EUCTR2020-002093-27-FR进行中（未招募）1 期
Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular DystrophyA phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long term safety follow up part, in ambulant boys. - Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Genethon
- 入组人数
- 51
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- Male
入选标准
- •2Being included in the GNT-014-MDYF study
- •36 to 10 years (inclusive)
- •4Positive gene testing with detailed genotyping confirmation of DMD, ie DMD mutations expected to abolish the production of dystrophin
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 51
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1Presence of neutralizing antibodies against AAV8
- •2Cardiomyopathy based on physical/cardiological examination and echocardiography with Left Ventricular Ejection Fraction (LVEF) below 55% and/or fractional shortening (SF) below 28%
- •3Any respiratory assistance needed including non-invasive daytime or nocturnal ventilation
- •4Inability to perform the planned respiratory functions tests
研究者
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A clinical study in 3 parts with a microdystrophin (called GNT0004), a new gene therapy in boys with Duchenne disease who can still walk. The study will start with finding the proper treatment dose (part 1). After that, a comparative study versus placebo will start to assess the safety and the effectiveness of the proper dose of this therapy (part 2). In the end, a follow up period will continue to investigate the treatment safety and efficacy over longer time (part 3).MedDRA version: 20.0Level: PTClassification code: 10013801Term: Duchenne muscular dystrophy Class: 100000004850Duchenne Muscular DystrophyCTIS2023-505187-11-00Genethon51
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