM&R) · Iowa City, IA
- Nancy L. Kuntz, M.D. Child Neurology · Chicago, IL
- Jessica Nance, MD Child Neurology · Baltimore, MD
- Partha S. Ghosh, MD Neurology · Rochester, MN
- Craig M. Zaidman, MD Child Neurology · Saint Louis, MO
- Mai K. El Mallah, MD Pediatric Pulmonology · Durham, NC
- Emma Ciafaloni, MD Neurology · Rochester, NY
- Michio Hirano, M.D. Neurology · New York, NY
- Anne M. Connolly, MD Child Neurology · Columbus, OH
- Erika L. Finanger, M.D. Child Neurology · Portland, OR
- Amy Waldman, MD Child Neurology · Philadelphia, PA
- Susan T. Iannaccone, MD Neurology · Dallas, TX
- Russell J. Butterfield, MD/PHD Child Neurology · Salt Lake City, UT
- Crystal M. Proud, M.D. Child Neurology · Norfolk, VA
- Nancy E. Bass, MD Child Neurology · Milwaukee, WI
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Strong Memorial Hospital, NY $132,175
- Children's Hospital Colorado, CO $98,161
- University of Utah Hospital and Clinics, UT $83,248
- Children's Hospital of Philadelphia, PA $45,571
- Duke University $44,694
- University of Florida $38,924
- Ronald Reagan UCLA Medical Center, CA $31,341
- Boston Children's Hospital, MA $24,369
- University of Iowa Hospital & Clinics, IA $22,160
- Childrens Hospital of Wisconsin $18,919
- Children's Specialty Group, PLLC $18,750
- Ohsu Hospital and Clinics, OR $16,978
- Johns Hopkins University School of Medicine $11,774
- Washington University $9,950
- The Research Institute at Nationwide Children's Hospital $9,866
- Washington University in St. Louis $9,513
- University of California San Diego Jacobs Medical Center $7,671
- The Trustees of Columbia University in The City of New York $7,155
- Ann & Robert H Lurie Childrens Hospital of Chicago, IL $7,111
- Arkansas Childrens Hospital Research Institute $5,400
- Arkansas Childrens Research $5,104
- University of California Davis Medical Center, CA $4,300
- The Regents of the University of California $3,511
- The University of Texas Southwestern Medical Center $2,449
- Medical College of Wisconsin $1,857
- Children's Research Institute $1,620
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.