A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 48
- 试验地点
- 28
- 主要终点
- Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels
研究概览
简要总结
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Subjects must have a PiZZ genotype confirmed at screening
- •Plasma AAT levels indicating severe deficiency at screening
排除标准
- •History of a medical condition that could negatively impact the ability to complete the study
- •Solid organ, or hematological transplantation or is currently on a transplant list
- •History of use of gene therapy or RNAi therapy at any time previously
- •Other protocol defined Inclusion/Exclusion criteria may apply
研究组 & 干预措施
Parts A1, A2 and B Combined: Placebo
Participants received placebo matched to VX-814 in the treatment period for 28 days.
干预措施: Placebo (Drug)
Part A1: VX-814 100 milligrams (mg)
Participants received VX-814 100 mg every 12 hours (q12h) in the treatment period for 28 days.
干预措施: VX-814 (Drug)
Part A1: VX-814 200 mg
Participants received VX-814 200 mg q12h in the treatment period for 28 days.
干预措施: VX-814 (Drug)
Parts A1 and A2 Combined: VX-814 400 mg
Participants received VX-814 400 mg q12h in the treatment period for 28 days.
干预措施: VX-814 (Drug)
Part B: VX-814 600 mg
Participants received VX-814 600 mg q12h in the treatment period for 28 days.
干预措施: VX-814 (Drug)
结局指标
主要结局
Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels
时间窗: From Baseline at Day 28
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Day 1 up to Week 8
次要结局
- Change in Plasma Antigenic AAT Levels(From Baseline at Day 28)
- Observed Pre-dose Plasma Concentration of VX-814(Pre-dose at Day 7, Day 14, Day 21, and Day 28)