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Recruiting NCT06191640

Sinus Disease in Young Children With Cystic Fibrosis

— Observational Cystic Fibrosis in Children Cystic Fibrosis Chronic Rhinosinusitis (Diagnosis) Olfactory Disorder

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Ivacaftor or elexacaftor/tezacaftor/ivacaftor.
Who it may be relevant to
Registry conditions: Cystic Fibrosis in Children, Cystic Fibrosis, Chronic Rhinosinusitis (Diagnosis), Olfactory Disorder. Basic parameters: 2 years — 8 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Overview

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.

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.

Interventions

  • 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.

Primary outcome measures

  • 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]
Secondary outcome measures (5)
  • Change in Pediatric Smell Wheel (PSW) Scores [Time frame: Baseline, 1-year, and 2-year follow-up]
  • Olfactory Cleft Opacification [Time frame: 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]
  • Change in Sinus and Nasal Quality of Life Survey (SN-5) - Symptom Frequency Domains [Time frame: 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]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

United States · 6 centers
  • Children's Hospital Colorado — Aurora
  • University of Iowa — Iowa City
  • University of Kansas Medical Center — Kansas City
  • Cincinnati Children's Hospital Medical Center — Cincinnati
  • University of Vermont — Colchester
  • University of Virginia — Charlottesville

Publications

  • 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 pr PMID 39811548

Identifiers

NCT: NCT06191640 · 22-000594

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗