A Randomized, Placebo-Controlled, Double-blind, Single Ascending Dose, First in Human Phase 1 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of QRL-101 in Healthy Participants
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 88
- 试验地点
- 1
- 主要终点
- Number of participants with one or more treatment emergent adverse events and serious adverse events.
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of ascending single doses of QRL-101 in male and female healthy participants. The findings from this study will be used to inform the development of QRL-101 for people living with ALS.
详细描述
Phase 1, single-site study to evaluate the safety, tolerability, and pharmacokinetics (PK) of ascending single doses of QRL-101 administered orally in healthy male and female participants. Up to 16 cohorts of 8 participants each, randomized 6:2 (QRL-101: placebo) will be tested. The approximate total duration of study participation for each participant may be up to 40 days.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
盲法说明
A participant and investigator-blinded, randomized, placebo-controlled design will be used to minimize bias in this study.
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
QRL-101
Single-ascending doses of QRL-101 will be administered orally to healthy participants
干预措施: QRL-101 (Drug)
Placebo
Single-ascending doses of comparator placebo will be administered orally to healthy participants
干预措施: Placebo (Other)
结局指标
主要结局
Number of participants with one or more treatment emergent adverse events and serious adverse events.
时间窗: Baseline through Follow up (Day 10)
Endpoints: A summary of treatment emergent adverse events (AEs), serious adverse events (SAEs), and other non-serious adverse events, regardless of causality, will be reported in the Reported Adverse Events module.
次要结局
- Pharmacokinetics (plasma): Time of maximum concentration of QRL-101(Baseline through Follow up (Day 10))
- Pharmacokinetics (plasma): Maximum observed concentration of QRL-101(Baseline through Follow up (Day 10))
- Pharmacokinetics (plasma): Area under the concentration time curve from 0 to 24 h (AUC 0-24h) of QRL-101(Baseline through Follow up (Day 10))