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.
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
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
University of California, Los Angeles
Lead sponsor
University of Kansas Medical Center
Collaborator
Children's Hospital Medical Center, Cincinnati
Collaborator
University of Iowa
Collaborator
University of Virginia
Collaborator
University of Vermont
Collaborator
University of North Carolina, Chapel Hill
Collaborator
Children's Hospital Colorado
Collaborator