Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis Randomized Pilot Study Utilizing Ivacaftor for Treatment of Refractory Gram-Negative Bacterial CRS

What this Trial Is About

Researchers are evaluating ivacaftor, a drug originally approved for cystic fibrosis, in patients with chronic rhinosinusitis (CRS) who have acquired CFTR dysfunction. The study aims to assess the safety and tolerability of ivacaftor in refractory CRS patients, and whether it can restore chloride ion secretion through CFTR channels as measured by specialized testing. The trial also examines improvements in CRS symptoms using validated tools like the SNOT-22 questionnaire, CT scans, and endoscopic scores, as well as effects on markers of nasal inflammation and infection. Participants are randomly assigned to one of two groups: one receiving standard care treatment with topical nasal steroid spray and culture-directed antibiotics combined with ivacaftor tablets (150 mg twice daily for 14 days), and the other receiving standard care treatment alone for the same duration. This early phase 1 pilot trial explores a novel approach to treating CRS by enhancing mucociliary clearance through CFTR potentiation, which has not been previously studied in non-cystic fibrosis CRS. During the study, participants undergo assessments including quality of life measurements from screening to day 30, nasal secretion testing, CT scans, and endoscopic evaluations to monitor disease changes. Researchers also track markers of sinonasal inflammation and bacterial presence. Safety is monitored closely, including pregnancy testing for women of childbearing potential. The total participation involves baseline testing, 14 days of treatment, and follow-up to evaluate improvements and side effects.

Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis