A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 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
- 已完成
- 入组人数
- 507
- 试验地点
- 110
- 主要终点
- Treatment Period: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
The study evaluates the long-term safety and tolerability of VX-445 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 i.e.VX17-445-102 (NCT03525444) and VX17-445-103 (NCT03525548); 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
ELX/TEZ/IVA
Treatment Period: Participants received ELX 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hrs (q12h) in the treatment period for 192 weeks.
Extension Period: Participants from certain countries participated in extension period and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the extension period for 48 weeks.
干预措施: ELX/TEZ/IVA (Drug)
ELX/TEZ/IVA
Treatment Period: Participants received ELX 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hrs (q12h) in the treatment period for 192 weeks.
Extension Period: Participants from certain countries participated in extension period and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the extension period for 48 weeks.
干预措施: IVA (Drug)
结局指标
主要结局
Treatment Period: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
时间窗: From Day 1 up to Week 196
次要结局
- Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 102/105 Efficacy Set(From Baseline at Week 192)
- Absolute Change in BMI Z-score for 103/105 Efficacy Set(From Baseline at Week 192)
- Number of Pulmonary Exacerbations (PEx) for 102/105 Efficacy Set(From Baseline at Week 192)
- Time to First PEx for 103/105 Efficacy Set(From Baseline at Week 192)
- Absolute Change in BMI Z-score for 102/105 Efficacy Set(From Baseline at Week 192)
- Absolute Change in Sweat Chloride (SwCl) for 103/105 Efficacy Set(From Baseline at Week 192)
- Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/105 Efficacy Set(From Baseline at Week 192)
- Absolute Change in Body Mass Index (BMI) for 103/105 Efficacy Set(From Baseline at Week 192)
- Absolute Change in Sweat Chloride (SwCl) for 102/105 Efficacy Set(From Baseline at Week 192)
- Number of Pulmonary Exacerbations (PEx) for 103/105 Efficacy Set(From Baseline at Week 192)
- Time to First PEx for 102/105 Efficacy Set(From Baseline at Week 192)
- Absolute Change in Body Mass Index (BMI) for 102/105 Efficacy Set(From Baseline at Week 192)
- Absolute Change in Body Weight for 102/105 Efficacy Set(From Baseline at Week 192)
- Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 103/105 Efficacy Set(From Baseline at Week 192)
- Absolute Change in Body Weight for 103/105 Efficacy Set(From Baseline at Week 192)
- Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 102/105 Efficacy Set(From Baseline at Week 192)