A Phase 2, Randomized, Placebo-Controlled, Double-blind Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 41
- 主要终点
- Absolute Change in Total Brody/CF-CT Score
研究概览
简要总结
The primary purpose of study is to evaluate the treatment effect of tezacaftor in combination with ivacaftor (TEZ/IVA) on chest imaging endpoints using low-dose computed tomography (LDCT) at Week 72, and to evaluate the safety of TEZ/IVA through Week 72.
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Homozygous for the F508del CFTR mutation
- •Confirmed diagnosis of CF
- •Percent predicted forced expiratory volume (ppFEV1) ≥70% of predicted normal for age, sex, and height during screening.
- •Stable CF disease as judged by the investigator
排除标准
- •History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.
- •An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1 (first dose of study drug)
- •Pregnant or nursing females.
- •Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.
- •Any contraindication to undergoing chest imaging, as per the site's institutional guidelines
研究组 & 干预措施
TEZ/IVA
Participants received TEZ 100 milligram (mg)/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks.
干预措施: Tezacaftor/Ivacaftor (Drug)
TEZ/IVA
Participants received TEZ 100 milligram (mg)/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks.
干预措施: Ivacaftor (Drug)
Placebo
Participants received placebo matched to TEZ/IVA fixed dose combination tablet orally once daily in the morning and placebo matched to IVA tablet orally once daily in the evening for 72 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Absolute Change in Total Brody/CF-CT Score
时间窗: From Baseline at Week 72
The exploratory Brody/CF-CT score semi-quantitatively scores the degree of structural lung disease as shown on CT in participants with CF. The score ranges from a minimum of 0 to a maximum of 219 with higher scores indicating more severe structural lung disease.
次要结局
- Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)(Day 1 up to Week 76)