Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation
Status:
Completed
Trial end date:
2011-04-01
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 6 to 11 years who have the G551D mutation in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective
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 adenosine monophosphate (AMP)-dependent protein kinase A (PKA)
activation.
Phase:
Phase 3
Details
Lead Sponsor:
Vertex Pharmaceuticals Incorporated
Collaborators:
Cystic Fibrosis Foundation Cystic Fibrosis Foundation Therapeutics