RecruitingEarly Phase 1NCT06641895
Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy
A Single-Arm, Open-Label, Single-Dose Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 Injection in Patients with Duchenne Muscular Dystrophy
Sponsor
Shanghai Jiao Tong University School of Medicine
Enrollment
6 participants
Start Date
Jul 25, 2024
Study Type
INTERVENTIONAL
Conditions
Summary
The purpose of the study is to assess the safety, tolerability, and efficacy of BBM-D101 to treat patients with Duchenne Muscular Dystrophy.
Eligibility
Sex: MALEMin Age: 4 YearsMax Age: 8 Years
Inclusion Criteria5
- 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;
- 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) ;
- Genetically confirmed diagnosis of DMD;
- 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;
- Ability to cooperate with motor assessment testing, magnetic resonance imaging (MRI) and muscle biopsy according to the requirements of the study.
Exclusion Criteria6
- 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;
- Receiving antiviral therapy for hepatitis B, hepatitis C, HIV, etc.;
- Left ventricular ejection fraction (LVEF) \<50% or ≥ class III cardiac function defined by New York Heart Association (NYHA);
- With severe or persistent arrhythmias and congenital heart disease.
- The subject\'s preventive treatment/cardiomyopathy treatment changes within 1 month before the start of the study treatment;
- 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;
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Interventions
GENETICBBM-D101
BBM-D101 is a recombinant adeno-associated virus vector-based gene therapy for DMD treatment. It is a suspension for single intravenous (IV) infusion.
Locations(1)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06641895
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