
The phase 2 randomized, double-blind, placebo-controlled trial investigated the safety and tolerability of ivacaftor in 40 patients with moderate to severe chronic obstructive pulmonary disease (COPD) exhibiting cystic fibrosis transmembrane conductance regulator (CFTR) dysfunction. Participants received ivacaftor (N=30) or placebo (N=10) for 12 weeks. Ivacaftor was found to be safe, with adverse event rates similar between groups; however, it did not enhance CFTR activity, mucociliary clearance, lung function, or respiratory symptoms. Given that serum concentrations achieved were lower than those observed in cystic fibrosis, further studies are warranted to determine optimal dosing and treatment efficacy of CFTR modulators in COPD.
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