Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy

Launched by SHANGHAI JIAO TONG UNIVERSITY SCHOOL OF MEDICINE · Oct 14, 2024

Keywords

ClinConnect Summary

This clinical trial is looking at a new treatment called BBM-D101 for boys with Duchenne Muscular Dystrophy (DMD), a genetic condition that causes muscle weakness. The main goals of the study are to see if BBM-D101 is safe to use and if it can help improve muscle function in young boys. The trial is currently recruiting participants who are boys aged between 4 and 8 years old and have a confirmed diagnosis of DMD. To participate, they should also show typical signs of the condition, such as difficulty walking or weakness in certain muscles.

If eligible, participants will undergo various assessments, including tests to check their muscle strength and imaging scans. It's important to note that boys with certain health issues, like significant heart problems or liver disease, cannot take part in the study. This trial is an important step in finding new ways to help manage DMD, and families interested in participating should discuss it with their healthcare provider to understand the potential benefits and risks.

Gender

MALE

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;