A Phase 3, Open-label Study Evaluating the Pharmacokinetics, Safety, and Tolerability of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 12 to Less Than 24 Months of Age
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 70
- 试验地点
- 19
- 主要终点
- Part A: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites
研究概览
简要总结
This study will evaluate the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD), and efficacy of ELX/TEZ/IVA in CF subjects 12 to less than (<) 24 months of age.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Months 至 24 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants who have at least 1 F508del mutation in the CF transmembrane conductance regulator (CFTR) gene or another ELX/TEZ/IVA-responsive CFTR mutation
排除标准
- •History of any illness or any clinical condition that, in the opinion of the investigator, might either confound the results of the study or pose an additional risk in administering study drug(s) to the participant
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Part A
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
干预措施: IVA (Drug)
Part A
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
干预措施: ELX/TEZ/IVA (Drug)
Part B
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening with the dose(s) to be based on the outcome of Part A.
干预措施: ELX/TEZ/IVA (Drug)
Part B
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening with the dose(s) to be based on the outcome of Part A.
干预措施: IVA (Drug)
结局指标
主要结局
Part A: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites
时间窗: Day 1 up to Day 15
Part 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 28
Part A: 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 Day 43
次要结局
- Part B: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites(Day 15 up to Week 16)
- Part B: Absolute Change in Sweat Chloride (SwCl)(From Baseline Through Week 24)