A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Effect of VX-770 on Lung Clearance Index in Subjects With Cystic Fibrosis, the G551D Mutation, and FEV1 >90% Predicted
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 21
- 主要终点
- Absolute Change From Baseline in Lung Clearance Index (LCI)
研究概览
简要总结
The purpose of this study is to evaluate the effect of ivacaftor (VX-770) on lung clearance index (LCI) in subjects aged 6 years and older with cystic fibrosis (CF) who have the G551D-CFTR mutation on at least 1 allele.
详细描述
Currently, limited objective measures are available to quantify lung function in CF patients with mild lung disease. Lung clearance index (LCI) derived from inert gas multiple-breath washout (MBW) testing hold considerable promise to evaluate early lung disease as studies have detected abnormalities in a high percentage of CF patients with normal spirometry in both infants and children.
This study explored the effect of ivacaftor on LCI and the efficacy of ivacaftor on other clinical and biomarker endpoints of CF lung disease in subjects aged 6 years and older with CF who have the G551D-CFTR mutation on at least 1 allele.
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
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female subjects with confirmed diagnosis of CF
- •Must have the G551D-CFTR mutation in at least 1 allele
- •FEV1 >90% of predicted normal for age, gender, and height
排除标准
- •Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within the 30 days prior to screening
- •Use of inhaled hypertonic saline treatment within 2 weeks of the Period 1, Day 1 visit
研究组 & 干预措施
Treatment Sequence 1
Ivacaftor administered in Treatment Period 1 and placebo administered in Treatment Period 2.
干预措施: Ivacaftor (Drug)
Treatment Sequence 1
Ivacaftor administered in Treatment Period 1 and placebo administered in Treatment Period 2.
干预措施: Placebo (Drug)
Treatment Sequence 2
Placebo administered in Treatment Period 1 and ivacaftor administered in Treatment Sequence 2.
干预措施: Ivacaftor (Drug)
Treatment Sequence 2
Placebo administered in Treatment Period 1 and ivacaftor administered in Treatment Sequence 2.
干预措施: Placebo (Drug)
结局指标
主要结局
Absolute Change From Baseline in Lung Clearance Index (LCI)
时间窗: Baseline through Day 29
Lung clearance index (LCI) is a measure of ventilation inhomogeneity that is derived from a multiple-breath washout test. The LCI was calculated as the number of lung volume turnovers (cumulative expired volume divided by the functional residual capacity \[FRC\]) required to reduce end-tidal SF6 concentration to 1/40th of the starting value.
次要结局
- Absolute Change From Baseline in Percent Predicted FEV1(Baseline through Day 29)
- Change From Baseline in Sweat Chloride(Baseline through Day 29)
- Change From Baseline in CF Questionnaire-Revised (CFQ-R) Score (Respiratory Domain Score, Pooled)(Baseline through Day 29)