Impact of Triple Combination CFTR Therapy on Sinus Disease.

Jennifer Taylor-Cousar

Status: Unknown

Conditions: Cystic Fibrosis

Treatments: Drug: Elexacaftor-tezacaftor-ivacaftor exposure

Study type: Observational

Funder types: Other

Identifiers

NCT04056702

HS-3236

Details and patient eligibility

About

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.

Full 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.

Enrollment: 31 estimated patients

Sex: All

Ages: 18 to 89 years old

Volunteers: No Healthy Volunteers

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.

Trial design

31 participants in 2 patient groups

Prescribed triple combination

Description:

A group of 60 people with CF who are clinically prescribed elexacaftor-tezacaftor-ivacaftor and have chronic sinusitis

Treatment:

Drug: Elexacaftor-tezacaftor-ivacaftor exposure

Not eligible for modulators

Description:

A group of 10 patients with two class I/II mutations who are ineligible for elexacaftor-tezacaftor-ivacaftor based on their genotype and have chronic sinusitis.