Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®.
Status:
Recruiting
Trial end date:
2023-02-01
Target enrollment:
Participant gender:
Summary
The purpose of the study is to investigate whether the correction of CFTR function by
Lumacaftor/Ivacaftor in a patient-derived primary nasal cell model is a surrogate biomarker
for respiratory improvement in Orkambi® treated patients.