A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.

Korábbi kezelés

• Currently receiving antiviral therapy for hepatitis B, hepatitis C, HIV, etc.
• Any changes in preventive/cardiomyopathy treatment (initiation of treatment, drug changes, dosing regimen changes, treatment interruption, termination, or restart) within 1 month before the infusion of the study drug.
• Severe infection (such as pneumonia, pyelonephritis, or meningitis) within 4 weeks before the treatment visit (enrollment may be postponed).

Más betegség

• 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.
• The investigator deems the subject has severe behavioral or cognitive disorders that may hinder participation in this study.
• Severe or persistent arrhythmias (such as atrial fibrillation, frequent ventricular premature beats, ventricular bigeminy, ventricular trigeminy, severe bundle branch block, etc.), and congenital heart disease that is evaluated by the investigator as unsuitable for participation in this study.
• History of liver diseases such as portal hypertension, splenomegaly, hepatic encephalopathy, liver fibrosis ≥ stage 3, or hepatic nodules/cysts found by ultrasound during screening, or elevated alpha-fetoprotein with clinical significance as determined by the investigator.
• History of gene therapy or cell therapy (such as stem cell transplantation).
• History of or current presence of autoimmune diseases, severe renal, gastrointestinal, neurological, or coagulation disorders, malignant tumors, or other diseases.
• Other diseases that the investigator deems unsuitable for participation in this study.

Laborértékek

• Left ventricular ejection fraction (LVEF) < 50% or New York Heart Association (NYHA) cardiac function class ≥ III.

Egyéb

• 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.

• 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.