Impact of Triple Combination CFTR Therapy on Sinus Disease.

Cystic Fibrosis Clinical Trial

Official title:

Impact of Elexacaftor-tezacaftor-ivacaftor Triple Combination CFTR Therapy on Sinus Disease: Quantitative Sinus Computed Tomography, Patient Reported Outcomes and Cellular and Molecular Changes

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04056702
• Other study ID #: HS-3236
• Status: Recruiting
• Start date: September 5, 2019
• Est. completion date: July 2022

Study information

• Verified date: March 2021
• Source: National Jewish Health
• Contact: Connor Balkissoon
• Phone: 877-225-5654
• Email: balkissoonc[@]njhealth.org
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The study's main goal is to observe how effective elexacaftor-tezacaftor-ivacaftor is for improving the symptoms and signs of CF-related sinus disease.

Description:

To determine if elexacfator-tezacaftor-ivacaftor improves the signs and symptoms of CF-related sinus disease, before and after initiation of triple combination therapy, each subject will undergo sinus CT scan and complete questionnaires related to signs and symptoms of sinus disease.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 31
• Est. completion date: July 2022
• Est. primary completion date: July 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 89 Years

Eligibility

Inclusion Criteria:

1. Subjects from 18 to 89 years old.

2. Subjects with cystic fibrosis and comorbid chronic sinus disease (historic diagnosis based on physician assessment of signs and symptoms and medical record).

3. Subjects who clinically elect to initiate elexacaftor-tezacaftor-ivacaftor therapy and/or that are ineligible for CFTR modulator therapy based on their genotype, but have class I/II mutations based on sweat chloride > 90 mmol/L.

Exclusion Criteria:

1. Subjects under the age of 18 or over the age of 89.

2. Subjects who do not elect to initiate elexacaftor-tezacaftor-ivacaftor for clinical reasons.

3. Subjects who had sinus surgery within the last 6 months or will have sinus surgery during the study period.

4. Subjects who have had a recent pulmonary exacerbation or viral infection within two weeks of initial visit.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Paranasal Sinus Diseases

Intervention

Drug:
• Elexacaftor-tezacaftor-ivacaftor exposure
Once a clinical decision has been made to prescribed a patient triple combination therapy, people with CF are eligible to participate in this study.

Locations

Country	Name	City	State
United States	National Jewish Health	Denver	Colorado

Sponsors (2)

Lead Sponsor	Collaborator
Jennifer Taylor-Cousar	Cystic Fibrosis Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Sinus CT opacification.	Calculated from an automated quantification of the size of sinuses.	Change in Sinus CT opacification between the initial and 6 month visits.
Secondary	Change in 22-item Sino-Nasal Outcome Test (SNOT-22) score.	Validated, disease-specific measure of sinus symptoms. Contains 22 questions that are all scored from 0 to 5, when summed a total score ranges form 0 to 110. Higher scores represent worse sinus symptoms.	Change in SNOT-22 score between the initial and 6 month visits.
Secondary	Change in Questionnaire for Olfactory Disorders (QOD) score	Validated, disease-specific measure of olfaction, i.e. the ability to smell. Contains 19 questions that are scored form 0 to 3, when summed a total score can range form 0 to 57. Higher scores indicate greater impact on a subjects sense of smell.	Change in QOD score between the initial and 6 month visits.