Impact of Elexacaftor-tezacaftor-ivacaftor Triple Combination CFTR Therapy on Sinus Disease: Quantitative Sinus Computed Tomography, Patient Reported Outcomes and Cellular and Molecular Changes
Trial Snapshot
- Phase
- Not Applicable
- Sponsor
- Enrollment
- 31
- Locations
- 1
- Primary Endpoint
- Change in Sinus CT opacification.
Study Overview
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 Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 18 Years to 89 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Subjects from 18 to 89 years old.
- •Subjects with cystic fibrosis and comorbid chronic sinus disease (historic diagnosis based on physician assessment of signs and symptoms and medical record).
- •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
- •Subjects under the age of 18 or over the age of
- •Subjects who do not elect to initiate elexacaftor-tezacaftor-ivacaftor for clinical reasons.
- •Subjects who had sinus surgery within the last 6 months or will have sinus surgery during the study period.
- •Subjects who have had a recent pulmonary exacerbation or viral infection within two weeks of initial visit.
Arms & Interventions
Prescribed triple combination
A group of 60 people with CF who are clinically prescribed elexacaftor-tezacaftor-ivacaftor and have chronic sinusitis
Intervention: Elexacaftor-tezacaftor-ivacaftor exposure (Drug)
Outcomes
Primary Outcomes
Change in Sinus CT opacification.
Time Frame: Change in Sinus CT opacification between the initial and 6 month visits.
Calculated from an automated quantification of the size of sinuses.
Secondary Outcomes
- Change in 22-item Sino-Nasal Outcome Test (SNOT-22) score.(Change in SNOT-22 score between the initial and 6 month visits.)
- Change in Questionnaire for Olfactory Disorders (QOD) score(Change in QOD score between the initial and 6 month visits.)
Investigators
Jennifer Taylor-Cousar
Professor of Medicine
National Jewish Health