Safety and Effectiveness Study of BBM-D101 Gene Therapy in Boys with Duchenne Muscular Dystrophy Aged 4 to 8 Years

What this Trial Is About

Researchers are evaluating BBM-D101, a gene addition therapy, to treat boys diagnosed with Duchenne Muscular Dystrophy DMD. This early phase 1 study aims to assess the safety, tolerability, and effectiveness of BBM-D101 by delivering a therapeutic protein gene to muscles to help prevent muscular dystrophy and improve muscle function. The study is sponsored by Shanghai Jiao Tong University School of Medicine and focuses on DMD boys aged 4 to under 8 years old. Participants will receive a single intravenous infusion of BBM-D101. The study includes a 52-week period after infusion to evaluate safety, tolerability, efficacy, pharmacokinetics, pharmacodynamics, and immune response. After this initial period, long-term safety and efficacy will be monitored for up to 5 years. The treatment involves a recombinant adeno-associated virus vector designed to deliver the therapeutic protein gene into muscle tissue. During the study, boys will undergo various assessments including motor tests like the North Star Ambulatory Assessment and timed walking or climbing tasks. Muscle biopsies will be performed to measure BBM-D101 gene copies and therapeutic protein levels. Researchers will monitor adverse events and serious adverse events over 52 weeks and dose limiting toxicity within 12 weeks. Participants progress and safety will be followed closely, with the total observation lasting up to 5 years post infusion.

Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy