Sinus Disease in Young Children With Cystic Fibrosis

Last updated: September 24, 2025
Sponsor: University of California, Los Angeles
Overall Status: Active - Recruiting

Phase

N/A

Condition

Cystic Fibrosis

Sinusitis

Nasal Obstruction

Treatment

Ivacaftor or elexacaftor/tezacaftor/ivacaftor

Clinical Study ID

NCT06191640
22-000594
  • Ages 2-8
  • All Genders

Study 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 Criteria

Inclusion

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 modulatortherapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor)

  • Clinician intent to prescribe ivacaftor or ETI so that enrollment is before start ofHEMT

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 orelexacaftor/tezacaftor/ivacaftor) based on CFTR mutation or clinical decision not toinitiate HEMT if eligible

Exclusion

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 toand including the first study visit

  • Use of chronic oral corticosteroids within the 28 days prior to and including thefirst study visit.

  • Sinus surgery within 180 days prior to the first study visit

Study Design

Total Participants: 80
Treatment Group(s): 1
Primary Treatment: Ivacaftor or elexacaftor/tezacaftor/ivacaftor
Phase:
Study Start date:
April 12, 2023
Estimated Completion Date:
April 30, 2026

Study Description

This multi-center, prospective, observational study investigates the effects of highly effective modulator therapy (HEMT) on chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) in young children with cystic fibrosis (YCwCF). The study spans two years and includes two distinct groups of children with cystic fibrosis: children ≤ 8 years old receiving HEMT and a control group of children ≤ 8 not receiving HEMT. The study aims to assess the efficacy of HEMT in improving sinus health and olfactory capabilities in this young demographic.

Key assessments include magnetic resonance imaging (MRI) sinus opacification, olfactory bulb volume measured via MRI, objective olfactory testing, and various quality (QOL) surveys. This investigation seeks to characterize the severity of CRS and OD in YCwCF, and to elucidate if early initiation of HEMT improves CRS and OD .

In the HEMT group, participants will have a pre-HEMT assessment followed by 1-year and 2-year post-HEMT evaluations. In the control/non-HEMT group, participants will undergo parallel assessments at baseline, 1-year, and 2-year intervals to track the natural progression of CRS and OD without HEMT.

Connect with a study center

  • Children's Hospital Colorado

    Aurora, Colorado 80045
    United States

    Site Not Available

  • Children's Hospital Colorado

    Aurora 5412347, Colorado 5417618 80045
    United States

    Active - Recruiting

  • University of Iowa

    Iowa City, Iowa 52242
    United States

    Site Not Available

  • University of Iowa

    Iowa City 4862034, Iowa 4862182 52242
    United States

    Active - Recruiting

  • University of Kansas Medical Center

    Kansas City, Kansas 66160
    United States

    Site Not Available

  • University of Kansas Medical Center

    Kansas City 4273837, Kansas 4273857 66160
    United States

    Active - Recruiting

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 45229-3039
    United States

    Site Not Available

  • Cincinnati Children's Hospital Medical Center

    Cincinnati 4508722, Ohio 5165418 45229-3039
    United States

    Active - Recruiting

  • University of Vermont

    Colchester, Vermont 05446
    United States

    Site Not Available

  • University of Vermont

    Colchester 5235024, Vermont 5242283 05446
    United States

    Active - Recruiting

  • University of Virginia

    Charlottesville, Virginia 22903
    United States

    Site Not Available

  • University of Virginia

    Charlottesville 4752031, Virginia 6254928 22903
    United States

    Active - Recruiting

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