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

NCT06641895 | Recruiting Early Phase 1

• 1 other identifier: BBM041-IIT1001
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Duchenne Muscular Dystrophy (DMD)

Keywords

Adeno-Associated Virus; gene therapy; Duchenne muscular dystrophy

Outcome Measures

Primary Outcomes (2)

• Incidence of dose limiting toxicity (DLT) events

  To access the numbers of DLT events determined by the Safety Data Review Committee (SRC) in DLT observation period after BBM-D101 injection infusion.

  12 weeks

• The incidence of adverse events (AEs) and serious adverse events (SAEs)

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

  52 weeks

Secondary Outcomes (7)

• Changes from baseline in the North Star Ambulatory Assessment (NSAA)

  52 weeks

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

  52 weeks

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

  52 weeks

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

  52 weeks

• Changes from baseline in the time to ascend 100-meter walk/run test (100MWR) without assistance

  52 weeks

Study Arms (1)

Single dose intravenous injection of BBM-D101

EXPERIMENTAL

Genetic: BBM-D101

Interventions

BBM-D101 GENETIC

BBM-D101 is a recombinant adeno-associated virus vector-based gene therapy for DMD treatment. It is a suspension for single intravenous (IV) infusion.

Single dose intravenous injection of BBM-D101

Eligibility Criteria

• Age: 4 Years - 8 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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;

Sponsors & Collaborators

• Shanghai Jiao Tong University School of Medicine: lead
• Shanghai Mianyi Biopharmaceutical Co., Ltd.: collaborator

Study Sites (1)

Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, 200127, China

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Jiwen Wang

  Shanghai Children's Medical Center, affiliated to Shanghai Jiao Tong University School of Medicine

  STUDY CHAIR

Central Study Contacts

Jiwen Wang

CONTACT

18916613192; wangjiwen@scmc.com.cn

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: study chair

Study Record Dates

• First Submitted: October 8, 2024
• First Posted: October 15, 2024
• Study Start: July 25, 2024
• Primary Completion (Estimated): July 31, 2026
• Study Completion (Estimated): July 31, 2030
• Last Updated: March 25, 2025

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)