Actively Recruiting
A Single-Arm, Open-Label, Single-Dose Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 Injection in Boys with Duchenne Muscular Dystrophy
Led by Shanghai Jiao Tong University School of Medicine · Updated on 2025-03-25
6
Participants Needed
1
Research Sites
208 weeks
Total Duration
On this page
Sponsors
S
Shanghai Jiao Tong University School of Medicine
Lead Sponsor
S
Shanghai Mianyi Biopharmaceutical Co., Ltd.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating BBM-D101, a gene addition therapy, to treat boys diagnosed with Duchenne Muscular Dystrophy (DMD). This early phase 1 study aims to assess the safety, tolerability, and effectiveness of BBM-D101 by delivering a therapeutic protein gene to muscles to help prevent muscular dystrophy and improve muscle function. The study is sponsored by Shanghai Jiao Tong University School of Medicine and focuses on DMD boys aged 4 to under 8 years old. Participants will receive a single intravenous infusion of BBM-D101. The study includes a 52-week period after infusion to evaluate safety, tolerability, efficacy, pharmacokinetics, pharmacodynamics, and immune response. After this initial period, long-term safety and efficacy will be monitored for up to 5 years. The treatment involves a recombinant adeno-associated virus vector designed to deliver the therapeutic protein gene into muscle tissue. During the study, boys will undergo various assessments including motor tests like the North Star Ambulatory Assessment and timed walking or climbing tasks. Muscle biopsies will be performed to measure BBM-D101 gene copies and therapeutic protein levels. Researchers will monitor adverse events and serious adverse events over 52 weeks and dose limiting toxicity within 12 weeks. Participants’ progress and safety will be followed closely, with the total observation lasting up to 5 years post infusion.
CONDITIONS
Brief Title
Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Legal guardian understands the study and signs informed consent
- Ambulatory male subjects aged 4 to less than 8 years
- Genetically confirmed diagnosis of Duchenne Muscular Dystrophy
- At least one clinical sign or lab abnormality typical of DMD (e.g., proximal muscle weakness, waddling gait, pseudo gastrocnemius hypertrophy, Gower's sign, pterygoid scapula)
- Ability to cooperate with motor assessments, MRI, and muscle biopsy as required by the study
You will not qualify if you...
- Positive for hepatitis B surface antigen, hepatitis B virus DNA ≥1000 U/mL, hepatitis C virus RNA positive, or HIV positive
- Receiving antiviral therapy for hepatitis B, hepatitis C, HIV, or similar
- Left ventricular ejection fraction less than 50% or New York Heart Association class III or higher cardiac function
- Severe or persistent arrhythmias or congenital heart disease
- Changes to preventive or cardiomyopathy treatment within 1 month before study treatment start
- Underlying liver disease such as portal hypertension, splenomegaly, hepatic encephalopathy, advanced liver fibrosis, or significant liver abnormalities judged clinically relevant by investigator
- Elevated alpha-fetoprotein or liver nodules/cysts detected during screening considered clinically significant by investigator
AI-Screening
AI-Powered Screening
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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 and are monitored for safety, tolerability, and efficacy.
Regular visits throughout 52 weeks for assessments
Duration - Up to 5 years
Participants are followed for up to 5 years post-infusion to evaluate long-term safety and efficacy of BBM-D101.
Periodic visits during long-term follow-up
Trial Site Locations
Total: 1 location
1
Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China, 200127
Actively Recruiting
Research Team
J
Jiwen Wang
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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