Tests BBM-D101 treatment in children with Duchenne muscular dystrophy
Tests the BBM-D101 gene treatment in children aged 4 to 8 with Duchenne muscular dystrophy.
Plain-language title generated from the registry record
Registry title: Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy
Key facts
- Where
- Shanghai, ChinaSee the site
- Phase
- Early phase 1
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 8 with genetically confirmed Duchenne muscular dystrophy who can still walk.
- Age
- At least 4 years old and less than 8 years old
- Sex
- Male only
- Disease stage
- Duchenne muscular dystrophy (DMD) with a genetic diagnosis; the boy must be able to walk and have at least 1 typical sign, such as proximal muscle weakness, waddling gait, pseudo gastrocnemius hypertrophy, Gower's sign or pterygoid scapula.
- Earlier treatment
- Preventive or heart muscle treatment must not have changed within 1 month before the study treatment starts. Antiviral treatment for hepatitis B, hepatitis C or HIV is not allowed.
- Biomarker or test result
- Genetically confirmed diagnosis of DMD.
Main reasons for not taking part
- Hepatitis B surface antigen positive, HBV-DNA of 1000U/mL or more, HCV-RNA positive, or HIV positive
- Receiving antiviral treatment for hepatitis B, hepatitis C or HIV
- Left ventricular ejection fraction below 50% or heart function of NYHA class III or worse
- Severe or persistent heart rhythm problems, or congenital heart disease
- Underlying liver disease, such as portal hypertension, hepatic encephalopathy or hepatic fibrosis of stage 3 or more
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 8 years old?
Source: NCT06641895, who can take part
Where in China
- Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of MedicineShanghai, 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
- Shanghai Jiao Tong University School of Medicine
- Trial code
- NCT06641895
- Record updated
- 25 March 2025
- Original description
The purpose of the study is to assess the safety, tolerability, and efficacy of BBM-D101 to treat patients with Duchenne Muscular Dystrophy.Read full original descriptionShow less
A Single-Arm, Open-Label, Single-Dose Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 Injection in Patients with Duchenne Muscular Dystrophy
The purpose of the study is to assess the safety, tolerability, and efficacy of BBM-D101 to treat patients with Duchenne Muscular Dystrophy.
Inclusion: 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) ; Genetically confirmed diagnosis of DMD; 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.
Exclusion: Receiving antiviral therapy for hepatitis B, hepatitis C, HIV, etc; The subject\'s preventive treatment/cardiomyopathy treatment changes within 1 month before the start of the study treatment; 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; With severe or persistent arrhythmias and congenital heart disease; Left ventricular ejection fraction (LVEF) \<50% or ≥ class III cardiac function defined by New York Heart Association (NYHA); 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.