Actively Recruiting
Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy
Led by Shanghai Jiao Tong University School of Medicine · Updated on 2025-03-25
6
Participants Needed
1
Research Sites
313 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
The purpose of the study is to assess the safety, tolerability, and efficacy of BBM-D101 to treat patients with Duchenne Muscular Dystrophy.
CONDITIONS
Official 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...
- The legal guardian understands the study and signs informed consent
- Ambulatory male subjects aged at least 4 years and less than 8 years
- Genetically confirmed diagnosis of Duchenne Muscular Dystrophy
- Presence of clinical signs or lab abnormalities typical of DMD such as proximal muscle weakness, waddling gait, pseudo gastrocnemius hypertrophy, Gower's sign, or 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 treatment for hepatitis B, hepatitis C, or HIV
- Left ventricular ejection fraction less than 50% or class III or higher cardiac function by NYHA
- Severe or persistent arrhythmias or congenital heart disease
- Changes in preventive or cardiomyopathy treatment within 1 month before study treatment
- Underlying liver disease such as portal hypertension, splenomegaly, hepatic encephalopathy, or stage 3 or higher hepatic fibrosis, or liver nodules/cysts or elevated alpha-fetoprotein judged clinically significant
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
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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