BBM-D101 gene therapy for boys with Duchenne muscular dystrophy

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.

This trial will try a single intravenous dose of BBM-D101 gene therapy in ambulatory boys aged 4 to under 9 with Duchenne muscular dystrophy to see if it is safe and helps muscle function.

Quick facts

• Phase: PHASE1; PHASE2
• Study type: Interventional
• Enrollment: 9 (estimated)
• Ages: 4 Years to 9 Years
• Sex: Male
• Sponsor: Belief BioMed (Beijing) Co., Ltd (industry)
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT07058662 on ClinicalTrials.gov

What this trial studies

This is a single-arm, open-label Phase 1/2 trial giving one intravenous infusion of BBM-D101, an AAV-based gene addition therapy designed to deliver a therapeutic protein to muscle. Participants will be followed closely for 52 weeks for safety, tolerability, pharmacokinetics, pharmacodynamics, immune responses, and preliminary efficacy, with additional long-term safety and efficacy follow-up up to 5 years. Key procedures include motor function assessments, MRI, muscle biopsy, and laboratory monitoring; participants must be ambulatory boys aged 4 to under 9 with genetically confirmed DMD and acceptable AAV antibody and lab profiles. The study focuses on whether the delivered protein can support the dystrophin-associated complex and improve muscle function while monitoring for immune or other adverse effects.

Who should consider this trial

Good fit: Ideal candidates are ambulatory male children aged 4 to under 9 with a confirmed DMD mutation who are on stable glucocorticoids and meet the trial's AAV antibody and laboratory criteria.

Not a fit: Patients who are non-ambulatory, outside the 4 to under 9 age range, have high anti-AAV antibodies, or have disqualifying laboratory or medical conditions are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, BBM-D101 could restore or improve muscle function and slow disease progression by delivering a therapeutic protein to affected muscles.

How similar studies have performed: Other AAV-based gene addition efforts for DMD, including micro-dystrophin trials, have shown early promise in improving function for some patients but have faced immune response and durability challenges.

Eligibility criteria

Inclusion Criteria:

1. 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.
2. Ambulatory male subjects aged 4 years and above but under 9 years (4 years ≤ age \< 9 years).
3. Any mutation in the DMD gene confirmed by genetic testing
4. Serum creatine kinase (CK) during the screening period meets the study requirements.
5. Receiving stable, standard-dose glucocorticoids before screening.
6. The subject's AAV capsid antibodies meet the clinical trial requirements.
7. Able to cooperate with motor function assessment, MRI, and muscle biopsy as required by the study.
8. Laboratory test results during the screening period and at baseline meet the standards.
9. 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 Criteria:

1. 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.
2. Currently receiving antiviral therapy for hepatitis B, hepatitis C, HIV, etc.
3. The investigator deems the subject has severe behavioral or cognitive disorders that may hinder participation in this study.
4. 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.
5. Left ventricular ejection fraction (LVEF) \< 50% or New York Heart Association (NYHA) cardiac function class ≥ III.
6. 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.
7. 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.
8. 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.
9. Severe infection (such as pneumonia, pyelonephritis, or meningitis) within 4 weeks before the treatment visit (enrollment may be postponed).
10. History of gene therapy or cell therapy (such as stem cell transplantation).
11. History of or current presence of autoimmune diseases, severe renal, gastrointestinal, neurological, or coagulation disorders, malignant tumors, or other diseases.
12. Other diseases that the investigator deems unsuitable for participation in this study.

Where this trial is running

Beijing, Beijing Municipality

• Peking Union Medical College Hospital — Beijing, Beijing Municipality, China (RECRUITING)

Study contacts

• Principal investigator: Yi Dai, MD — Peking Union Medical College Hospital
• Study coordinator: Hanyang Hu, Ph.D

Conditions: DMD, gene therapy, Adeno-Associated Virus