NCT05643495终止2 期
A Phase 2, Open-label Study Evaluating Efficacy and Safety of VX-864 in Subjects With Alpha-1 Antitrypsin Deficiency Who Have the PiZZ Genotype, Over 48 Weeks
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 20
- 试验地点
- 13
- 主要终点
- Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels
研究概览
简要总结
The purpose of this study evaluates the efficacy and safety of VX-864 in participants with the PiZZ genotype over 48 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
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
- •Participants 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 Ribonucleic acid interference (RNAi) therapy at any time previously
- •Participants for whom discontinuation of augmentation therapy is not considered to be in their best interest, based on the clinical judgement of the treating physician
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Group A
Experimental
Participants received VX-864 every 12 hours (q12h) for 48 weeks or until study drug dosing was terminated.
干预措施: VX-864 (Drug)
Group B
Experimental
Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated, and undergo a second liver biopsy at either Week 24 or Week 48.
干预措施: VX-864 (Drug)
结局指标
主要结局
Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels
时间窗: From Baseline at Week 48
次要结局
- Change in Blood Functional AAT Levels(From Baseline up to Week 48)
- Change in Blood Antigenic AAT Levels(From Baseline up to Week 48)
- Change in Blood Z-polymer Levels(From Baseline up to Week 48)
- Group B: Change in Z-polymer Accumulation in the Liver(From Baseline up to Week 48)
- Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)(Day 1 up to Week 52)
研究者
研究点 (13)
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