Impact of CFTR Modulators on Sinus Health in Young Children with Cystic Fibrosis
Sinus Disease in Young Children With Cystic Fibrosis
This study is testing if a new treatment for cystic fibrosis can improve sinus health and smell in young children aged 2-8.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 80 (estimated) |
| Ages | 2 Years to 8 Years |
| Sex | All |
| Sponsor | University of California, Los Angeles Academic / other |
| Locations | 6 sites (Aurora, Colorado and 5 other locations) |
| Trial ID | NCT06191640 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are children aged 2-8 years with a confirmed diagnosis of cystic fibrosis, either receiving or not receiving highly effective modulator therapy.
Not a fit: Patients who have undergone sinus surgery or are using investigational drugs or corticosteroids may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved management of sinus health and olfactory function in young children with cystic fibrosis.
How similar studies have performed: While studies on CFTR modulators have shown promise in other areas, this specific investigation into sinus health and olfactory function in young children is novel.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Aurora, Colorado and 5 other locations
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.