Study of Sinus Disease and Smell Problems in Children Ages 2 to 8 With Cystic Fibrosis Taking or Not Taking Highly Effective Modulator Therapy

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 elexacaftortezacaftorivacaftor, 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.

Sinus Disease in Young Children With Cystic Fibrosis