Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation
Kris De Boeck, Anne Munck, Seth Walker, Albert Faro, Peter Hiatt, Geoffrey Gilmartin, Mark Higgins, Kris De Boeck, Anne Munck, Seth Walker, Albert Faro, Peter Hiatt, Geoffrey Gilmartin, Mark Higgins
Abstract
Background: Ivacaftor is used to treat patients with CF and a G551D gating mutation; the KONNECTION study assessed the efficacy and safety of ivacaftor in patients with CF and a non-G551D gating mutation.
Methods: Patients with CF ≥6-years- old with non-G551D gating mutations received ivacaftor 150mg q12h or placebo for 8weeks in this 2-part, double-blind crossover study (Part 1) with a 16-week open-label extension (Part 2). The primary efficacy outcome was absolute change in FEV1 through 8 and 24weeks of ivacaftor treatment; secondary outcomes were changes in BMI, sweat chloride, and CFQ-R and safety through 8 and 24weeks of treatment.
Results: Eight weeks of ivacaftor resulted in significant improvements in percent predicted FEV1, BMI, sweat chloride, and CFQ-R scores that were maintained through 24weeks. Ivacaftor was generally well tolerated.
Conclusions: Ivacaftor was efficacious in a group of patients with CF who had selected non-G551D gating mutations.
Keywords: G551D; Gating mutation; Ivacaftor; Potentiator.
Copyright © 2014 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.
Source: PubMed