Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis

Description

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).

Conditions

Chronic Rhinosinusitis (Diagnosis)

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Study Overview

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Study Details

Study overview

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).

Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis (Randomized Pilot Study Utilizing Ivacaftor for the Treatment of Refractory Gram-Negative Bacterial CRS)

Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis

Condition
Chronic Rhinosinusitis (Diagnosis)
Intervention / Treatment

-

Contacts and Locations

Birmingham

University of Alabama at Birmingham, Birmingham, Alabama, United States, 35233

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * \< 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

Ages Eligible for Study

18 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

University of Alabama at Birmingham,

Bradford Woodworth, MD, PRINCIPAL_INVESTIGATOR, University of Alabama at Birmingham

Study Record Dates

2025-05