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Recruiting NCT06641895

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

Early Phase I Interventional Duchenne Muscular Dystrophy (DMD)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: BBM-D101.
Who it may be relevant to
Registry conditions: Duchenne Muscular Dystrophy (DMD). Basic parameters: 4 years — 8 years · Male.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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

Detailed description

This is a single-arm, open-label study to evaluate the safety, tolerability, efficacy, pharmacokinetic, pharmacodynamic, and immune response of BBM-D101 within 52 weeks after a single intravenous infusion in DMD boys, as well as the long-term safety and efficacy of BBM-D101 for up to 5 years post infusion.

BBM-D101 is 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.

Interventions

  • Genetic BBM-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.

Primary outcome measures

  • Incidence of dose limiting toxicity (DLT) events [Time frame: 12 weeks]
  • The incidence of adverse events (AEs) and serious adverse events (SAEs) [Time frame: 52 weeks]
Secondary outcome measures (7)
  • Changes from baseline in the North Star Ambulatory Assessment (NSAA) [Time frame: 52 weeks]
  • Changes from baseline in the time to ascend 10-meter walk/run test (10MWR) without assistance [Time frame: 52 weeks]
  • Changes from baseline in the time to ascend time to rise (TTR) without assistance without assistance [Time frame: 52 weeks]
  • Changes from baseline in the time to ascend 4 steps (4-stair climb, 4SC) without assistance [Time frame: 52 weeks]
  • Changes from baseline in the time to ascend 100-meter walk/run test (100MWR) without assistance [Time frame: 52 weeks]
  • Changes from baseline in BBM-D101 genome copies in muscle biopsy samples [Time frame: 52 weeks]
  • Changes from baseline in BBM-D101 therapeutic protein level in muscle biopsy samples [Time frame: 52 weeks]

Eligibility criteria

Inclusion criteria

  • 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 criteria

  • 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;

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

China · 1 center
  • Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of M — Shanghai

Identifiers

NCT: NCT06641895 · BBM041-IIT1001

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗