A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects With Non-F508del CFTR Genotypes
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 297
- 试验地点
- 181
- 主要终点
- Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
This study will evaluate the long-term safety, efficacy and pharmacodynamics of ELX/TEZ/IVA in participants with cystic fibrosis (CF) with at least 1 non-F508del ELX/TEZ/IVA-responsive CF transmembrane conductance regulator (CFTR) gene 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
- •Part A: Completed study drug treatment in parent study 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
- •Part B: Completed study drug treatment in Part A or had study drug interruption(s) in Part A but completed study visits up to the last scheduled visit of the treatment period of Part A
排除标准
- •History of study drug intolerance in the parent study
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
ELX/TEZ/IVA
Part A: Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening for 96 weeks.
Part B: Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening for an additional 96 weeks.
干预措施: ELX/TEZ/IVA (Drug)
ELX/TEZ/IVA
Part A: Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening for 96 weeks.
Part B: Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening for an additional 96 weeks.
干预措施: IVA (Drug)
结局指标
主要结局
Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Day 1 up to Week 196
次要结局
- Part A: Absolute Change in Weight(From Baseline up to Week 96)
- Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score(From Baseline up to Week 96)
- Part A: Absolute Change in Body Mass Index (BMI)(From Baseline up to Week 96)
- Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 second (ppFEV1)(From Baseline up to Week 96)
- Part A: Absolute Change in Sweat Chloride (SwCl)(From Baseline up to Week 96)
- Part A: Number of Pulmonary Exacerbations (PEx)(From Baseline up to Week 96)