Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation.

ABSTRACT:

Background

Ivacaftor (VX-770) is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that was approved in the United States for the treatment of cystic fibrosis (CF) in patients ≥ 6 years of age who have a G551D mutation; however, the most prevalent disease-causing CFTR mutation, F508del, causes a different functional defect. The objectives of this study were to evaluate the safety of ivacaftor in a larger population and for a longer time period than tested previously and to assess the efficacy of ivacaftor in subjects with CF who are homozygous for F508del-CFTR.

Methods

This was a phase 2 study with a 16-week randomized (4:1), double-blind, placebo-controlled period (part A) and an open-label extension (part B) for subjects who met prespecified criteria

SUBMITTER: Flume PA 

PROVIDER: S-EPMC3435140 | biostudies-literature | 2012 Sep

REPOSITORIES: biostudies-literature