Evaluating BBM-D101 for treating Duchenne Muscular Dystrophy

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

This study is testing a new gene therapy called BBM-D101 to see if it can help boys aged 4 to 8 with Duchenne Muscular Dystrophy improve their muscle function and slow down the disease.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 6 (estimated)
• Ages: 4 Years to 8 Years
• Sex: Male
• Sponsor: Shanghai Jiao Tong University School of Medicine (Academic / other)
• Locations: 1 site (Shanghai, Shanghai Municipality)
• Trial ID: NCT06641895 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates are ambulatory male children aged 4 to less than 8 years with a genetically confirmed diagnosis of DMD.

Not a fit: Patients with active viral infections such as hepatitis or HIV may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve muscle function and quality of life for patients with Duchenne Muscular Dystrophy.

How similar studies have performed: Other studies using gene therapy approaches for DMD have shown promise, indicating potential for success with this novel treatment.

Eligibility criteria

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 this trial is running

Shanghai, Shanghai Municipality

• 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

Conditions Duchenne Muscular Dystrophy; Adeno-Associated Virus; gene therapy; Duchenne muscular dystrophy