Kris De Boeck1, Anne Munck2, Seth Walker3, Albert Faro4, Peter Hiatt5, Geoffrey Gilmartin6, Mark Higgins6. 1. University Hospital Gasthuisberg, Leuven, Belgium. Electronic address: Christiane.DeBoeck@uzleuven.be. 2. Assistance publique-Hôpitaux de Paris, Hôpital Robert Debré, Paris, France. 3. Emory University, Atlanta, GA, USA. 4. Washington University School of Medicine, St. Louis, MO, USA. 5. Baylor College of Medicine, Houston, TX, USA. 6. Vertex Pharmaceuticals Incorporated, Boston, MA, USA.
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.
RCT Entities:
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.
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