Impact of Triple Combination CFTR Therapy on Sinus Disease.

NCT04056702 | Unknown

• 1 other identifier: HS-3236
• Study Type: observational
• Target: 31
• Locations: 1 country
• Sites: 1

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.

Conditions

Cystic Fibrosis

Keywords

Chronic Sinusitis

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• 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.

Study Arms (2)

Prescribed triple combination

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

Drug: Elexacaftor-tezacaftor-ivacaftor exposure

Not eligible for modulators

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.

Interventions

Elexacaftor-tezacaftor-ivacaftor exposure DRUG

Once a clinical decision has been made to prescribed a patient triple combination therapy, people with CF are eligible to participate in this study.

Prescribed triple combination

Eligibility Criteria

• Age: 18 Years - 89 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

People with cystic fibrosis and chronic sinusitis.

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Jennifer Taylor-Cousar: lead
• Cystic Fibrosis Foundation: collaborator

Study Sites (1)

National Jewish Health

Denver, Colorado, 80206, United States

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Jennifer TaylorCousar, M.D.

  National Jewish Health

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Connor Balkissoon

CONTACT

877-225-5654; balkissoonc@njhealth.org

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Medicine

Study Record Dates

• First Submitted: July 27, 2019
• First Posted: August 14, 2019
• Study Start: September 5, 2019
• Primary Completion: July 1, 2021
• Study Completion: July 1, 2022
• Last Updated: March 11, 2021

Record last verified: 2021-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)