Testing Drug Efficacy in Cystic Fibrosis Through N-of-1 Trials

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.

Timeline

Start

2021-05-01

Primary completion

2028-12-31

Completion

2030-01-31

Drugs

Indications