Study design (if review, criteria of inclusion for studies)
randomised, double-blind, placebo-controlled, dose-escalation and safety study
Participants
19 adults with CF (homozygous deltaF508)
Interventions
Three dose levels (20, 30, or 40 g divided t.i.d.) of drug or placebo were given for 1 week.
Outcome measures
Serial measurements of chloride transport by nasal potential difference (NPD) testing and metabolic safety testing were performed.
Main results
A maximum tolerated dose of 20 g was defined based on minimal adverse reactions, the safety profile, and a statistically significant induction of chloride transport that was maximal by day 3.
Authors' conclusions
This short-term phase I/II study demonstrates proof of principle that modulation of deltaF508 CFTR biosynthesis and trafficking is a viable therapeutic approach for cystic fibrosis.