Tests BBM-D101 gene therapy in children with Duchenne muscular dystrophy
Tests one intravenous dose of BBM-D101 in children with Duchenne muscular dystrophy.
Plain-language title generated from the registry record
Registry title: A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.
Key facts
- Where
- Beijing, ChinaSee the site
- Phase
- Phase 1/2
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for boys aged 4 to under 9 with Duchenne muscular dystrophy who can still walk. They must have a confirmed change in the DMD gene.
- Age
- 4 years and above but under 9 years
- Sex
- Male only
- Disease stage
- Duchenne muscular dystrophy; the boy must still be able to walk (ambulatory).
- Earlier treatment
- Must be on a stable, standard dose of glucocorticoids before screening. Cannot have had gene therapy or cell therapy, such as stem cell transplantation.
- Biomarker or test result
- Any change (mutation) in the DMD gene confirmed by genetic testing. Serum creatine kinase (CK) and AAV capsid antibody results must meet the study requirements.
Main reasons for not taking part
- Positive for hepatitis B, hepatitis C, HIV or Treponema pallidum antibodies, or on antiviral treatment for these
- Left ventricular ejection fraction (LVEF) below 50%, or NYHA heart function class III or higher
- Poorly controlled asthma, or a clear decline in lung function
- Liver disease such as portal hypertension or liver fibrosis stage 3 or higher
- Severe infection within 4 weeks before the treatment visit
Read by machine on 9 November 2026.
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 4 and 9 years old?
Source: NCT07058662, who can take part
Where in China
- Peking Union Medical College HospitalBeijing, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Belief BioMed (Beijing) Co., Ltd
- Trial code
- NCT07058662
- Record updated
- 4 December 2025
- Original description
The purpose of the study is to evaluate the safety, tolerability, and efficacy of BBM-D101 to treat participants with Duchenne Muscular Dystrophy.Read full original descriptionShow less
A Phase 1/2, Open-label Clinical Study to Evaluate the Safety, Tolerability and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.
The purpose of the study is to evaluate the safety, tolerability, and efficacy of BBM-D101 to treat participants with Duchenne Muscular Dystrophy.
Inclusion: 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.
Exclusion: 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); 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; Left ventricular ejection fraction (LVEF) < 50% or New York Heart Association (NYHA) cardiac function class ≥ III; 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.