Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis (Randomized Pilot Study Utilizing Ivacaftor for the Treatment of Refractory Gram-Negative Bacterial CRS)
University of Alabama at Birmingham
Summary
The purpose of this pilot study is to explore wither ivacaftor in refractory CRS patients will demonstrate safety and tolerability; restore CFTR-mediated Cl- secretions as measured by EDSPD testing; produce detectable improvements in validated measures of CRS including the SNOT-22 questionnaire, Lund-MacKay CT scan grading, and Lund-Kennedy endoscopic scores; and provide beneficial effects on readily measured markers of sinonasal inflammation and infection (IP-10, IL-8, and Pseudomonas CFUs).
Description
There is a significant clinical need for new treatment modalities for chronic sinus disease. Chronic rhinosinusitis (CRS) causes substantial morbidity and detracts from quality of life for 16% of the US population. The disease accrues an estimated aggregated cost of $8.6b annual in healthcare expenditures. Patients with CRS describe poorer scores for physical pain and social functioning on quality of life questionnaires than those suffering from chronic obstructive pulmonary disease (COPD), congestive heart failure, or angina. Conventional CRS treatments are comprised of antibiotics, steroids,…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * 18 years of age * Patient has provided informed consent * Diagnosis of CRS made by one of the investigators * Standard of care CT scan with definitive demonstration of isolated or diffuse mucosal thickening, bone changes, and air fluid levels, obtained within 30 days of treatment * Positive culture of at least one gram negative bacteria (e.g. Pseudomonas, E. coli, Steenotrophomonas) within 30 days prior to testing * Previous surgery with (at least) exposed maxillary and ethmoid sinuses * Ability to perform EDSPD testing such that nasal cavity space and sinus openings are…
Interventions
- DrugIvacaftor
150 mg tablet PO BID x 14 days
- Drugstandard of care treatment
topical nasal steroid spray and culture-directed antibiotics x 14 days
Location
- University of Alabama at BirminghamBirmingham, Alabama