A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-121/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Through 11 Years of Age
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 210
- 试验地点
- 76
- 主要终点
- Part A: 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
The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
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
- 1 Year 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene
排除标准
- •History of solid organ, hematological transplantation, or cancer
- •Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
- •Lung infection with organisms associated with a more rapid decline in pulmonary status
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Part A: VX-121/TEZ/D-IVA
Participants will receive VX-121/TEZ/D-IVA in the morning.
干预措施: VX-121/TEZ/D-IVA (Drug)
Part B: VX-121/TEZ/D-IVA
Participants will receive VX-121/TEZ/D-IVA in the morning with the dose(s) to be based on the outcome of Part A.
干预措施: VX-121/TEZ/D-IVA (Drug)
结局指标
主要结局
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: From Day 1 up to Day 50
Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: From Day 1 up to Week 28
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites
时间窗: From Day 1 up to Day 22
次要结局
- Part B: Absolute Change in Body Mass Index (BMI)(From Baseline at Week 24)
- Part B: Absolute Change in BMI-for-age Z-score(From Baseline at Week 24)
- Part B: Absolute Change in Weight-for-age Z-score(From Baseline at Week 24)
- Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites(From Day 1 up to Week 16)
- Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)(From Baseline Through Week 24)
- Part B: Proportion of Participants With SwCl <30 mmol/L(From Baseline Through Week 24)
- Part B: Number of CF-Related Hospitalizations(From Baseline Through Week 24)
- Part B: Absolute Change in Weight(From Baseline at Week 24)
- Part B: Absolute Change in Weight-for-length(From Baseline at Week 24)
- Part B: Absolute Change in Height(From Baseline at Week 24)
- Part B: Absolute Change in Height-for-age Z-score(From Baseline at Week 24)
- Part B: Absolute Change in Sweat Chloride (SwCl)(From Baseline Through Week 24)
- Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale(At Day 1 and Week 24)
- Part B: Number of Pulmonary Exacerbation (PEx)(From Baseline Through Week 24)
- Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score(From Baseline Through Week 24)
- Part B: Absolute Change in Length(From Baseline at Week 24)
- Part B: Absolute Change in Length-for-age Z-score(From Baseline at Week 24)
- Part B: Absolute Change in Weight-for-length Z-score(From Baseline at Week 24)
- Part B: Proportion of Participants With SwCl <60 millimole per liter (mmol/L)(From Baseline Through Week 24)