CTIS2023-505187-11-00招募中1 期
Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy: A 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 - GNT-016-MDYF
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 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
入排标准
- 年龄范围
- 0 至 17（—）
- 性别
- Male
入选标准
- •Ambulant Male, Being included in the GNT-014-MDYF study, 6 to 10 years (inclusive), Body weight =75th percentile of the BMI scale (validated chart in force in country site), Positive gene testing with detailed genotyping confirmation of Duchenne Muscular Dystrophy (DMD), i.e. DMD mutations expected to abolish the production of dystrophin except subjects with any mutations affecting exons 1 through 17
排除标准
- •DMD subjects with any mutations affecting exons 1 through 17 and/or with any mutations affecting exons 29 and 30, Presence of neutralizing antibodies against AAV8, Cardiomyopathy based on physical/cardiological examination and echocardiography with Left Ventricular Ejection Fraction (LVEF) below 55% and/or fractional shortening (SF) below 28%, Any respiratory assistance needed including non-invasive daytime or nocturnal ventilation, Inability 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 dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersDuchenne Muscular DystrophyEUCTR2020-002093-27-FRGenethon51
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