A Phase 3, Open-label Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 484
- 试验地点
- 100
- 主要终点
- Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
This study will evaluate the long-term safety and tolerability of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Completed study drug treatment in a parent study; or had study drug interruption(s) in a parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study.
排除标准
- •History of drug intolerance in a parent study that would pose an additional risk to the subject in the opinion of the investigator.
- •Current participation in an investigational drug trial (other than a parent study)
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
VX-659/TEZ/IVA TC
Participants from parent studies VX17-659-102 (NCT03447249) or VX17-659-103 (NCT03460990) were administered VX-659 240 milligrams (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the TC treatment period for up to 96 weeks in the current study VX17-659-105.
干预措施: VX-659/TEZ/IVA (Drug)
VX-659/TEZ/IVA TC
Participants from parent studies VX17-659-102 (NCT03447249) or VX17-659-103 (NCT03460990) were administered VX-659 240 milligrams (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the TC treatment period for up to 96 weeks in the current study VX17-659-105.
干预措施: IVA (Drug)
结局指标
主要结局
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: From Day 1 up to 28 Days After Last Dose of Study Drug or to the Completion of Study Participation Date, Whichever Occurs First in the Current Study 659-105 (up to Week 100)
次要结局
- Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for Participants From the Parent Study 659-102(From Baseline at Week 72 (Study 659-105))
- Absolute Change in BMI Z-score for Participants From the Parent Study 659-102 (Participants <=20 Years Old at Parent Study Baseline)(From Baseline at Week 60 (Study 659-105))
- Absolute Change in Body Weight for Participants From the Parent Study 659-103(From Baseline at Week 72 (Study 659-105))
- Absolute Change in ppFEV1 for Participants From the Parent Study 659-103(From Baseline at Week 72 (Study 659-105))
- Absolute Change in SwCl for Participants From the Parent Study 659-103(From Baseline at Week 24 (Study 659-105))
- Number of Participants With at Least One PEx for Participants From the Parent Study 659-102(From Baseline up to Week 96 (Study 659-105))
- Absolute Change in BMI for Participants From the Parent Study 659-103(From Baseline at Week 72 (Study 659-105))
- Number of Participants With at Least One PEx for Participants From the Parent Study 659-103(From Baseline up to Week 96 (Study 659-105))
- Absolute Change in CFQ-R Respiratory Domain Score for Participants From the Parent Study 659-103(From Baseline at Week 72 (Study 659-105))
- Number of Pulmonary Exacerbations (PEx) for Participants From the Parent Study 659-102(From Baseline up to Week 96 (Study 659-105))
- Absolute Change in Body Weight for Participants From the Parent Study 659-102(From Baseline at Week 72 (Study 659-105))
- Absolute Change in Sweat Chloride (SwCl) for Participants From the Parent Study 659-102(From Baseline at Week 24 (Study 659-105))
- Number of PEx for Participants From the Parent Study 659-103(From Baseline up to Week 96 (Study 659-105))
- Absolute Change in Body Mass Index (BMI) for Participants From the Parent Study 659-102(From Baseline at Week 72 (Study 659-105))
- Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for Participants From the Parent Study 659-102(From Baseline at Week 72 (Study 659-105))
- Absolute Change in BMI Z-score for Participants From The Parent Study 659-103 (Participants <=20 Years Old at Parent Study Baseline)(From Baseline at Week 60 (Study 659-105))