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).
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
< 18 years of age
Acute illness other than sinusitis within 2 weeks before start of study treatment that, in the opinion of the investigator, would preclude participation
Currently taking medications that are moderate or strong CP3A inhibitors
History of asthma attack requiring emergency room visit or treatment with oral steroids within 2 months prior to study treatment
Parallel
Ivacaftor
Drug150 mg tablet PO BID x 14 days
standard of care treatment
Drugtopical nasal steroid spray and culture-directed antibiotics x 14 days
Improvement in quality of life measures
Comparison of 22-tem Sino-Nasal Outcomes Test (SNOT-22) scores collected at Screening, Day 1, Day 14, and Day 30. The SNOT-22 is a 22-item questionnaire about rhinosinusitis symptoms and the social/emotional consequences for quality of life. The Likert scale ranges from 1-5, where 1 = "No Problem" and 5 = "Problem as bad as it can be." A total score is collected, ranging from 0-110; the higher the score, the worse the outcome.
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University of Alabama at Birmingham
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Collaborator
