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 Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 251
- 试验地点
- 84
- 主要终点
- Part A: 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
This study evaluated the long-term safety, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for the F508del mutation and a gating or residual function mutation (F/G and F/RF genotypes).
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 parent study (VX18-445-104); or had study drug interruption(s) in 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 study drug intolerance in parent study
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
ELX/TEZ/IVA
Part A: Participants received ELX (elexacaftor) 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks.
Part B: Participants from certain countries participated in Part B and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks.
干预措施: ELX/TEZ/IVA (Drug)
ELX/TEZ/IVA
Part A: Participants received ELX (elexacaftor) 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks.
Part B: Participants from certain countries participated in Part B and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks.
干预措施: IVA (Drug)
结局指标
主要结局
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
时间窗: From Baseline up to Week 100
次要结局
- Part A: Absolute Change From Parent Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score(From Baseline at Week 96)
- Part A: Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl)(From Baseline at Week 96)
- Part A: Absolute Change From Parent Study Baseline in Body Mass Index (BMI)(From Baseline at Week 96)
- Part A: Absolute Change From Parent Study Baseline in BMI Z-score(From Baseline at Week 96)
- Part A: Absolute Change From Parent Study Baseline in Body Weight(From Baseline at Week 96)
- Part A: Absolute Change From Parent Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)(From Baseline at Week 96)