U.S., Oct. 16 -- ClinicalTrials.gov registry received information related to the study (NCT07874516) titled 'BBM-L101 Gene Therapy in Participants With LGMD2D/R3' on Oct. 08.
Brief 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 ...