2023-505187-11-00招募中2 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
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- Genethon
- 入组人数
- 20
- 试验地点
- 6
- 主要终点
- NSAA: change from baseline at week 52
研究概览
简要总结
A phase I/II/III study consisting of 3 parts:
- Part 1: To determine the dose of IMP: a safe and tolerable dose with acceptable gene expression, to carry over to part 2.
- Part 2: To demonstrate clinical efficacy of IMP vs placebo at 1 year after inclusion. To assess the safety and tolerability of IMP vs placebo at 1 year after inclusion.
- Part 3. To assess the safety and tolerability of IMP
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 性别
- Male
- 接受健康志愿者
- 是
入选标准
- •Ambulant Male
- •Being included in the GNT-014-MDYF study
- •6 to 10 years (inclusive)
- •For participants enrolled in Part 1: BMI scale ≤75th percentile (validated chart in force in country site) For participants enrolled in Part 2: Body weight ≤95th percentile of the BMI or body weight 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
排除标准
- •DMD patients with any mutations affecting: - exons 1 through 17, (and any mutations affecting other exons as per a country’s requirement which will be applicable to this specific country) for Part 1 participants - affecting exons 8 and/or 9 (and any mutations affecting other exons as per a country’s requirement which will be applicable to this specific country) for Part 2 participants
- •Presence of neutralizing antibodies against AAV8
- •Cardiomyopathy based on physical/cardiological examination and echocardiography (or cardiac MRI if available) with Left Ventricular Ejection Fraction (LVEF) below 55% and fractional shortening (SF) below 28%
- •Any respiratory assistance needed including non-invasive daytime or nocturnal ventilation
- •Inability to perform the planned respiratory functions tests
结局指标
主要结局
NSAA: change from baseline at week 52
NSAA: change from baseline at week 52
次要结局
- - Safety and tolerability, measured by the incidence of adverse event (AE) or serious adverse event (SAE) evaluated by changes in laboratory parameters, vital signs and in the physical examination
- PK/PD endpoints including vector shedding quantification in blood, urine, saliva, feces
- Clinical efficacy endpoints including NSAA, Time to 10 Meters Walk/Run Test (10MWRT), Time to Rise From Floor (RFF), 6-Minutes Walk Test (6MWT)
研究者
Clinical Development Department
Scientific
Genethon
研究点 (6)
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