NCT07058662

A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.

A Phase 1/2, Open-label Clinical Study to Evaluate the Safety, Tolerability and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: Belief BioMed (Beijing) Co., Ltd · Industry
• Sex: Male
• Age: 4 Years – 9 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the study is to evaluate the safety, tolerability, and efficacy of BBM-D101 to treat participants with Duchenne Muscular Dystrophy.

Detailed description

This is a single-arm, open-label study to evaluate the safety, tolerability, efficacy, pharmacokinetic, pharmacodynamic, and immune response of BBM-D101 within 52 weeks after a single intravenous infusion in DMD boys, as well as the long-term safety and efficacy of BBM-D101 for up to 5 years post infusion. BBM-D101 is a gene addition therapy based on engineered AAV delivery therapeutic protein gene cassette into muscle for treating DMD. Therapeutic protein could mediate the dystrophin-associated protein complex to prevent muscular dystrophy and to rescue the function of muscle.

Conditions

• DMD

Interventions

Type	Name	Description
GENETIC	Single dose intravenous of BBM-D101	BBM-D101 is a gene addition therapy based on engineered AAV delivery therapeutic protein gene cassette into muscle for treating DMD. Therapeutic protein could mediate the dystrophin-associated protein complex to prevent muscular dystrophy and to rescue the function of muscle.The administration is completed by a single intravenous infusion.

Timeline

• Start date: 2025-07-31
• Primary completion: 2027-06-30
• Completion: 2031-06-30
• First posted: 2025-07-10
• Last updated: 2025-12-04

Locations

1 site across 1 country: China

Regulatory

• FDA-regulated drug study