Sinus Disease in Young Children With Cystic Fibrosis

May 6, 2026 updated by: Daniel M. Beswick, University of California, Los Angeles
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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed 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.

Study Type

Observational

Enrollment (Estimated)

80

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • Children's Hospital Colorado
        • Contact:
          • Edith Zemanick, MD
        • Contact:
          • Mary Cross
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Recruiting
        • University of Iowa
        • Contact:
          • Sean B Fain, PhD
        • Contact:
          • Mary Teresi
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • Recruiting
        • University of Kansas Medical Center
        • Contact:
          • Michael Lewis, MD
        • Contact:
          • Larry Scott
    • Ohio
      • Cincinnati, Ohio, United States, 45229-3039
        • Recruiting
        • Cincinnati Children's Hospital Medical Center
        • Contact:
          • Jason Woods, PhD
        • Contact:
          • Priyanka Desirazu
    • Vermont
      • Colchester, Vermont, United States, 05446
        • Recruiting
        • University of Vermont
        • Contact:
          • Jillian S Sullivan, MD
        • Contact:
          • Julie Sweet
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • Recruiting
        • University of Virginia
        • Contact:
          • Jaime Mata, MS, PhD
        • Contact:
          • Roselove N Nunoo-Asare

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Participants for this study will be enrolled into one of two groups. The first group (HEMT Group) will enroll children between 2-8 years of age who have a clinical intent to initiate HEMT, which is defined as ivacaftor or elexacaftor/tezacaftor/ivacaftor based on underlying CFTR mutation status. The second group (non-HEMT Group) will consist of children under the age of 8 without intent to clinically initiate HEMT. Children for both groups will be identified and recruited from cystic fibrosis clinics.

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
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.
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.
Other Names:
  • Kalydeco or Trikafta; Vertex (VX)-770 or VX-445/VX-661/VX-770
Non-HEMT/Control Group
Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in MRI sinus opacification
Time Frame: Baseline, 1-year, and 2-year follow-up
Measurement involves calculating percent total sinus opacification (%) using MRI, where sinus contents are differentiated into air and soft tissue/fluid.
Baseline, 1-year, and 2-year follow-up
Change in olfactory bulb volume
Time Frame: Baseline, 1-year, and 2-year follow-up
Assessment includes segmenting the olfactory bulb on consecutive coronal slices using MRI, then combining these into a 3-dimensional region. The volume of this region is then calculated in cubic millimeters (mm3).
Baseline, 1-year, and 2-year follow-up

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Olfactory Cleft Opacification
Time Frame: Baseline, 1-year, and 2-year follow-up
Olfactory cleft opacification is quantified by segmenting the olfactory cleft on MRI and applying pixel intensity thresholds to calculate the percentage (%) of opacification.
Baseline, 1-year, and 2-year follow-up
Change in Pediatric Smell Wheel (PSW) Scores
Time Frame: Baseline, 1-year, and 2-year follow-up
The PSW test involves children identifying microencapsulated odorants. The score (Range: 0-11) is based on the number of correctly identified smells, with higher scores indicating better olfactory function.
Baseline, 1-year, and 2-year follow-up
Change in Brief Questionnaire of Olfactory Disorders (BQOD) Scores
Time Frame: Baseline, 1-year, and 2-year follow-up
This BQOD is a parent-completed survey for assessing olfactory-specific quality of life (QOL). The survey scores (Range: 0-21) quantify the degree of olfactory QOL impairment, with higher scores indicating greater olfactory QOL impairment.
Baseline, 1-year, and 2-year follow-up
Change in Sinus and Nasal Quality of Life Survey (SN-5) - Symptom Frequency Domains
Time Frame: Baseline, 1-year, and 2-year follow-up
SN-5 is a parent-completed survey assessing sinus symptoms and QOL in children. It captures sinus infections, nasal obstruction, allergy symptoms, emotional distress, activity limitations, and overall QOL. For the first five symptom domain questions, scores range 1-7 for each domain, higher scores indicating higher frequency at which symptom affected patient over the past 4 weeks.
Baseline, 1-year, and 2-year follow-up
Change in Sinus and Nasal Quality of Life Survey (SN-5) - Overall Quality of Life (QOL) Domain
Time Frame: Baseline, 1-year, and 2-year follow-up
SN-5 is a parent-completed survey assessing sinus symptoms and QOL in children. It captures sinus infections, nasal obstruction, allergy symptoms, emotional distress, activity limitations, and overall QOL. The final question assessing the child's overall QOL as a result of nose or sinus problems is scored on a scale 0-10, with higher scores indicating better QOL.
Baseline, 1-year, and 2-year follow-up

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of California, Los Angeles

Collaborators

University of Iowa
Children's Hospital Colorado
University of Kansas Medical Center
University of North Carolina, Chapel Hill
University of Vermont
Children's Hospital Medical Center, Cincinnati
University of Virginia

Investigators

  • Principal Investigator: Daniel M Beswick, MD, University of California, Los Angeles

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 12, 2023

Primary Completion (Estimated)

June 30, 2029

Study Completion (Estimated)

June 30, 2029

Study Registration Dates

First Submitted

December 19, 2023

First Submitted That Met QC Criteria

December 19, 2023

First Posted (Actual)

January 5, 2024

Study Record Updates

Last Update Posted (Actual)

May 11, 2026

Last Update Submitted That Met QC Criteria

May 6, 2026

Last Verified

August 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 22-000594

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Participant data will be securely stored using REDCap software.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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