Testing Drug Efficacy in Cystic Fibrosis Through N-of-1 Trials
Status:
Not yet recruiting
Trial end date:
2027-01-31
Target enrollment:
Participant gender:
Summary
The purpose of this study is to validate and utilize a personalized medicine approach to
identify potential treatments with current FDA approved CFTR modifiers for non-approved CF
gene mutations. The study will perform ex vivo testing of CFTR function and current marketed
CFTR modulating drugs on expanded nasal cells at Cincinnati Children's Human Nasal Epithelium
(HNE) Core Laboratory. The results will be confirmed and translated into bedside care through
an N of 1 trial to determine effectiveness of treatment.