NCT04226274已完成1 期
A Phase 1b, Open-label Study to Evaluate the Safety and Tolerability of 12 Weeks Treatment With Oral REN001 in Patients With McArdle Disease (Glycogen Storage Disorder 5)
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 19
- 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 McArdle Disease
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
- 18 Years 至 —（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 McArdle Disease
- •Subjects must be able to remain on stable medication through the study and specifically must not commence or have changes to agents that affect metabolism such as medication for diabetes
- •Follow a stable dietary regimen as documented by a 3-day dietary record obtained during the screening period.
排除标准
- •Documented history of ongoing rhabdomyolysis
- •Evidence of acute crisis from their underlying disease
- •Currently following or planning to start a ketogenic diet
- •Treatment with an investigational drug within 1 month or within 5 half-lives, whichever is longer
- •Have been hospitalized within the 3 months prior to screening for any major medical condition
- •Severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the Investigator in discussion with the Medical Monitor, would make the subject inappropriate for entry into this study
- •Pregnant or nursing females
研究组 & 干预措施
REN001
Experimental
Oral
干预措施: REN001 (Drug)
结局指标
主要结局
Adverse Events
时间窗: up to Week 12
Number of participants with Adverse Events (AEs) as a measure of safety and tolerability
次要结局
未报告次要终点
研究者
研究点 (2)
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