• J. Cyst. Fibros. · Dec 2014

    Randomized Controlled Trial

    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, and Mark Higgins.
    • University Hospital Gasthuisberg, Leuven, Belgium. Electronic address: Christiane.DeBoeck@uzleuven.be.
    • J. Cyst. Fibros. 2014 Dec 1;13(6):674-80.

    BackgroundIvacaftor 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.MethodsPatients 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.ResultsEight 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.ConclusionsIvacaftor was efficacious in a group of patients with CF who had selected non-G551D gating mutations.Copyright © 2014 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.

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