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.
The purpose of the study is to evaluate the safety, tolerability, and efficacy of BBM-D101 to treat participants with Duchenne Muscular Dystrophy.
• The Participants and/or his legal guardian must fully understand the purpose, nature, methods, and potential risks of the study, and sign a written informed consent form.
• Ambulatory male subjects aged 4 years and above but under 9 years (4 years ≤ age \< 9 years).
• Any mutation in the DMD gene confirmed by genetic testing
• Serum creatine kinase (CK) during the screening period meets the study requirements.
• Receiving stable, standard-dose glucocorticoids before screening.
• The subject's AAV capsid antibodies meet the clinical trial requirements.
• Able to cooperate with motor function assessment, MRI, and muscle biopsy as required by the study.
• Laboratory test results during the screening period and at baseline meet the standards.
• The subject and/or his legal guardian must fully understand the study procedures, be willing to actively cooperate, commit to high compliance with the protocol, and ensure that the subject attends all scheduled visits.