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.
Single group
Single dose intravenous of BBM-D101
GeneticBBM-D101 is a gene addition therapy based on engineered AAV delivery therapeutic protein gene cassette into muscle for treating DMD. Therapeutic protein could mediate the dystrophin-associated protein complex to prevent muscular dystrophy and to rescue the function of muscle.The administration is completed by a single intravenous infusion.
Incidence of dose limiting toxicity (DLT) events
To evaluate the rate of incidence of DLT events determined by the Safety Data Review Committee (SRC) in DLT observation period after BBM-D101 infusion.
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
To evaluate the safety of BBM-D101 by AEs and SAEs.
Changes from baseline in BBM-D101 therapeutic protein level of muscle biopsy samples
To assess changes of therapatic protein from baseline in muscle after BBM-D101 Infusion.
Changes from baseline in serum Creatine Kinase (CK) level
To assess changes of serum CK after BBM-D101 Infusion.
Changes from baseline in the time to ascend time to rise (TTR) without assistance
To assess changes in TTR from baseline within 52 weeks after BBM-D101 infusion.
Changes from baseline in the time to ascend 10-meter walk/run test (10MWR) without assistance
To assess changes in 10MWR from baseline within 52 weeks after BBM-D101 infusion.
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Belief BioMed (Beijing) Co., Ltd
Lead sponsor
Shanghai Mianyi Biopharmaceutical Co., Ltd.
Collaborator
Shanghai Xinzhi BioMed Co., Ltd.
Collaborator
Belief BioMed Limited
Collaborator
