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J Cyst Fibros. 2014 Dec;13(6):674-80. doi: 10.1016/j.jcf.2014.09.005. Epub 2014 Sep 26.

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

Author information

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.

KEYWORDS:

G551D; Gating mutation; Ivacaftor; Potentiator

PMID:
25266159
DOI:
10.1016/j.jcf.2014.09.005
[Indexed for MEDLINE]
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