RECRUITING

Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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).

Official Title

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

Quick Facts

Study Start:2019-05-02

Study Completion:2025-05

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT03439865

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.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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

Inclusion Criteria

Exclusion 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

Contacts and Locations

Study Contact

Norma Miller, RN

CONTACT

(205) 975-6169

ncmiller@uabmc.edu

Principal Investigator

Bradford Woodworth, MD

PRINCIPAL_INVESTIGATOR

University of Alabama at Birmingham

Study Locations (Sites)

University of Alabama at Birmingham

Birmingham, Alabama, 35233

United States

Collaborators and Investigators

Sponsor: University of Alabama at Birmingham

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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2019-05-02

Study Completion Date2025-05

Study Record Updates

Study Start Date2019-05-02

Study Completion Date2025-05

Terms related to this study

Additional Relevant MeSH Terms

Chronic Rhinosinusitis (Diagnosis)