Impact of CFTR Modulators on Sinus Health in Young Children with Cystic Fibrosis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study examines how highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators affect chronic rhinosinusitis and olfactory dysfunction in children aged 2-8 with cystic fibrosis. It includes two groups: one receiving modulator therapy and a control group not receiving it. Over two years, participants will undergo MRI scans, olfactory tests, and quality of life surveys to assess the impact of treatment on sinus health and smell capabilities. The study aims to clarify the benefits of early modulator therapy in this young population.

Why it matters

Who can join: the full rules

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

Where it's running

• Children's Hospital Colorado
  Aurora, Colorado, United States Recruiting
• University of Iowa
  Iowa City, Iowa, United States Recruiting
• University of Kansas Medical Center
  Kansas City, Kansas, United States Recruiting
• Cincinnati Children's Hospital Medical Center
  Cincinnati, Ohio, United States Recruiting
• University of Vermont
  Colchester, Vermont, United States Recruiting
• University of Virginia
  Charlottesville, Virginia, United States Recruiting

Study contacts

• Principal investigator: Daniel M Beswick, MD — University of California, Los Angeles
• Study coordinator: Daniel M Beswick, MD
• Email: dbeswick@mednet.ucla.edu
• Phone: 310-206-8457

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.