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)
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.
• 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 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: Non-ambulatory per protocol specified criteria at the time of Screening.
• Cohort 6: Is ambulatory, and ≥2 to \<3 years of age at the time of Screening.
• Cohort 7: Non-ambulatory per protocol-specified criteria at the time of Screening.
• Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥3 at the Screening visit and has a total PUL score of ≥20 and ≤40 at the time of Screening.
• Ability to cooperate with motor assessment testing.
• 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.
• Cohorts 4 and 6: Do not yet require use of chronic steroids for treatment of their DMD, in the opinion of the Investigator, and are not receiving steroids at the time of Screening.
• rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
• Genetic mutation inclusion criteria vary by cohort.