Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation
Status:
Terminated
Trial end date:
2013-05-01
Target enrollment:
Participant gender:
Summary
The purpose of this study was to evaluate the safety and efficacy of ivacaftor in
participants with cystic fibrosis (CF) who were aged 12 years or older and were homozygous
for the F508del-CF transmembrane conductance regulator (CFTR) mutation. 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 adenosine monophosphate (AMP)-dependent
protein kinase A (PKA) activation.
Phase:
Phase 2
Details
Lead Sponsor:
Vertex Pharmaceuticals Incorporated
Collaborators:
Cystic Fibrosis Foundation Cystic Fibrosis Foundation Therapeutics