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Gene Therapy Trial Report

Summary

A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.


NCTID NCT07058662 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Duchenne Muscular Dystrophy (DMD)
Disease Ontology Term DOID:11723
Compound Name BBM-D101
Sponsor Belief BioMed (Beijing) Co., Ltd
Funder Type Industry
Recruitment Status
Enrollment Count 9 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant Undisclosed
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Undisclosed
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell Undisclosed
Delivery System Viral transduction
Vector Type AAV
Editor Type none
Dose 1 Undisclosed dose

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2025-07-01
Completion Date 2031-06-30
Last Update 2025-12-04

Participation Criteria


Eligible Age 4 Years - 9 Years
Standard Ages Child
Sexes Eligible for Study MALE
Eligibility Criteria
Inclusion Criteria: 1. The Participants and/or his legal guardian must fully understand the purpose, nature, methods, and potential risks of the study, and sign a written informed consent form. 2. Ambulatory male subjects aged 4 years and above but under 9 years (4 years ≤ age \< 9 years). 3. Any mutation in the DMD gene confirmed by genetic testing 4. Serum creatine kinase (CK) during the screening period meets the study requirements. 5. Receiving stable, standard-dose glucocorticoids before screening. 6. The subject's AAV capsid antibodies meet the clinical trial requirements. 7. Able to cooperate with motor function assessment, MRI, and muscle biopsy as required by the study. 8. Laboratory test results during the screening period and at baseline meet the standards. 9. The subject and/or his legal guardian must fully understand the study procedures, be willing to actively cooperate, commit to high compliance with the protocol, and ensure that the subject attends all scheduled visits. Exclusion Criteria: 1. Positive for hepatitis B surface antigen (HBsAg), hepatitis B virus deoxyribonucleic acid (HBV-DNA) ≥ 1000 U/mL, hepatitis C virus ribonucleic acid (HCV-RNA) positive, human immunodeficiency virus (HIV) positive, or positive for Treponema pallidum antibodies. 2. Currently receiving antiviral therapy for hepatitis B, hepatitis C, HIV, etc. 3. The investigator deems the subject has severe behavioral or cognitive disorders that may hinder participation in this study. 4. Poorly controlled asthma, or Duchenne Muscular Dystrophy (DMD) leading to significant decline in lung function, or recurrent infectious pneumonia that the investigator considers may affect respiratory function. 5. Left ventricular ejection fraction (LVEF) \< 50% or New York Heart Association (NYHA) cardiac function class ≥ III. 6. Severe or persistent arrhythmias (such as atrial fibrillation, frequent ventricular premature beats, ventricular bigeminy, ventricular trigeminy, severe bundle branch block, etc.), and congenital heart disease that is evaluated by the investigator as unsuitable for participation in this study. 7. Any changes in preventive/cardiomyopathy treatment (initiation of treatment, drug changes, dosing regimen changes, treatment interruption, termination, or restart) within 1 month before the infusion of the study drug. 8. History of liver diseases such as portal hypertension, splenomegaly, hepatic encephalopathy, liver fibrosis ≥ stage 3, or hepatic nodules/cysts found by ultrasound during screening, or elevated alpha-fetoprotein with clinical significance as determined by the investigator. 9. Severe infection (such as pneumonia, pyelonephritis, or meningitis) within 4 weeks before the treatment visit (enrollment may be postponed). 10. History of gene therapy or cell therapy (such as stem cell transplantation). 11. History of or current presence of autoimmune diseases, severe renal, gastrointestinal, neurological, or coagulation disorders, malignant tumors, or other diseases. 12. Other diseases that the investigator deems unsuitable for participation in this study.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates IND cleared January 2025

Resources/Links