Conditions: Cystic Fibrosis
Allocation: RANDOMIZED | Intervention model: CROSSOVER
Enrollment: 21
Healthy volunteers: no

Brief summary:
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.

Detailed description:
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.

Arms:
arm 1: Ivacaftor administered in Treatment Period 1 and placebo administered in Treatment Period 2. arm 2: Placebo administered in Treatment Period 1 and ivacaftor administered in Treatment Sequence 2.

Interventions: intervention 1: Ivacaftor intervention 2: Placebo
Intervention descriptions: intervention 1: 150 mg tablet, oral use, twice daily every 12 hours (q12h) intervention 2: Tablet, oral use, twice daily every 12 hours (q12h)

Protocol document: