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
Condition(s) studied
Investigational drug(s) / intervention(s)
delandistrogene moxeparvovec: Single IV infusion of delandistrogene moxeparvovec
Study summary
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.
Eligibility
Primary outcome measure(s)
- Part 1 (Cohorts 1 to 5): Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot — Baseline, Week 12
- Part 1 (Cohorts 6 to 8): Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot — Week 12
- Cohort 8: Number of Participants with Acute Liver Injury (ALI) — Baseline up to Week 72
Trial sites (12)
| Facility | City | Region | Status |
|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | Recruiting |
| University of California, San Diego | La Jolla | California | Recruiting |
| University of California, Los Angeles | Los Angeles | California | Recruiting |
| Stanford University | Palo Alto | California | Recruiting |
| University of California, Davis | Sacramento | California | Recruiting |
| University of Florida | Gainesville | Florida | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | Recruiting |
| Washington University in St. Louis | St Louis | Missouri | Recruiting |
| Duke University Medical Center | Durham | North Carolina | Recruiting |
| Nationwide Children's Hospital | Columbus | Ohio | Active Not Recruiting |
| Neurology Rare Disease Center | Flower Mound | Texas | Recruiting |
| Children's Hospital of The King's Daughters | Norfolk | Virginia | Recruiting |
More Sarepta Therapeutics, Inc. trials in the USA
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT04626674 on ClinicalTrials.gov ↗ ← All trials in the USA