A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR Mutation
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 130
- 试验地点
- 55
- 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 evaluates the long-term safety and tolerability of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 years and older, 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
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Completed the Week 24 Visit in Study 113 Part B or the Week 8 Visit in Study 115
- •Eligible CFTR Mutation
排除标准
- •Pregnant and nursing females
- •History of poor compliance with study drug and/or procedures in a previous study as deemed by the investigator
- •Ongoing participation in another study with investigational drug
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
TEZ/IVA
Part A: Participants weighing less than (<)40 kilograms (kg) at Day 1 received tezacaftor (TEZ) 50 milligrams (mg) once daily (qd)/ivacaftor (IVA) 75 mg every 12 hours (q12h) and the participants weighing greater than or equals to (>=) 40 kg at Day 1 received TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 96 weeks. Doses were adjusted upward for changes in body weight and/or age.
Part B: Participants weighing <30 kg at Day 1 received TEZ 50 mg qd/IVA 75 mg q12h and the participants weighing >=30 kg at Day 1 received TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 192 weeks. Doses were adjusted upward for changes in body weight and/or age.
干预措施: TEZ/IVA (Drug)
TEZ/IVA
Part A: Participants weighing less than (<)40 kilograms (kg) at Day 1 received tezacaftor (TEZ) 50 milligrams (mg) once daily (qd)/ivacaftor (IVA) 75 mg every 12 hours (q12h) and the participants weighing greater than or equals to (>=) 40 kg at Day 1 received TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 96 weeks. Doses were adjusted upward for changes in body weight and/or age.
Part B: Participants weighing <30 kg at Day 1 received TEZ 50 mg qd/IVA 75 mg q12h and the participants weighing >=30 kg at Day 1 received TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 192 weeks. Doses were adjusted upward for changes in body weight and/or age.
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: Day 1 up to Week 100
次要结局
- Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 115/116 FAS (TEZ/IVA Group)(From Parent Study 115 Baseline at Week 96 (Study 116))
- Part A: Absolute Change in Body Mass Index (BMI) for 115/116 FAS (TEZ/IVA Group)(From Parent Study 115 Baseline at Week 96 (Study 116))
- Part A: Absolute Change in CFQ-R Respiratory Domain Score for 113B/116 FAS(From Parent Study 113B Baseline at Week 96 (Study 116))
- Part A: Absolute Change in BMI for 113B/116 FAS(From Parent Study 113B Baseline at Week 96 (Study 116))
- Part A: Absolute Change in Lung Clearance Index2.5 (LCI2.5) for 115/116 FAS (TEZ/IVA Group)(From Parent Study 115 Baseline at Week 96 (Study 116))
- Part A: Absolute Change in LCI2.5 for 113B/116 LCI FAS(From Parent Study 113B Baseline at Week 96 (Study 116))
- Part A: Absolute Change in Sweat Chloride (SwCl) for 115/116 FAS (TEZ/IVA Group)(From Parent Study 115 Baseline at Week 96 (Study 116))
- Part A: Absolute Change in SwCl for 113B/116 FAS(From Parent Study 113B Baseline at Week 96 (Study 116))
- Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)(Day 1 up to Week 192)