RecruitingNCT06191640

Sinus Disease in Young Children With Cystic Fibrosis

Sponsor

University of California, Los Angeles

Enrollment

80 participants

Start Date

Apr 12, 2023

Study Type

OBSERVATIONAL

Conditions

Cystic Fibrosis Chronic Rhinosinusitis (Diagnosis)Olfactory Impairment Cystic Fibrosis in Children Olfactory Disorder

Summary

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.

Eligibility

Min Age: 2 YearsMax Age: 8 Years

Inclusion Criteria9

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 Criteria5

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

Print for Your Doctor

Interested in this trial?

Get notified about updates and connect with the research team.