A Phase 1/2, Open-label Clinical Study to Evaluate the Safety, Tolerability and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.

Status: Recruiting
Location: See location...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY

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

Eligibility
Participation Requirements
Sex: Male
Minimum Age: 4
Maximum Age: 9
Healthy Volunteers: f
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• 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.

Locations
Other Locations
China
Peking Union Medical College Hospital
RECRUITING
Beijing
Contact Information
Primary
Hanyang Hu, Ph.D
huhanyang@beliefbiomed.com
+86-021-33588288
Time Frame
Start Date: 2025-07-31
Estimated Completion Date: 2031-06-30
Participants
Target number of participants: 9
Treatments
Experimental: Arm of BBM-D101
Single-dose treatment
Sponsors
Leads: Belief BioMed (Beijing) Co., Ltd
Collaborators: Belief BioMed Limited, Shanghai Mianyi Biopharmaceutical Co., Ltd., Shanghai Xinzhi BioMed Co., Ltd.

This content was sourced from clinicaltrials.gov