Safety and Gene Expression Study of Delandistrogene Moxeparvovec in Male Children with Duchenne Muscular Dystrophy Non-Ambulatory Participants Receiving Single Intravenous Gene Therapy Infusion

What this Trial Is About

This research aims to evaluate the safety and gene expression of delandistrogene moxeparvovec, a gene transfer therapy, in males with Duchenne Muscular Dystrophy DMD. The study focuses on non-ambulatory participants in Cohort 8, while enrollment for earlier cohorts has been completed. The study is open-label and conducted by Sarepta Therapeutics, Inc., with a maximum participant duration of 156 weeks. Participants will receive a single intravenous infusion of delandistrogene moxeparvovec on Day 1. The study measures dystrophin protein expression at 12 weeks post-infusion, as well as safety outcomes including acute liver injury and other adverse events up to 72 weeks for Cohort 8. Additional assessments monitor vector shedding, antibody levels, treatment-emergent adverse events, and steroid use for up to 156 weeks. During the study, participants undergo various assessments including laboratory tests, biomarker evaluations, and motor function testing. Researchers will collect samples such as urine, saliva, and stool to track vector shedding and measure immune responses. Safety monitoring includes tracking liver-related events and infections. The total study participation may last up to about 3 years, allowing for long-term follow-up of treatment effects and safety.

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