A Randomized, Placebo-Controlled, Double-Blind, Single-Dose, Phase 1 Study to Explore the PK/PD Relationship of QRL-101 in People Living With ALS
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- QRL-101 plasma concentration
研究概览
简要总结
This Phase 1 randomized, placebo-controlled, double-blind study will evaluate the PK/PD relationship of single doses of QRL-101 in 12 ALS participants. The study will also assess safety and tolerability in participants receiving either QRL-101 or placebo.
详细描述
This is a randomized, placebo-controlled, double-blind, single-dose, single-site, Phase 1 study to explore the pharmacokinetics (PK) and pharmacodynamics (PD) of single doses of QRL-101 in participants with ALS. In addition, the safety and tolerability of QRL-101 will be evaluated. Single doses of QRL-101 or placebo will be administered as an oral liquid to approximately 12 participants with ALS. The study will explore the PK, PD, and PK/PD relationship among three dose levels of QRL-101 and placebo. The randomization will be 1:1:1:1 for the four study groups and will be conducted in 3 blocks (cohorts) of 4, where participants will be randomized 3:1 (QRL-101:placebo) for increasing dose levels of QRL-101.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
QRL-101
Single doses of QRL-101 will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data.
干预措施: QRL-101 (Drug)
Placebo
Single doses of comparator placebo will be orally administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data.
干预措施: Placebo (Other)
结局指标
主要结局
QRL-101 plasma concentration
时间窗: Baseline through Follow up (Day 10)
PK/PD Relationship: Strength-Duration Time Constant (SDTC)
时间窗: Baseline through Follow up (Day 10)
次要结局
- Safety and Tolerability: Adverse Events (AEs)(Baseline through Follow up (Day 10))
- Safety and Tolerability: Serious Adverse Events (SAEs)(Baseline through Follow up (Day 10))
- Plasma PK profile: Maximum concentration observed (Cmax)(Baseline through Follow up (Day 10))
- Plasma PK profile: Time of maximum drug concentration (Tmax)(Baseline through Follow up (Day 10))
- Plasma PK profile: Area under the concentration time curve from time 0 to 24 hours (AUC0-24hr)(Baseline through Follow up (Day 10))