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

Официальное название

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

Обзор

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

Подробное описание

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.

Вмешательства

• Генная терапия 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.

Первичные конечные точки

• Incidence of dose limiting toxicity (DLT) events [Срок оценки: 12 weeks]
• The incidence of adverse events (AEs) and serious adverse events (SAEs) [Срок оценки: 52 weeks]

Вторичные конечные точки (7)

• Changes from baseline in the North Star Ambulatory Assessment (NSAA) [Срок оценки: 52 weeks]
• Changes from baseline in the time to ascend 10-meter walk/run test (10MWR) without assistance [Срок оценки: 52 weeks]
• Changes from baseline in the time to ascend time to rise (TTR) without assistance without assistance [Срок оценки: 52 weeks]
• Changes from baseline in the time to ascend 4 steps (4-stair climb, 4SC) without assistance [Срок оценки: 52 weeks]
• Changes from baseline in the time to ascend 100-meter walk/run test (100MWR) without assistance [Срок оценки: 52 weeks]
• Changes from baseline in BBM-D101 genome copies in muscle biopsy samples [Срок оценки: 52 weeks]
• Changes from baseline in BBM-D101 therapeutic protein level in muscle biopsy samples [Срок оценки: 52 weeks]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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;

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of M — Шанхай

Идентификаторы

NCT: NCT06641895 · BBM041-IIT1001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗