NCT02392234已完成3 期
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy and Safety of Ivacaftor and 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 Allele With a CFTR Mutation Predicted to Have Residual Function
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 248
- 主要终点
- Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Average of Week 4 and Week 8
研究概览
简要总结
The purpose of this study is to evaluate the efficacy and safety of VX-661 in combination with ivacaftor (IVA, VX-770) and IVA monotherapy in participants with Cystic Fibrosis (CF) who are heterozygous for F508del-CFTR allele and a second allele with a CFTR mutation predicted to have residual function.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
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 and a second allele with a CFTR mutation predicted to have residual function
- •Forced Expiratory Volume in 1 Second (FEV1) greater than or equal to (≥) 40 percent (%) and less than or equal to (≤) 90% of predicted normal for age, sex, and height during screening
- •Sweat chloride value ≥60 millimole per liter (mmol/L) during screening OR as documented in the participant's medical record
- •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 participant
- •An acute upper or lower respiratory infection, pulmonary exacerbation
- •History of solid organ or hematological transplantation
- •Ongoing or prior participation in an investigational drug study (including studies investigating VX-661, lumacaftor [VX-809], and/or ivacaftor) within 30 days of screening
- •Pregnant and nursing females
- •Sexually active participants of reproductive potential who are not willing to follow the contraception requirements
研究组 & 干预措施
VX-661/Ivacaftor combination
Experimental
干预措施: VX-661/Ivacaftor (Drug)
VX-661/Ivacaftor combination
Experimental
干预措施: Ivacaftor (Drug)
VX-661/Ivacaftor combination
Experimental
干预措施: Placebo matched to Ivacaftor (Drug)
Ivacaftor monotherapy
Experimental
干预措施: Ivacaftor (Drug)
Ivacaftor monotherapy
Experimental
干预措施: Placebo matched to VX-661/ ivacaftor (Drug)
Placebo
Placebo Comparator
干预措施: Placebo matched to VX-661/ ivacaftor (Drug)
Placebo
Placebo Comparator
干预措施: Placebo matched to Ivacaftor (Drug)
结局指标
主要结局
Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Average of Week 4 and Week 8
时间窗: Baseline, Week 4 and Week 8 of each treatment period
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
次要结局
- Absolute Change From Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Average of Week 4 and Week 8(Baseline, Week 4 and Week 8 of each treatment period)
- Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)(Day 1 up to Week 28)
- Relative Change From Study Baseline in ppFEV1 at Average of Week 4 and Week 8(Baseline, Week 4 and Week 8 of each treatment period)
- Absolute Change From Study Baseline in Sweat Chloride at Average of Week 4 and Week 8(Baseline, Week 4 and Week 8 of each treatment period)
- Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1 VX-661), IVA and IVA Metabolite (M1 IVA) After Administration of VX-661/IVA Combination Therapy(Pre-morning dose on Week 8 of each treatment period)
- Ctrough of IVA and IVA Metabolite (M1 IVA) After Administration of IVA Monotherapy(Pre-morning dose on Week 8 of each treatment period)
研究者
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