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

Sponsor
Belief BioMed (Beijing) Co., Ltd
Study ID
NCT07058662
Phase
PHASE1/PHASE2
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
MALE
Age
4 Years - 9 Years
Healthy Volunteers
Not accepted

Interventions

  • Single dose intravenous of BBM-D101 — GENETIC
    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.

Study Details

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

Key Dates

Start date
Jul 31, 2025
Status verified
Nov 2025
Primary completion
Jun 30, 2027
Completion
Jun 30, 2031

Study Design

Enrollment
9 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Arm of BBM-D101
    Single-dose treatment

Primary Outcome Measure

Incidence of dose limiting toxicity (DLT) events [ Time Frame: Within 12 weeks ]

Central Contacts

Related Studies