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

NCT07058662 | Recruiting Phase 1 FDA Drug

• 1 other identifier: BBM041-CLN1001
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of the study is to evaluate the safety, tolerability, and efficacy of BBM-D101 to treat participants with Duchenne Muscular Dystrophy.

Conditions

DMD

Keywords

DMD; gene therapy; Adeno-Associated Virus

Outcome Measures

Primary Outcomes (2)

• Incidence of dose limiting toxicity (DLT) events

  To evaluate the rate of incidence of DLT events determined by the Safety Data Review Committee (SRC) in DLT observation period after BBM-D101 infusion.

  Within 12 weeks

• Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)

  To evaluate the safety of BBM-D101 by AEs and SAEs.

  Within 12 weeks

Secondary Outcomes (8)

• Changes from baseline in BBM-D101 therapeutic protein level of muscle biopsy samples

  Within 52 weeks

• Changes from baseline in serum Creatine Kinase (CK) level

  Within 52 weeks

• Changes from baseline in the time to ascend time to rise (TTR) without assistance

  Within 52 weeks

• Changes from baseline in the time to ascend 10-meter walk/run test (10MWR) without assistance

  Within 52 weeks

• Changes from baseline in the time to ascend 4 steps (4-stair climb, 4-SC) without assistance.

  Within 52 weeks

Study Arms (1)

Arm of BBM-D101

EXPERIMENTAL

Single-dose treatment

Genetic: Single dose intravenous of BBM-D101

Interventions

Single dose intravenous of BBM-D101 GENETIC

BBM-D101 is a gene addition therapy based on engineered AAV delivery therapeutic protein gene cassette into muscle for treating DMD. Therapeutic protein could mediate the dystrophin-associated protein complex to prevent muscular dystrophy and to rescue the function of muscle.The administration is completed by a single intravenous infusion.

Arm of BBM-D101

Eligibility Criteria

• Age: 4 Years - 9 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.
• Ambulatory male subjects aged 4 years and above but under 9 years (4 years ≤ age \< 9 years).
• Any mutation in the DMD gene confirmed by genetic testing
• Serum creatine kinase (CK) during the screening period meets the study requirements.
• Receiving stable, standard-dose glucocorticoids before screening.
• The subject's AAV capsid antibodies meet the clinical trial requirements.
• Able to cooperate with motor function assessment, MRI, and muscle biopsy as required by the study.
• Laboratory test results during the screening period and at baseline meet the standards.
• 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.

You may not qualify if:

• 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.
• Currently receiving antiviral therapy for hepatitis B, hepatitis C, HIV, etc.
• The investigator deems the subject has severe behavioral or cognitive disorders that may hinder participation in this study.
• 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.
• Left ventricular ejection fraction (LVEF) \< 50% or New York Heart Association (NYHA) cardiac function class ≥ III.
• 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.
• 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.
• 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.
• Severe infection (such as pneumonia, pyelonephritis, or meningitis) within 4 weeks before the treatment visit (enrollment may be postponed).
• History of gene therapy or cell therapy (such as stem cell transplantation).
• History of or current presence of autoimmune diseases, severe renal, gastrointestinal, neurological, or coagulation disorders, malignant tumors, or other diseases.
• Other diseases that the investigator deems unsuitable for participation in this study.

Sponsors & Collaborators

• Belief BioMed (Beijing) Co., Ltd: lead
• Shanghai Mianyi Biopharmaceutical Co., Ltd.: collaborator
• Shanghai Xinzhi BioMed Co., Ltd.: collaborator
• Belief BioMed Limited: collaborator

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

RECRUITING

Study Officials

• Yi Dai, MD

  Peking Union Medical College Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Hanyang Hu, Ph.D

CONTACT

+86-021-33588288; huhanyang@beliefbiomed.com

DMD Clinical Trial Team

CONTACT

DMDclinicaltrial@beliefbiomed.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 1, 2025
• First Posted: July 10, 2025
• Study Start: July 31, 2025
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): June 30, 2031
• Last Updated: December 4, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)