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

Sponsor

University of Alabama at Birmingham (Other)

Overall Status

Active, not recruiting

CT.gov ID

NCT02997020

Collaborator

National Heart, Lung, and Blood Institute (NHLBI) (NIH)

Enrollment

Location

66.5

Anticipated Duration (Months)

0.5

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
CRS	Other: EDSPD

Detailed 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.

Study Design

Study Type:

Observational

Actual Enrollment :

30 participants

Observational Model:

Case-Control

Time Perspective:

Prospective

Official Title:

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

Actual Study Start Date :

May 18, 2017

Actual Primary Completion Date :

Dec 13, 2019

Anticipated Study Completion Date :

Dec 1, 2022

Arms and Interventions

Arm	Intervention/Treatment
CRS Patients 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.	Other: EDSPD The equipment & conditions for measuring bioelectric changes across the sinus mucosa are based on the standard NPD protocol, including use of agar-filled PE90 tubing for the probing electrode and limiting perfusion solutions to Ringer, Ringer + amiloride (100 µM), chloride-free gluconate with amiloride + isoproterenol (10 µM). 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.
Control Patients 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.	Other: EDSPD The equipment & conditions for measuring bioelectric changes across the sinus mucosa are based on the standard NPD protocol, including use of agar-filled PE90 tubing for the probing electrode and limiting perfusion solutions to Ringer, Ringer + amiloride (100 µM), chloride-free gluconate with amiloride + isoproterenol (10 µM). 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.

Arm

Intervention/Treatment

CRS Patients

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.

Other: EDSPD

The equipment & conditions for measuring bioelectric changes across the sinus mucosa are based on the standard NPD protocol, including use of agar-filled PE90 tubing for the probing electrode and limiting perfusion solutions to Ringer, Ringer + amiloride (100 µM), chloride-free gluconate with amiloride + isoproterenol (10 µM). 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.

Control Patients

Other: EDSPD

Outcome Measures

Primary Outcome Measures

Ability to measure CFTR difference using EDSPD testing [3 Years]
To establish the relationship of EDSPD findings with other validated instruments and markers of CRS disease

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Alabama at Birmingham	Birmingham	Alabama	United States	35294

Sponsors and Collaborators

University of Alabama at Birmingham
National Heart, Lung, and Blood Institute (NHLBI)

Investigators

Principal Investigator: Brad Woodworth, MD, University of Alabama at Birmingham

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Brad Woodworth, MD, Professor of Otolaryngology, University of Alabama at Birmingham

ClinicalTrials.gov Identifier:

NCT02997020

Other Study ID Numbers:

IRB-160516002
1R01HL133006

First Posted:

Dec 19, 2016

Last Update Posted:

Jun 27, 2022

Last Verified:

Jun 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Study Results

No Results Posted as of Jun 27, 2022