At a glance
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Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients
In Brief
A clinical study evaluating ivacaftor and Placebo for Cystic Fibrosis. Completed, enrolled 10 participants across 1 site.
Detailed Summary
This is a study of the short-term effects of ivacaftor on sweat chloride concentration and lung function in cystic fibrosis (CF) patients who fall outside current FDA approval. This new, first of its kind drug is approved for use only in CF patients with the G551D mutation in whom it safely confers considerable benefits. However, it is highly likely that CF patients with many other mutations can benefit similarly from this drug, some of whom can be identified by phenotype or genotype. We will enroll up to 30 CF subjects with clinical presentations in which there is one or more signs of residual CF channel function. The signs of residual function include: normal digestion, concentration of chloride in sweat between 55 and 85, or milder than expected CF disease in a CF patient with severe gene mutations. The primary outcome measure will be the difference in sweat chloride concentration measured in subjects on placebo and on ivacaftor. Secondary outcome measured will be lung function.
Study Details
Timeline
Interventions
Eligible subjects were randomized to ivacaftor 150 mg by mouth twice a day for 14 days followed by placebo for 14 days, or vice versa. Randomization was based on a computer-generated schedule produced by the research pharmacy, which was concealed from study personnel until study completion. Ivacaftor was purchased at full retail cost and encapsulated with sucrose to match the sucrose-filled placebo capsules. Prior to beginning study drug, there was a 2-week run-in period to ensure clinical stability, assessed by modified Fuchs criteria and pulmonary function. There was a washout period of a minimum of 14 days between study drug cycles to account for carryover effect. The washout period was extended to 6 weeks for subjects on alternating cycles of inhaled antibiotics to coordinate the study drug cycle with the inhaled antibiotic cycle.
Eligible subjects were randomized to ivacaftor 150 mg by mouth twice a day for 14 days followed by placebo for 14 days, or vice versa. Randomization was based on a computer-generated schedule produced by the research pharmacy, which was concealed from study personnel until study completion. Ivacaftor was purchased at full retail cost and encapsulated with sucrose to match the sucrose-filled placebo capsules. Prior to beginning study drug, there was a 2-week run-in period to ensure clinical stability, assessed by modified Fuchs criteria and pulmonary function. There was a washout period of a minimum of 14 days between study drug cycles to account for carryover effect. The washout period was extended to 6 weeks for subjects on alternating cycles of inhaled antibiotics to coordinate the study drug cycle with the inhaled antibiotic cycle.