LATE-BREAKING ABSTRACT: Significant improvement in refractory chronic cough with inhaled PA101 in patients with idiopathic pulmonary fibrosis: Results from phase 2 trial

Abstract

<b>Introduction</b> Cough can be a debilitating symptom of idiopathic pulmonary fibrosis (IPF) and is difficult to treat. PA101 is a novel formulation of cromolyn sodium delivered via a high-efficiency nebuliser (PARI eFlow^®) that achieves significantly higher lung deposition compared to previous formulations. We investigated the efficacy and safety of inhaled PA101 on chronic cough in IPF patients in a randomised, double-blind, placebo controlled, 2-period cross-over trial. <b>Methods</b> 24 IPF patients with refractory cough (mean age 67yr, male 63%, mean FVC 73% and median cough duration 4.0yr) were randomized to receive PA101 (40mg tid) or matching placebo for 2 weeks, followed by 2 weeks wash out, and then crossed over. Objective 24hr cough frequency (Leicester Cough Monitor) and subjective cough-specific QoL (Leicester Cough Questionnaire, LCQ) were assessed at Day 14. Analysis: ITT/Mixed Effects Model. <b>Results</b> There was a significant 31% reduction in daytime cough frequency at Day 14 vs baseline with PA101 when adjusted for placebo (p=0.02). LCQ scores improved with PA101 compared to placebo (mean diff 1.1, p=0.09). PA101 was well tolerated, adverse events were comparable to placebo, and there were no serious AEs. <b>Conclusions</b> Inhaled PA101 appears to be a promising treatment option for refractory cough in IPF and warrants further investigation. <b>Daytime average cough frequency.</b>