Sinus Disease in Young Children With Cystic Fibrosis

NCT06191640 | Recruiting

• 1 other identifier: 22-000594
• Study Type: observational
• Target: 80
• Locations: 1 country
• Sites: 6

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

Keywords

Cystic Fibrosis; Chronic Rhinosinusitis; Olfactory Dysfunction

Outcome Measures

Primary Outcomes (2)

• Change in MRI sinus opacification

  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

  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 Outcomes (5)

• Change in Pediatric Smell Wheel (PSW) Scores

  Baseline, 1-year, and 2-year follow-up

• Olfactory Cleft Opacification

  Baseline, 1-year, and 2-year follow-up

• Change in Brief Questionnaire of Olfactory Disorders (BQOD) Scores

  Baseline, 1-year, and 2-year follow-up

• Change in Sinus and Nasal Quality of Life Survey (SN-5) - Symptom Frequency Domains

  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

  Baseline, 1-year, and 2-year follow-up

Study Arms (2)

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

Non-HEMT/Control Group

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

Interventions

Ivacaftor or elexacaftor/tezacaftor/ivacaftor DRUG

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

Also known as: Kalydeco or Trikafta; Vertex (VX)-770 or VX-445/VX-661/VX-770

HEMT Group

Eligibility Criteria

• Age: 2 Years - 8 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of California, Los Angeles: lead
• University of Kansas Medical Center: collaborator
• Children's Hospital Medical Center, Cincinnati: collaborator
• University of Iowa: collaborator
• University of Virginia: collaborator
• University of Vermont: collaborator
• University of North Carolina, Chapel Hill: collaborator
• Children's Hospital Colorado: collaborator

Study Sites (6)

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

RECRUITING

University of Iowa

Iowa City, Iowa, 52242, United States

RECRUITING

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229-3039, United States

RECRUITING

University of Vermont

Colchester, Vermont, 05446, United States

RECRUITING

University of Virginia

Charlottesville, Virginia, 22903, United States

RECRUITING

Related Publications (1)

• Liu CM, Fischer JL, Zemanick ET, Woods JC, Markarian KK, Fain SB, Froh D, Heltshe SL, Hoffman LR, Humphries SM, Kramer EL, Ode KL, Lewis M, Li DA, Mata J, Milla SS, Niedbalski PJ, Sawatzky BD, Sim MS, Sullivan JS, Trout AT, Goss CH, Taylor-Cousar JL, Beswick DM. The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol. ERJ Open Res. 2025 Jan 13;11(1):00137-2024. doi: 10.1183/23120541.00137-2024. eCollection 2025 Jan.

  PMID: 39811548 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis; Disease; Olfaction Disorders

Interventions

elexacaftor, ivacaftor, tezacaftor drug combination; ivacaftor

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Sensation Disorders; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms

Study Officials

• Daniel M Beswick, MD

  University of California, Los Angeles

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Daniel M Beswick, MD

CONTACT

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

Marlene Florian

CONTACT

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

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD; Associate Professor-in-Residence; Associate Residency Program Director; Co-Chair, Equity, Diversity, and Inclusion Committee Department of Head and Neck Surgery

Study Record Dates

• First Submitted: December 19, 2023
• First Posted: January 5, 2024
• Study Start: April 12, 2023
• Primary Completion (Estimated): June 30, 2029
• Study Completion (Estimated): June 30, 2029
• Last Updated: May 11, 2026

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)