Study Summary
This phase 1 study will evaluate a single intravenous infusion of BBM-L101, an investigational AAV-based gene therapy designed to deliver a functional SGCA gene, in ambulatory children aged 4 to \<13 years with genetically confirmed SGCA-related limb-girdle muscular dystrophy (LGMD2D/R3). The primary objective is to assess safety and tolerability. The study will also explore muscle protein expression, serum creatine kinase, motor function, biodistribution/vector shedding, and immune responses. Participants will be followed for 52 weeks in the main study and through 5 years after infusion for long-term safety and efficacy.
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BBM-L101 InjectionGENETIC
Investigational recombinant adeno-associated virus gene therapy comprising the engineered N500 capsid carrying the shCK.SGCA-GLOB.hpolyA expression cassette. Administered once by intravenous infusion under medical monitoring. Cohort 1: 8.0 x 10\^13 vg/kg. Cohort 2: dose selected by the SRC according to protocol-defined dose-exploration rules.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Peking University First Hospital | Beijing | Beijing Municipality | China |