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.
Positive for hepatitis B surface antigen (HBsAg), hepatitis B virus deoxyribonucleic acid (HBV-DNA) ≥ 1000 U/mL, hepatitis C virus ribonucleic acid (HCV-RNA) positive, human immunodeficiency virus (HIV) positive, or positive for Treponema pallidum antibodies.
Currently receiving antiviral therapy for hepatitis B, hepatitis C, HIV, etc.
The investigator deems the subject has severe behavioral or cognitive disorders that may hinder participation in this study.
Poorly controlled asthma, or Duchenne Muscular Dystrophy (DMD) leading to significant decline in lung function, or recurrent infectious pneumonia that the investigator considers may affect respiratory function.
Belief BioMed (Beijing) Co., Ltd
Lead sponsor
Shanghai Mianyi Biopharmaceutical Co., Ltd.
Collaborator
Shanghai Xinzhi BioMed Co., Ltd.
Collaborator
Belief BioMed Limited
Collaborator