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Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation

Ivacaftor · Phase 2

Registry ID
NCT00953706
Phase
Phase 2
Status
Terminated
Drug / intervention
Ivacaftor
Sponsor
Vertex Pharmaceuticals Incorporated
Start
2009-09
Enrollment
140
Locations
34

About this trial

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.

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Medical disclaimer. This page summarizes public trial data for NCT00953706 for information only. It is not medical advice, an endorsement, or an offer to enroll. Confirm details on ClinicalTrials.gov and with a qualified physician.