Actively Recruiting
A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.
Led by Belief BioMed (Beijing) Co., Ltd · Updated on 2025-12-04
9
Participants Needed
1
Research Sites
308 weeks
Total Duration
On this page
Sponsors
B
Belief BioMed (Beijing) Co., Ltd
Lead Sponsor
S
Shanghai Mianyi Biopharmaceutical Co., Ltd.
Collaborating Sponsor
AI-Summary
What this Trial Is About
The purpose of the study is to evaluate the safety, tolerability, and efficacy of BBM-D101 to treat participants with Duchenne Muscular Dystrophy.
CONDITIONS
Official Title
A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- The participant and/or legal guardian must understand the study purpose, methods, risks, and sign informed consent.
- Ambulatory male children aged 4 years and above but under 9 years.
- Confirmed mutation in the DMD gene by genetic testing.
- Serum creatine kinase levels meet study requirements during screening.
- Receiving stable, standard-dose glucocorticoids before screening.
- AAV capsid antibody levels meet clinical trial requirements.
- Able to cooperate with motor function assessment, MRI, and muscle biopsy.
- Laboratory test results meet required standards at screening and baseline.
- Willing to actively cooperate, comply with protocol, and attend all scheduled visits.
You will not qualify if you...
- Positive for hepatitis B surface antigen, HBV-DNA ≥ 1000 U/mL, hepatitis C virus RNA positive, HIV positive, or Treponema pallidum antibodies.
- Currently receiving antiviral therapy for hepatitis B, hepatitis C, or HIV.
- Severe behavioral or cognitive disorders that hinder study participation.
- Poorly controlled asthma, significant lung function decline from DMD, or recurrent infectious pneumonia affecting respiratory function.
- Left ventricular ejection fraction less than 50% or NYHA cardiac function class III or higher.
- Severe or persistent arrhythmias or congenital heart disease unsuitable for study participation.
- Changes in preventive or cardiomyopathy treatment within one month before study drug infusion.
- History of liver diseases such as portal hypertension, splenomegaly, hepatic encephalopathy, advanced liver fibrosis, hepatic nodules or cysts, or clinically significant elevated alpha-fetoprotein.
- Severe infection within four weeks before treatment visit.
- History of gene therapy or cell therapy such as stem cell transplantation.
- History or current autoimmune diseases, severe renal, gastrointestinal, neurological, coagulation disorders, malignant tumors, or other significant diseases.
- Other conditions deemed unsuitable for participation by the investigator.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China, 100730
Actively Recruiting
Research Team
H
Hanyang Hu, Ph.D
CONTACT
D
DMD Clinical Trial Team
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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