Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients
Status:
Completed
Trial end date:
2015-12-01
Target enrollment:
Participant gender:
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.