Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation

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 150 mg q12h or placebo for 8 weeks 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 24 weeks of ivacaftor treatment; secondary outcomes were changes in BMI, sweat chloride, and CFQ-R and safety through 8 and 24 weeks 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 24 weeks. Ivacaftor was generally well tolerated. Conclusions: Ivacaftor was efficacious in a group of patients with CF who had selected non-G551D gating mutations. (C) 2014 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.