Testing Drug Efficacy in Cystic Fibrosis Through N-of-1 Trials
Children's Hospital Medical Center, Cincinnati
50 participants
May 1, 2021
INTERVENTIONAL
Conditions
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.
Eligibility
Plain Language Summary
Simplified for easier understanding
This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.
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Interventions
Participants with rare mutations will receive active therapy in N-of-1 design with participants serving as their own control
Locations(1)
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NCT04580368