Elsevier

Journal of Cystic Fibrosis

Volume 13, Issue 6, December 2014, Pages 674-680
Journal of Cystic Fibrosis

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

https://doi.org/10.1016/j.jcf.2014.09.005Get rights and content
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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 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.

Keywords

Ivacaftor
Gating mutation
Potentiator
G551D

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