Actively Recruiting
A Phase 1/2 Open-label Study Evaluating the Safety, Tolerability, and Efficacy of BBM-D101 Gene Therapy in Boys with Duchenne Muscular Dystrophy
Led by Belief BioMed (Beijing) Co., Ltd · Updated on 2025-12-04
9
Participants Needed
1
Research Sites
208 weeks
Total Duration
On this page
Sponsors
B
Belief BioMed (Beijing) Co., Ltd
Lead Sponsor
S
Shanghai Mianyi Biopharmaceutical Co., Ltd.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and effectiveness of BBM-D101, a gene addition therapy, for boys with Duchenne Muscular Dystrophy (DMD). This single-arm, open-label study focuses on boys aged 4 to under 9 years with confirmed DMD gene mutations. BBM-D101 aims to deliver a therapeutic protein to muscle cells that may help support muscle function and prevent muscular dystrophy. Participants will receive a single intravenous infusion of BBM-D101. The study includes an initial 52-week period to assess safety, immune response, pharmacokinetics, and therapeutic effects, followed by a long-term follow-up of up to 5 years to monitor ongoing safety and efficacy. The treatment involves one dose only, with no placebo group. During the study, participants will undergo motor function assessments, muscle biopsy, MRI scans, and laboratory tests including serum creatine kinase levels. Researchers will monitor adverse events and changes in muscle function and biomarkers over time. Follow-up visits will continue for several years to observe long-term effects and safety, with careful tracking of any serious or dose-limiting side effects within the first 12 weeks.
CONDITIONS
Brief Title
A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant or legal guardian understands the study and signs informed consent
- Male, ambulatory boys aged 4 to under 9 years
- Confirmed DMD gene mutation by genetic testing
- Serum creatine kinase meets study requirements
- Stable standard-dose glucocorticoid treatment before screening
- AAV capsid antibodies meet trial requirements
- Able to cooperate with motor function tests, MRI, and muscle biopsy
- Laboratory tests at screening and baseline meet required standards
- Commitment to active participation and attending all scheduled visits
You will not qualify if you...
- Positive for hepatitis B surface antigen, high HBV-DNA, hepatitis C RNA, HIV, or syphilis antibodies
- Currently receiving antiviral therapy for hepatitis B, C, or HIV
- Severe behavioral or cognitive disorders hindering study participation
- Poorly controlled asthma, significant lung function decline from DMD, or recurrent pneumonia affecting breathing
- Left ventricular ejection fraction less than 50% or severe heart failure (NYHA class III or above)
- Severe or persistent arrhythmias or unsuitable congenital heart disease
- Recent changes in cardiomyopathy treatments within 1 month before infusion
- History of serious liver diseases or clinically significant liver abnormalities
- Severe infections within 4 weeks before treatment
- Previous gene or cell therapy history
- Autoimmune diseases, severe kidney, gastrointestinal, neurological, coagulation disorders, cancer, or other serious diseases
- Any other condition deemed unsuitable by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 52 weeks
Participants receive a single intravenous infusion of BBM-D101 gene therapy to evaluate safety, tolerability, and efficacy.
Multiple visits during the first 12 weeks for safety monitoring; additional visits up to 52 weeks for efficacy assessments
Duration - Up to 5 years post infusion
Participants are monitored for long-term safety and efficacy of BBM-D101 gene therapy.
Periodic visits over 5 years for long-term assessment
Trial Site Locations
Total: 1 location
1
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China, 100730
Actively Recruiting
Research Team
H
Hanyang Hu, Ph.D
D
DMD Clinical Trial Team
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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