NCT03362242已完成1 期
A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Effect of ARO-AAT on Serum Alpha-1 Antitrypsin Levels in Normal Adult Volunteers
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
- 45
- 试验地点
- 1
- 主要终点
- Number of Participants With Adverse Events (AEs) Possibly or Probably Related to Treatment
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ARO-AAT in healthy adult volunteers.
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 55 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception
- •Willing to provide written informed consent and to comply with study requirements
- •Non-smoker for at least one year
- •Normal lung function
- •No abnormal finding of clinical relevance at Screening
- •Normal AAT level at Screening visit
排除标准
- •Clinically significant health concerns
- •Regular use of alcohol within one month prior to Screening
- •Use of an investigational agent or device within 30 days prior to dosing or current participation in an investigational study
- •Recent use of illicit drugs
- •Use of any drugs or dietary/herbal supplements know to interfere with liver metabolism
- •NOTE: additional inclusion/exclusion criteria may apply, per protocol
研究组 & 干预措施
ARO-AAT
Active Comparator
干预措施: ARO-AAT Injection (Drug)
Placebo
Placebo Comparator
干预措施: Sterile Normal Saline (0.9% NaCl) (Other)
结局指标
主要结局
Number of Participants With Adverse Events (AEs) Possibly or Probably Related to Treatment
时间窗: Part A (single-ascending dose [SAD] phase): up to 29 (+/- 2) days post-dose; Part B (multiple-ascending dose [MAD] phase): up to 113 (+/- 2) days post-dose
次要结局
- Pharmacokinetics (PK) of ARO-AAT: Maximum Observed Plasma Concentration (Cmax)(Part A (single-ascending dose [SAD] phase): up to 48 hours post-dose; Part B (multiple-ascending dose [MAD] phase): up to 48 hours post-dose)
- PK of ARO-AAT: Time to Maximum Plasma Concentration (Tmax)(Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose)
- PK of ARO-AAT: Terminal Elimination Half-Life (t½)(Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose)
- PK of ARO-AAT: Area Under the Plasma Concentration Versus Time Curve From Zero to 24 Hours (AUC0-24)(Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose)
- PK of ARO-AAT: Area Under the Plasma Concentration Versus Time Curve From Zero to Infinity (AUCinf)(Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose)
- Percent Change in Serum Alpha-1 Antitrypsin (AAT) Levels From Day 1 Pre-Dose Baseline to Nadir(Part A (SAD phase): up to 29 (+/- 2) days; Part B (MAD phase): up to 113 (+/- 2) days)
- Duration of Response of Serum AAT levels From Nadir Back to Above 20% of Baseline or Above 90 mg/dL(Part A (SAD phase): up to 29 (+/- 2) days; Part B (MAD phase): up to 113 (+/- 2) days)
研究者
研究点 (1)
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