Evaluating BBM-D101 for treating Duchenne Muscular Dystrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study assesses the safety, tolerability, and efficacy of BBM-D101, a gene therapy designed to treat Duchenne Muscular Dystrophy (DMD) in boys aged 4 to 8. It is a single-arm, open-label trial that will monitor participants for 52 weeks after a single intravenous infusion, with long-term follow-up for up to 5 years. The therapy aims to deliver a therapeutic protein to muscle tissue to help restore function and prevent the progression of muscular dystrophy.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. 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;
2. 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) ;
3. Genetically confirmed diagnosis of DMD;
4. 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;
5. Ability to cooperate with motor assessment testing, magnetic resonance imaging (MRI) and muscle biopsy according to the requirements of the study.

Exclusion Criteria:

1. 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;
2. Receiving antiviral therapy for hepatitis B, hepatitis C, HIV, etc.;
3. Left ventricular ejection fraction (LVEF) \<50% or ≥ class III cardiac function defined by New York Heart Association (NYHA);
4. With severe or persistent arrhythmias and congenital heart disease.
5. The subject\'s preventive treatment/cardiomyopathy treatment changes within 1 month before the start of the study treatment;
6. 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;

Where it's running

• Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine
  Shanghai, Shanghai Municipality, China Recruiting

Study contacts

• Study coordinator: Jiwen Wang
• Email: wangjiwen@scmc.com.cn
• Phone: 18916613192

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.