Sweat chloride as a biomarker of CFTR activity: proof of concept and ivacaftor clinical trial data.

ABSTRACT:

Background

We examined data from a Phase 2 trial {NCT00457821} of ivacaftor, a CFTR potentiator, in cystic fibrosis (CF) patients with aG551D mutation to evaluate standardized approaches to sweat chloride measurement and to explore the use of sweat chloride and nasal potential difference (NPD) to estimate CFTR activity.

Methods

Sweat chloride and NPD were secondary endpoints in this placebo-controlled, multicenter trial. Standardization of sweat collection, processing,and analysis was employed for the first time. Sweat chloride and chloride ion transport (NPD) were integrated into a model of CFTR activity.

Results

Within-patient sweat chloride determinations showed sufficient precision to detect differences between dose-groups and assess ivacaftor treatment effects. A

SUBMITTER: Accurso FJ 

PROVIDER: S-EPMC4102431 | biostudies-literature | 2014 Mar

REPOSITORIES: biostudies-literature