Recruiting

Cystic Fibrosis

Sponsor:

UCLA

Code:

NCT06191640

Conditions

Cystic Fibrosis in Children

Cystic Fibrosis

Chronic Rhinosinusitis (Diagnosis)

Olfactory Disorder

Olfactory Impairment

Eligibility Criteria

Sex: All

Age: 2 - 8

Healthy Volunteers: Not accepted

Interventions

Ivacaftor or elexacaftor/tezacaftor/ivacaftor

Study Details

Brief 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.

Conditions

Cystic Fibrosis in Children

Cystic Fibrosis

Chronic Rhinosinusitis (Diagnosis)

Olfactory Disorder

Olfactory Impairment

Study ID

NCT06191640

Start date

Apr 12, 2023

Status verified date

Aug, 2025

Completion date

Jun 30, 2029

Anticipated

Primary completion date

Jun 30, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 8

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

80 participants

Anticipated

Interventions and Outcome Measures

Arms

HEMT Group

Children with CF planning to start ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy. Participants from the non-HEMT group of this study may enroll into the HEMT cohort if they become eligible for these CFTR modulator therapies and plan to start them.

Non-HEMT/Control Group

Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy.

Interventions

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.

Primary outcome measure

  • Change in MRI sinus opacification [ Time Frame: Baseline, 1-year, and 2-year follow-up ]
  • Change in olfactory bulb volume [ Time Frame: Baseline, 1-year, and 2-year follow-up ]

Central Contacts and Locations

Central contacts

Daniel M Beswick, MD

310-206-8457dbeswick@mednet.ucla.edu

Marlene Florian

424-946-5862mflorian@mednet.ucla.edu

Locations

Children's Hospital Colorado

Recruiting

Aurora, Colorado, United States, 80045

University of Iowa

Recruiting

Iowa City, Iowa, United States, 52242

University of Kansas Medical Center

Recruiting

Kansas City, Kansas, United States, 66160

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati, Ohio, United States, 45229-3039

University of Vermont

Recruiting

Colchester, Vermont, United States, 05446

University of Virginia

Recruiting

Charlottesville, Virginia, United States, 22903

More Information

Sponsor

University of California, Los Angeles

Last update posted

May 11, 2026

Last verified

Aug, 2025

Keywords

  • Cystic Fibrosis
  • Chronic Rhinosinusitis
  • Olfactory Dysfunction

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by University of California, Los Angeles on 2026-05-11.