An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects with Duchenne Muscular Dystrophy (ENDEAVOR)
Phase: Phase 1 - Active, Not Recruiting
Condition(s): Duchenne Muscular Dystrophy (DMD)
Keyword(s): delandistrogene moxeparvovec-rokl , ELEVIDYS
NCT Number: NCT04626674 Other Study ID Number(s): SRP-9001-103
Study Overview
To evaluate the safety and protein expression in the muscle from delandistrogene moxeparvovec (ELEVIDYS) infusion in participants with Duchenne muscular dystrophy
Sponsor: Sarepta Therapeutics
Participation Criteria
- Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- Aged 2-<18
- 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.
- rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
Study Plan
Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec (ELEVIDYS) and will follow with visits to Nationwide Children's Hospital for monitoring for three years.
Principal Investigator
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