Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis (EDSPD Protocol)

The University of Alabama at Birmingham

Status: Active, not recruiting

Conditions: CRS

Treatments: Other: EDSPD

Study type: Observational

Funder types: Other; NIH

Identifiers

NCT02997020

IRB-160516002

1R01HL133006 (U.S. NIH Grant/Contract)

Details and patient eligibility

About

The purpose of this study is to establish the novel endoscopically-directed sinus potential difference (EDSPD) assay as an endpoint for therapy of sinus disease.

Full description

The purpose of this study is to establish the novel endoscopically-directed sinus potential difference (EDSPD) assay as an endpoint for therapy of sinus disease. This assay provides a means to measure Cl- secretion across the sinus epithelium in human subjects with chronic rhinosinusitis (CRS) and test the relationship between this data and endoscopic findings of inflammation. The technique is appropriate for investigating acquired CFTR dysfunction in sinus epithelium and examining the therapeutic potential of CFTR potentiators for CRS.

Enrollment: 30 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Patient eligibility criteria are designed to limit enrollment otherwise healthy individuals who are scheduled for standard-of-care nasal endoscopy in the clinic or sinus/skull base surgery.

Inclusion criteria:

• Age > 18 years
• Able to give consent
• A diagnosis of CRS, as determined by the Sinus and Allergy Health Partnership criteria
• Has cultured out at least one gram negative bacteria (e.g. Pseudomonas aeruginosa, Escherichia coli, Stenotrophomonas maltophila) within the previous month
• Is scheduled for either routine endoscopic surgery or has previously undergone endoscopic surgery and presents to clinic for routine endoscopic evaluation

Exclusion criteria:

• Age < 18 years
• Unable to give consent
• Active diagnosis of cancer, autoimmune disease, or any know immunodeficiency
• Women who are pregnant or breastfeeding

Trial design

30 participants in 2 patient groups

CRS Patients

Description:

Endoscopically-directed sinus potential difference (EDSPD) will be conducted in either the operating room or in the rhinology clinic setting to quantify CFTR activity in the sinus cavities. The potential difference will be monitored in actively inflamed areas as judged by endoscopy in comparison to an agar-filled reference butterfly electrode placed in the volar aspect of the forearm. A stable potential with the mean value of a 10-s scoring interval after perfusion of each solution will be recorded by a blinded investigator.

Treatment:

Other: EDSPD

Control Patients

Description:

Endoscopically-directed sinus potential difference (EDSPD) will be conducted in either the operating room or in the rhinology clinic setting to quantify CFTR activity in the sinus cavities. The potential difference will be monitored in actively inflamed areas as judged by endoscopy in comparison to an agar-filled reference butterfly electrode placed in the volar aspect of the forearm. A stable potential with the mean value of a 10-s scoring interval after perfusion of each solution will be recorded by a blinded investigator.

Treatment:

Other: EDSPD