Evaluation of the Safety and Efficacy of BBM-D101 Gene Therapy to Treat Patients with Duchenne Muscular Dystrophy

Actively RecruitingEarly Phase 1Gene Therapy

The legal guardian of the subject fully understands the purpose, nature, methods, and possible risks of the study, and signs a written informed consent form;
The study includes ambulatory male subjects who are at least 4 years old and less than 8 years old (4 years old ≤ age < 8 years old) ;
Have at least 1 of the following typical clinical signs or laboratory abnormalities of DMD: proximal muscle weakness, waddling gait, pseudo gastrocnemius hypertrophy, Gower's sign, pterygoid scapula;
Ability to cooperate with motor assessment testing, magnetic resonance imaging (MRI) and muscle biopsy according to the requirements of the study.

Hepatitis B surface antigen (HBsAg) positive, hepatitis B virus deoxyribonucleic acid (HBV-DNA) ≥1000U/mL, hepatitis C virus ribonucleic acid (HCV-RNA) positive or human immunodeficiency virus (HIV) positive;
Receiving antiviral therapy for hepatitis B, hepatitis C, HIV, etc.;
Left ventricular ejection fraction (LVEF) <50% or ≥ class III cardiac function defined by New York Heart Association (NYHA);
With severe or persistent arrhythmias and congenital heart disease.
The subject's preventive treatment/cardiomyopathy treatment changes within 1 month before the start of the study treatment;
With underlying liver disease, such as previous diagnosis of portal hypertension, splenomegaly, hepatic encephalopathy, or hepatic fibrosis ≥ stage 3; or nodules, cysts found by B-ultrasound in the past, or elevated alpha-fetoprotein in laboratory tests during the screening period, etc., and these abnormalities are judged by the investigator to be clinically significant;

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NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

Study Requirements and Criteria