A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort
Recruiting · Phase 1 · NCT04626674
Conditions: Duchenne Muscular Dystrophy
At a glance
- Research payments reported, 2025
- $230,150
- U.S. investigators named
- 5
- Sites paid
- 8
- Planned or actual enrollment
- 83
About the trial
- Sponsor
- Sarepta Therapeutics, Inc. (with Hoffmann-La Roche)
- Tested
- delandistrogene moxeparvovec (genetic)
- Dates
- Started 2020-11-23; completion 2028-02-29
- Results posted
- Not yet
Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed. This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). The maximum participant duration for this study is 156 weeks.
From ClinicalTrials.gov, last updated 2026-09-11. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Sarepta Therapeutics, Inc. reported $230,150 in research payments for this study in 2025 (201 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
- John W. Day, MD Physical Medicine & Rehabilitation (PM&R) · Stanford, CA
- Craig M. Mcdonald, M.D. Physical Medicine & Rehabilitation (PM&R) · Sacramento, CA
- Craig M. Zaidman, MD Child Neurology · Saint Louis, MO
- Anne M. Connolly, MD Child Neurology · Columbus, OH
- Crystal M. Proud, M.D. Child Neurology · Norfolk, VA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- University of California Davis Medical Center, CA $97,242
- Washington University $76,912
- The Board of Trustees of the Leland Standford Junior $23,773
- Children's Research Institute $15,744
- Washington University in St. Louis $10,557
- Children's Specialty Group, PLLC $3,089
- The Research Institute at Nationwide Children's Hospital $2,127
- Stanford University $706
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.