Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis
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
20 participants
May 2, 2019
INTERVENTIONAL
Conditions
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).
Eligibility
Inclusion Criteria8
- 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 sufficient for catheter placement
- Negative pregnancy test for females of childbearing potential within 72 hours of testing and start of study treatment
Exclusion Criteria9
- \< 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
- History of solid organ or hematological transplantation
- History of known immunodeficiency, autoimmune or granulomatous disorder
- Serum creatinine \> 1.5x upper normal limit
- Abnormal liver function, as defined by serum AST \> 2x upper normal limit, serum ALT \> 2x upper normal limit, Alkaline phosphatase \> 2x upper normal limit, Total bilirubin \> 2x upper normal limit
- Women who are pregnant or breastfeeding
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Interventions
150 mg tablet PO BID x 14 days
topical nasal steroid spray and culture-directed antibiotics x 14 days
Locations(1)
View Full Details on ClinicalTrials.gov
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NCT03439865