Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation
Status:
Completed
Trial end date:
2012-11-01
Target enrollment:
Participant gender:
Summary
The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects
with cystic fibrosis aged 12 years and older who have the 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 3
Details
Lead Sponsor:
Vertex Pharmaceuticals Incorporated
Collaborators:
Cystic Fibrosis Foundation Cystic Fibrosis Foundation Therapeutics