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)
Sarepta Therapeutics, Inc.
Summary
This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with DMD. The maximum participant duration for this study is 156 weeks.
Description
Enrollment for Cohorts 1 through 7 has been completed. Cohort 8 is currently enrolling new participants.
Eligibility
- Age range
- 2+ years
- Sex
- Male
- Healthy volunteers
- No
Inclusion Criteria: * For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing. * Cohort 1: Is ambulatory, and ≥4 to \<8 years of age at the time of Screening. * Cohort 2: Is ambulatory, and ≥8 to \<18 years of age at the time of Screening. * Cohort 3: Non-ambulatory per protocol specified criteria at the time of Screening. * Cohort 4: Is ambulatory and ≥3 to \<4 years of age at the time of Screening. * Cohort 5a: Is ambulatory and ≥4 to \<9 years of age with time to rise from the floor ≤7 seconds at the screening visit. * Cohort 5b: N…
Interventions
- Geneticdelandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec
Locations (7)
- Arkansas Children's HospitalLittle Rock, Arkansas
- Stanford UniversityPalo Alto, California
- University of California, DavisSacramento, California
- Washington University in St. LouisSt Louis, Missouri
- Nationwide Children's HospitalColumbus, Ohio
- Neurology Rare Disease CenterFlower Mound, Texas