A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy of Lumacaftor/Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis Who Have an A455E-CFTR Mutation
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 20
- 试验地点
- 2
- 主要终点
- Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 8
研究概览
简要总结
This is a Phase 2, randomized, double-blind, placebo-controlled, multicenter, crossover study that will evaluate the efficacy of LUM/IVA in subjects with CF 12 years of age and older who have at least one A455E mutation.
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
- 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
- •Male or female with confirmed diagnosis of CF
- •All subjects must have an A455E mutation on at least 1 CFTR allele; no more than 10 subjects may have an F508del mutation on 1 CFTR allele.
- •Forced expiratory volume in one second (FEV1) ≥30% of predicted and ≤90% of predicted at the Screening Visit, based on the Global Lung Function Initiative (GLI)-2012 multi ethnic all-age reference equations.
- •Stable CF disease as judged by the investigator.
- •Willing to remain on a stable medication regimen for CF from 4 weeks before Day 1 through the Follow up Visit.
排除标准
- •History of any comorbidity reviewed at the Screening Visit 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.
- •A G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H mutation on at least one CFTR allele.
- •Pregnant or breastfeeding.
- •Any abnormal laboratory values at the Screening Visit.
- •History of cataract/lens opacity, or evidence of cataract/lens opacity determined to be clinically significant by the ophthalmologist or optometrist during the ophthalmologic examination at the Screening Visit.
- •Use of strong inhibitors or strong inducers of CYP3A, including consumption of certain herbal medications and certain fruit and fruit juices, within 14 days before Day 1
- •Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements
研究组 & 干预措施
Treatment Sequence 1
LUM/IVA in Treatment Period 1; washout; placebo in Treatment Period 2
干预措施: LUM/IVA (Drug)
Treatment Sequence 1
LUM/IVA in Treatment Period 1; washout; placebo in Treatment Period 2
干预措施: Placebo (Drug)
Treatment Sequence 2
Placebo in Treatment Period 1; washout; LUM/IVA in Treatment Period 2
干预措施: LUM/IVA (Drug)
Treatment Sequence 2
Placebo in Treatment Period 1; washout; LUM/IVA in Treatment Period 2
干预措施: Placebo (Drug)
结局指标
主要结局
Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 8
时间窗: Study 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
未报告次要终点