NCT03862846终止1 期
An Open-label Study to Evaluate the Safety and Tolerability of 12 Weeks Treatment With Oral REN001 in Patients With Primary Mitochondrial Myopathy (PMM), With an Optional Extension of Treatment
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 23
- 试验地点
- 2
- 主要终点
- Adverse Events
研究概览
简要总结
The purpose of this study is to assess REN001 safety in subjects with primary mitochondrial myopathy
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects must give written, signed and dated informed consent
- •Confirmed diagnosis of PMM according to the 2016 Rome Consensus recommendations
- •Confirmed mitochondrial mutation with evidence of myopathy
- •Able to remain on stable medication throughout the study
排除标准
- •Documented evidence of ongoing rhabdomyolysis
- •Subjects with motor abnormalities other than related to mitochondrial disease
- •Treatment with an investigational drug within 3 months prior to Day 1
- •Hospitalised within 3 months prior to screening for any major medical condition
- •Pregnant or nursing females
研究组 & 干预措施
Group
Experimental
REN001 Low Dose
干预措施: REN001 (Drug)
结局指标
主要结局
Adverse Events
时间窗: Comparing Baseline to Week 12
Number of participants with Adverse Events as a measure of safety and tolerability
次要结局
- Adverse Events(Continous to Week 48)
研究者
研究点 (2)
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