A Phase 3, 2-part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 61
- 试验地点
- 27
- 主要终点
- Part B : 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 will evaluate the safety and pharmacokinetics (PK) of lumacaftor (LUM) and ivacaftor (IVA) in participants 1 to less than 2 years of age with cystic fibrosis (CF), homozygous for F508del (F/F).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Months 至 23 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants will be 1 to less than 2 years of age on day 1 of the relevant part of the study
- •Homozygous for F508del (F/F)
排除标准
- •Any clinically significant laboratory abnormalities at the screening visit that would interfere with the study assessments or pose an undue risk for the participants
- •Solid organ or hematological transplantation
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Part A: LUM/IVA
Participants weighing 7 to less than (<)10 kilograms (kg) at screening received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 10 to <14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Participants weighing greater than or equal to (>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days.
干预措施: LUM (Drug)
Part A: LUM/IVA
Participants weighing 7 to less than (<)10 kilograms (kg) at screening received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 10 to <14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Participants weighing greater than or equal to (>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days.
干预措施: IVA (Drug)
Part B: LUM/IVA
Participants weighing 7 to <9 kg at screening received LUM 75 mg/IVA 94 mg FDC q12h and those weighing 9 to <14 kg received LUM 100 mg/IVA 125 mg q12h for 24 weeks. Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight.
干预措施: LUM (Drug)
Part B: LUM/IVA
Participants weighing 7 to <9 kg at screening received LUM 75 mg/IVA 94 mg FDC q12h and those weighing 9 to <14 kg received LUM 100 mg/IVA 125 mg q12h for 24 weeks. Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight.
干预措施: IVA (Drug)
结局指标
主要结局
Part B : 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 26
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA
时间窗: Pre-dose at Day 8 and Day 15
Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA
时间窗: Day 1 and Day 15
次要结局
- Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)(Pre-dose at Day 8 and Day 15)
- Part B: Absolute Change in Sweat Chloride(From Baseline at Week 24)
- Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)(Pre-dose at Day 15, Week 4, Week 12 and Week 24)
- Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)(From Day 1 up to Day 25)