The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol.
Christine M Liu, Jakob L Fischer, Edith T Zemanick...
https://pubmed.ncbi.nlm.nih.gov/39811548Actively Recruiting
Age: 2Years - 8Years
All Genders
ID06191640
Sinus Disease in Young Children With Cystic Fibrosis Observational Study Evaluating Highly Effective CFTR Modulator Therapy
Led by University of California, Los Angeles · Updated on 2026-05-11
80
Participants Needed
6
Research Sites
N/A
Total Duration
On this page
Sponsors
U
University of California, Los Angeles
Lead Sponsor
U
University of Kansas Medical Center
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are investigating how highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators affect chronic rhinosinusitis (CRS) and problems with the sense of smell (olfactory dysfunction) in young children with cystic fibrosis. This observational study includes two groups of children aged 2 to 8 years: those receiving highly effective modulator therapy (HEMT) and a control group not receiving HEMT. The goal is to understand if early use of HEMT improves sinus health and smell function over two years. Children in the HEMT group will have assessments before starting the modulator therapy and then again at one year and two years after starting treatment. Meanwhile, the control group will have similar assessments at baseline, one year, and two years without HEMT to observe the natural progression of their condition. The therapy studied includes ivacaftor or elexacaftor/tezacaftor/ivacaftor, prescribed by their doctors. Participants will undergo sinus MRI scans to measure sinus opacification and olfactory bulb volume, as well as objective smell tests and quality of life surveys at each visit. These evaluations will help track changes in sinus disease and olfactory function over time. The study is led by the University of California, Los Angeles and will continue through June 2029.
CONDITIONS
Brief Title
Sinus Disease in Young Children With Cystic Fibrosis
Who Can Participate
Age: 2Years - 8Years
All Genders
Eligibility Criteria
You may qualify if you...
- Children aged 2 to 8 years at first study visit
- Documented diagnosis of cystic fibrosis
- For HEMT group: CFTR mutation eligible for ivacaftor or elexacaftor/tezacaftor/ivacaftor and clinician plans to start therapy
- For Non-HEMT group: ineligible for HEMT based on mutation or clinical decision not to start treatment
You will not qualify if you...
- Use of investigational drugs within 28 days prior to first study visit
- Use of ivacaftor or elexacaftor/tezacaftor/ivacaftor within 180 days prior to and including first study visit
- Use of chronic oral corticosteroids within 28 days prior to and including first study visit
- Sinus surgery within 180 days prior to first study visit
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Monitoring
Duration - 2 years
Participants undergo assessments to track sinus health and olfactory function over time.
3 visits at baseline, 1 year, and 2 years
Trial Site Locations
Total: 6 locations
1
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
2
University of Iowa
Iowa City, Iowa, United States, 52242
Actively Recruiting
3
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
Actively Recruiting
4
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229-3039
Actively Recruiting
5
University of Vermont
Colchester, Vermont, United States, 05446
Actively Recruiting
6
University of Virginia
Charlottesville, Virginia, United States, 22903
Actively Recruiting
Loading map...
Research Team
D
Daniel M Beswick, MD
M
Marlene Florian
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here