A new study demonstrates that idrevloride in hypertonic saline is safe and associated with a significant improvement in lung function over a 28-day period in individuals with primary ciliary dyskinesia (PCD) when compared to hypertonic saline alone.
The results are published in The Lancet Respiratory Medicine.
“Before this manuscript, there were only two published, randomized controlled trials of any treatment for PCD, and much of our current practice has generally been extrapolated from other forms of bronchiectasis, like cystic fibrosis,” says Thomas W. Ferkol, Jr, MD, a pediatric pulmonologist at University of North Carolina (UNC) Health and chief of pulmonology in the UNC Department of Pediatrics, in a release.
CLEAN-PCD was a phase 2, randomized, double-blind, placebo-controlled crossover trial conducted at 32 adult and pediatric care centers and university hospitals around the world. A total of 123 adults and adolescents with primary ciliary dyskinesia aged 12 years or older were randomly assigned to receive idrevloride in hypertonic saline, hypertonic saline alone, idrevloride alone, or placebo.
Idrevloride is an inhaled investigational epithelial sodium channel (ENaC) inhibitor that is formulated to hydrate the mucus in the lung and improve clearance of mucus by cough and lung function. The ENaC inhibitor has been well-tolerated in multiple clinical trials in healthy volunteers and patients with other respiratory diseases who accumulate excessively concentrated mucus in their lungs, including primary ciliary dyskinesia.
“There is a clear need for newer and better treatments for this rare lung disease, and hopefully the planned, more extensive clinical trials will further show the benefits of inhaled idrevloride in hypertonic saline,” says Ferkol in a release.
The CLEAN-PCD study results were also presented at the European Respiratory Society International Congress 2023 during the Abstracts Leading to Evolution in Respiratory Medicine Trials session.