A Phase 3, Randomized, Double-Blind, Ivacaftor-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation and a Second CFTR Allele With a Gating Defect That Is Clinically Demonstrated to be Ivacaftor Responsive
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 156
- 主要终点
- Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8
研究概览
简要总结
This is a Phase 3, randomized, double-blind, ivacaftor-controlled, parallel-group, multicenter study of tezacaftor in combination with ivacaftor in subjects aged 12 years and older with CF who are heterozygous for the F508del-CFTR mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Heterozygous for F508del-CFTR mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive
- •FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height during screening
- •Stable CF disease as judged by the investigator.
排除标准
- •History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.
- •Pregnant and nursing females (females of childbearing potential must have a negative pregnancy test at Screening and Week -4 Visits).
- •Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements
研究组 & 干预措施
Ivacaftor (Run-in Period)
Ivacaftor 150 milligram (mg) tablet orally every 12 hours for 4 weeks.
干预措施: Ivacaftor (Drug)
VX-661 + Ivacaftor (Active comparator period)
VX-661 100 mg and ivacaftor 150 mg fixed-dose combination tablet orally once daily in the morning and ivacaftor 150 mg tablet orally once daily in the evening for 8 weeks.
干预措施: Ivacaftor (Drug)
VX-661 + Ivacaftor (Active comparator period)
VX-661 100 mg and ivacaftor 150 mg fixed-dose combination tablet orally once daily in the morning and ivacaftor 150 mg tablet orally once daily in the evening for 8 weeks.
干预措施: Tezacaftor/Ivacaftor (Drug)
Ivacaftor monotherapy (Active comparator period)
Ivacaftor 150 mg tablet orally every 12 hours as monotherapy for 8 weeks.
干预措施: Ivacaftor (Drug)
结局指标
主要结局
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8
时间窗: Baseline, Through Week 8
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
次要结局
- Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline Through Week 8(Baseline, Through Week 8)
- Relative Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8(Baseline, Through Week 8)
- Absolute Change From Baseline in Sweat Chloride Through Week 8(Baseline, Through Week 8)
- Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)(Baseline up to Week 16)
- Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1-VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)(Predose on Week -2 for Run-in period; Pre-dose on Week 2 for Active comparator period)