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RECRUITINGOBSERVATIONAL

Sinus Disease in Young Children With Cystic Fibrosis

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

This is a prospective, observational study examining the impact of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators on chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) in young children with cystic fibrosis (YCwCF). This study involves two groups: children 2-8 years old, inclusive at initial visit, receiving highly effective modulator therapy (HEMT), and a control group of children 2-8 years old, inclusive at initial visit, not receiving HEMT. Outcomes will include sinus magnetic resonance imaging (MRI) scans, olfactory tests, and quality of life surveys obtained over a two-year period.

Who May Be Eligible (Plain English)

Who May Qualify: HEMT Group: - Children with documentation of a CF diagnosis - Age 2-8 years old at first study visit - CFTR mutation consistent with FDA labeled indication of highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor) - Clinician intent to prescribe ivacaftor or ETI so that enrollment is before start of HEMT Non-HEMT/Control Group: - Children with documentation of a CF diagnosis - Age 2-8 years at first study visit - Ineligible for highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor) based on CFTR mutation or clinical decision not to initiate HEMT if eligible Who Should NOT Join This Trial: For Both Groups: - Use of an investigational drug within 28 days prior to the first study visit - Use of ivacaftor or elexacaftor/tezacaftor/ivacaftor within the 180 days prior to and including the first study visit - Use of chronic oral corticosteroids within the 28 days prior to and including the first study visit. - Sinus surgery within 180 days prior to the first study visit Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: HEMT Group: * Children with documentation of a CF diagnosis * Age 2-8 years old at first study visit * CFTR mutation consistent with FDA labeled indication of highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor) * Clinician intent to prescribe ivacaftor or ETI so that enrollment is before start of HEMT Non-HEMT/Control Group: * Children with documentation of a CF diagnosis * Age 2-8 years at first study visit * Ineligible for highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor) based on CFTR mutation or clinical decision not to initiate HEMT if eligible Exclusion Criteria: For Both Groups: * Use of an investigational drug within 28 days prior to the first study visit * Use of ivacaftor or elexacaftor/tezacaftor/ivacaftor within the 180 days prior to and including the first study visit * Use of chronic oral corticosteroids within the 28 days prior to and including the first study visit. * Sinus surgery within 180 days prior to the first study visit

Treatments Being Tested

DRUG

Ivacaftor or elexacaftor/tezacaftor/ivacaftor

HEMT's are prescribed at the discretion of the treating physician and is not dictated by the principal investigator of this study.

Locations (6)

Children's Hospital Colorado
Aurora, Colorado, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
University of Vermont
Colchester, Vermont, United States
University of Virginia
Charlottesville, Virginia, United States