[HTML][HTML] Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation
K De Boeck, A Munck, S Walker, A Faro, P Hiatt… - Journal of Cystic …, 2014 - Elsevier
K De Boeck, A Munck, S Walker, A Faro, P Hiatt, G Gilmartin, M Higgins
Journal of Cystic Fibrosis, 2014•ElsevierBackground 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 FEV 1 through 8 and 24 weeks of ivacaftor …
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 FEV 1 through 8 and 24 weeks of ivacaftor …
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.
Elsevier