Randomised, phase 1/2a trial of ION-827359, an antisense oligonucleotide inhibitor of ENaC.

Sivagurunathan SutharsanRainald FischerWolfgang GleiberAlex Robert HorsleyJeff CrosbyShuling GuoShuting XiaRosie YuKenneth B NewmanJ Stuart Elborn

Published in: ERJ open research (2024)

The tolerability and safety of ION-827359 appear favourable at this stage of investigation. Reduction in ENaC mRNA supports mechanistic efficacy at the doses and regimens tested, and supports further investigation of ION-827359 in pwCF.

Keyphrases

open label
clinical trial
study protocol
phase iii
double blind
phase ii
placebo controlled
randomized controlled trial