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BBM-L101 Gene Therapy in Participants With LGMD2D/R3

StatusNot Yet Recruiting
PhasePhase 1
Started
View on ClinicalTrials.gov ↗

Record History

  1. Study First Submitted
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.
Trial Details
NCT Number NCT07874516
Lead Sponsor Peking University First Hospital
Conditions
(from sponsor)		 Limb Girdle Muscular Dystrophy Type 2D/R3
Enrollment 8 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov