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 (ENDEAVOR)
Actively RecruitingPhase 1Gene Therapy
Study Overview
- Age
- 2 years and older
- Phase
- Phase 1
- Sponsor
- Sarepta Therapeutics
- Therapeutic Approach
- Gene Therapy
- Variant Requirement
- For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- Eligible Sex
- Male
- Ambulation
- Ambulatory and Non-Ambulatory
- Countries
- Japan
Study Requirements and Criteria
Steroid Use
Cohorts 1, 2, 3, 5, 7 and 8 only: Stable dose equivalent of oral glucocorticoids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the first year of the study.
Inclusion Criteria
Male with DMD aged 4–16 years. Confirmed mutation amenable to exon 53 skipping. Able to cooperate with study procedures.
Exclusion Criteria
Prior exon 53 skipping therapy. Uncontrolled cardiac arrhythmia. Severe respiratory insufficiency requiring ventilation.
Clinical Trial Registry
NCT ID
NCT03933171This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.