Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis

Recruiting now · Early Phase 1

Conditions studied: Chronic Rhinosinusitis (Diagnosis)

In brief

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).

Key facts

Study ID

NCT03439865

Run by

University of Alabama at Birmingham

People needed

20

Starts

2019-05-02

Expected to finish

2027-05-01

Last updated by the study team

2026-04-22

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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 sufficient for catheter placement
• Negative pregnancy test for females of childbearing potential within 72 hours of testing and start of study treatment

You may not qualify if…

• < 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

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States (enrolling)

Full record on ClinicalTrials.gov

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