Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation.

ABSTRACT:

Background

A new approach in the treatment of cystic fibrosis involves improving the function of mutant cystic fibrosis transmembrane conductance regulator (CFTR). VX-770, a CFTR potentiator, has been shown to increase the activity of wild-type and defective cell-surface CFTR in vitro.

Methods

We randomly assigned 39 adults with cystic fibrosis and at least one G551D-CFTR allele to receive oral VX-770 every 12 hours at a dose of 25, 75, or 150 mg or placebo for 14 days (in part 1 of the study) or VX-770 every 12 hours at a dose of 150 or 250 mg or placebo for 28 days (in part 2 of the study).

Results

At day 28, in the group of subjects who received 150 mg of VX-770, the median change in the nasal potential difference (in response to the administration of a chloride-fr

SUBMITTER: Accurso FJ 

PROVIDER: S-EPMC3148255 | biostudies-literature | 2010 Nov

REPOSITORIES: biostudies-literature