Impact of Triple Combination CFTR Therapy on Sinus Disease.

• Conditions: Cystic Fibrosis
• Interventions: Drug: Elexacaftor-tezacaftor-ivacaftor exposure

• Registration Number: NCT04056702
• Lead Sponsor: Jennifer Taylor-Cousar

Brief 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.

Detailed 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.

Study Timeline

• Study First Submitted: 2019-07-27
• Study First Submitted QC: 2019-08-12
• Study First Posted: 2019-08-14
• Study Start: 2019-09-05
• Status Verified: 2021-03
• Last Update Submitted: 2021-03-09
• Last Update Posted: 2021-03-11
• Primary Completion: 2021-07
• Study Completion: 2022-07

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 31

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Prescribed triple combination	Elexacaftor-tezacaftor-ivacaftor exposure	A group of 60 people with CF who are clinically prescribed elexacaftor-tezacaftor-ivacaftor and have chronic sinusitis

Primary Outcome Measures

Name	Time	Method
Change in Sinus CT opacification.	Change in Sinus CT opacification between the initial and 6 month visits.	Calculated from an automated quantification of the size of sinuses.

Secondary Outcome Measures

Name	Time	Method
Change in 22-item Sino-Nasal Outcome Test (SNOT-22) score.	Change in SNOT-22 score between the initial and 6 month visits.	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 Questionnaire for Olfactory Disorders (QOD) score	Change in QOD score between the initial and 6 month visits.	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.

Trial Locations

Locations (1)

National Jewish Health; 🇺🇸 Denver, Colorado, United States