Sinus Disease in Young Children With Cystic Fibrosis

Launched by UNIVERSITY OF CALIFORNIA, LOS ANGELES · Dec 19, 2023

Keywords

ClinConnect Summary

This clinical trial is studying how a new type of treatment called highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators affects young children with cystic fibrosis (CF) who also have chronic sinus issues and smell problems. The study involves two groups of children aged 2 to 8 years. One group will receive this new treatment, while the other group will not be on this treatment. Researchers will use special scans of the sinuses (MRI), tests to check how well kids can smell, and surveys about their quality of life over a two-year period to see how the treatment helps.

To be eligible for the study, children must have a documented diagnosis of cystic fibrosis and be between 2 and 8 years old. The first group must be starting the new treatment, while the second group cannot be on it either due to their specific genetic type or a doctor's decision. Participants will have regular check-ups and tests to monitor their progress, and this is a great opportunity to contribute to research that may improve treatments for cystic fibrosis in the future.

Gender

ALL

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