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
← catalyst calendarNCT04626674 · readout in 498 d
Sponsored by Sarepta Therapeutics, Inc. (industry) · SRPT — their whole pipeline →. With Hoffmann-La Roche.
Phase
Phase 1
Status
Recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
83estimated
Sites
7
Country
United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2020-11-23 | actual | When the study began enrolling |
| Primary completion | 2027-12-31 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2028-02-29 | estimated | The whole study's end, after follow-up |
| First posted | 2020-11-12 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-06-24 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Duchenne Muscular Dystrophy
Intervention
- Genetic: delandistrogene moxeparvovec — also filed as SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS
Primary outcomes
what the primary-completion date above is the date OFPart 1 (Cohorts 1 to 5): Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot
measured Baseline, Week 12
Part 1 (Cohorts 6 to 8): Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
measured Week 12
Cohort 8: Number of Participants with Acute Liver Injury (ALI)
measured Baseline up to Week 72
Publications
- PMID 39747998 — linked by the registry
- PMID 37539981 — linked by the registry
Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.
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