Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis

Launched by UNIVERSITY OF ALABAMA AT BIRMINGHAM · Feb 14, 2018

Keywords

ClinConnect Summary

This clinical trial is looking at a medication called ivacaftor to see if it can help patients with chronic rhinosinusitis (CRS) who have not found relief from other treatments. The study aims to determine if ivacaftor is safe and well-tolerated, if it can improve the function of a specific protein that helps with mucus clearance in the sinuses, and if it can lead to measurable improvements in symptoms and inflammation related to CRS. Participants will be evaluated using questionnaires, imaging tests, and other medical assessments to track their progress.

To participate in this study, individuals must be at least 18 years old and have a confirmed diagnosis of CRS, along with specific findings on a recent CT scan. They should also have a positive culture for certain bacteria related to sinus infections. However, some people may not be eligible, such as those with recent acute illnesses, certain medical conditions, or who are pregnant or breastfeeding. Participants will receive the study medication and will be closely monitored throughout the trial to ensure their safety and gather important information about the treatment's effects.

Gender

ALL

Eligibility criteria

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