A Double-Blind, Placebo-Controlled, Dose-Escalation, Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION-827359, an Antisense Oligonucleotide Inhibitor of ENaC, Administered to Healthy Volunteers and Patients With Cystic Fibrosis
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 98
- 试验地点
- 8
- 主要终点
- Safety and Tolerability as Measured by the Number of Participants with at least one Treatment-Emergent Adverse Event
研究概览
简要总结
This Phase 1/2a study is a double-blinded (subject and Investigator), randomized, placebo-controlled, dose-escalation study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple nebulized doses of IONIS-ENaCRx.
详细描述
This study will be conducted in 3 parts: a single ascending dose (SAD) leading to a multiple ascending dose (MAD) in healthy volunteers, followed by a MAD in patients with cystic fibrosis. The study will enroll up to 88 participants.
The study will consist of 4 single-dose randomized cohorts. Participants enrolled will receive a single inhaled dose of the Study Drug (IONIS-ENaCRx or placebo) on Day 1.
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)
入排标准
- 年龄范围
- 18 Years 至 50 Years（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
IONIS-ENaCRx
Ascending single and multiple doses of IONIS-ENaCRx inhaled or nebulized.
干预措施: IONIS-ENaCRx (Drug)
Placebo
Placebo comparator calculated volume to match active comparator inhaled or nebulized.
干预措施: Placebo (Drug)
结局指标
主要结局
Safety and Tolerability as Measured by the Number of Participants with at least one Treatment-Emergent Adverse Event
时间窗: Up to 113 Days
次要结局
- Tmax: time taken to reach maximal concentration in plasma of IONIS-ENaCRx(Up to 113 Days)
- CL/F: apparent total clearance of IONIS-ENaCRx(Up to 113 Days)
- Cmax: maximum observed drug concentration in plasma of IONIS-ENaCRx(Up to 113 Days)
- t1/2λz: termination half-life of IONIS-ENaCRx(Up to 113 Days)
- AUCt: area under the plasma concentration-time curve from time zero to time t for IONIS-ENaCRx(Up to 113 Days)
- The amount of administered dose of IONIS-ENaCRx excreted in urine over a 24-hour period(Up to 113 Days)