Safety Study of Ivacaftor in Subjects With Cystic Fibrosis
Status:
Completed
Trial end date:
2008-08-01
Target enrollment:
Participant gender:
Summary
The purpose of this study was to evaluate the safety and tolerability of ivacaftor in
patients with cystic fibrosis (CF) who were aged 18 years or older and have a G551D mutation
in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent
and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR
protein. Potentiators are pharmacological agents that increase the chloride ion transport
properties of the channel in the presence of cyclic AMP-dependent protein kinase A (PKA)
activation.
Phase:
Phase 2
Details
Lead Sponsor:
Vertex Pharmaceuticals Incorporated
Collaborators:
Cystic Fibrosis Foundation Cystic Fibrosis Foundation Therapeutics