A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-659/TEZ/IVA Triple Combination Therapy in Cystic Fibrosis Subjects 6 Through 11 Years of Age
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 18
- 试验地点
- 6
- 主要终点
- Observed Pre-Dose Concentration (Ctrough) of VX-659, TEZ, and IVA
研究概览
简要总结
This study will evaluate the pharmacokinetics (PK), safety, tolerability, efficacy, and pharmacodynamic effect of VX-659, tezacaftor (TEZ), and ivacaftor (IVA) when dosed in triple combination (TC) in Cystic Fibrosis (CF) subjects with F/F or F/MF genotypes.
The study was discontinued after completion of Part A due to Sponsor's discretion.
研究设计
- 研究类型
- Interventional
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Homozygous or heterozygous for F508del mutation (F/F or F/MF genotypes)
- •Forced expiratory volume in 1 second (FEV1) value ≥40% of predicted mean for age, sex, and height.
排除标准
- •Clinically significant cirrhosis with or without portal hypertension
- •Lung infection with organisms associated with a more rapid decline in pulmonary status.
- •Solid organ or hematological transplantation.
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
VX-659/TEZ/IVA
Participants who received VX-659 120 milligram (mg)/TEZ 50 mg/ IVA 75 mg as fixed-dose combination (FDC) in the morning and IVA 75 mg as a mono tablet in the evening in the triple combination (TC) treatment period.
干预措施: VX-659/TEZ/IVA (Drug)
VX-659/TEZ/IVA
Participants who received VX-659 120 milligram (mg)/TEZ 50 mg/ IVA 75 mg as fixed-dose combination (FDC) in the morning and IVA 75 mg as a mono tablet in the evening in the triple combination (TC) treatment period.
干预措施: IVA (Drug)
结局指标
主要结局
Observed Pre-Dose Concentration (Ctrough) of VX-659, TEZ, and IVA
时间窗: Day 8 and Day 15
Maximum Observed Concentration (Cmax) of VX-659, TEZ, and IVA
时间窗: Day 1 and Day 15
Area Under the Concentration Versus Time Curve From Time 0 to 6 Hours (AUC0-6h) of VX-659, TEZ, and IVA
时间窗: Day 1 and Day 15
次要结局
- Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)(From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to 6 weeks))
- Observed Pre-Dose Concentration (Ctrough) of TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)(Day 8 and Day 15)
- Maximum Observed Concentration (Cmax) of TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)(Day 1 and Day 15)
- Area Under the Concentration Versus Time Curve From Time 0 to 6 Hours (AUC0-6h) of TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)(Day 1 and Day 15)