title: Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation.
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.
authors.affiliation: University Hospital Gasthuisberg, Leuven, Belgium. Electronic address: Christiane.DeBoeck@uzleuven.be.
authors.forename: Kris
authors.initials: K
authors.lastname: De Boeck
authors.affiliation: Assistance publique-Hôpitaux de Paris, Hôpital Robert Debré, Paris, France.
authors.forename: Anne
authors.initials: A
authors.lastname: Munck
authors.affiliation: Emory University, Atlanta, GA, USA.
authors.forename: Seth
authors.initials: S
authors.lastname: Walker
authors.affiliation: Washington University School of Medicine, St. Louis, MO, USA.
authors.forename: Albert
authors.initials: A
authors.lastname: Faro
authors.affiliation: Baylor College of Medicine, Houston, TX, USA.
authors.forename: Peter
authors.initials: P
authors.lastname: Hiatt
authors.affiliation: Vertex Pharmaceuticals Incorporated, Boston, MA, USA.
authors.forename: Geoffrey
authors.initials: G
authors.lastname: Gilmartin
authors.affiliation: Vertex Pharmaceuticals Incorporated, Boston, MA, USA.
authors.forename: Mark
authors.initials: M
authors.lastname: Higgins
mesh_terms: D000293:Adolescent; D000328:Adult; D000627:Aminophenols / Q000627:therapeutic use*; D015992:Body Mass Index; D002648:Child; D018592:Cross-Over Studies; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000235:genetics*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics*; D004311:Double-Blind Method; D005260:Female; D005541:Forced Expiratory Volume; D006801:Humans; D015640:Ion Channel Gating / Q000235:genetics*; D008297:Male; D008875:Middle Aged; D009154:Mutation / Q000235:genetics*; D017063:Outcome Assessment, Health Care; D015363:Quinolones / Q000627:therapeutic use*; D055815:Young Adult
chemical_list: D000627:Aminophenols; D015363:Quinolones; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; C545203:ivacaftor
keywords: G551D; Gating mutation; Ivacaftor; Potentiator