A CFTR potentiator in patients with cystic fibrosis and the G551D mutation.

ABSTRACT:

Background

Increasing the activity of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein is a potential treatment for cystic fibrosis.

Methods

We conducted a randomized, double-blind, placebo-controlled trial to evaluate ivacaftor (VX-770), a CFTR potentiator, in subjects 12 years of age or older with cystic fibrosis and at least one G551D-CFTR mutation. Subjects were randomly assigned to receive 150 mg of ivacaftor every 12 hours (84 subjects, of whom 83 received at least one dose) or placebo (83, of whom 78 received at least one dose) for 48 weeks. The primary end point was the estimated mean change from baseline through week 24 in the percent of predicted forced expiratory volume in 1 second (FEV(1)).

Results

The change from baseline throu

SUBMITTER: Ramsey BW 

PROVIDER: S-EPMC3230303 | biostudies-literature | 2011 Nov

REPOSITORIES: biostudies-literature