Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
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Randomized Controlled Trial Multicenter Study
Sweat chloride as a biomarker of CFTR activity: proof of concept and ivacaftor clinical trial data.
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. ⋯ Sweat chloride is useful in multicenter trials as a biomarker of CFTR activity and to test the effect of CFTR potentiators.