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Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®. — PREDICT-CF  

Cystic Fibrosis Clinical Trial

Official title:
Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®. Pathway to Personalized Therapy in Cystic Fibrosis

Clinical Trial 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.

Clinical Trial Description

Orkambi® is a combination of Ivacaftor (a CFTR channel potentiator) and Lumacaftor (a corrector partially rescuing the traffic of mutated CFTR). This treatment is now marketed in France for patients homozygotes for the mutation p.Phe508del, aged 12 and above. Systematic use of this product is a concern due to the lack of predictive markers of efficacy, the highly variable respiratory improvement in patients and potential serious side effects. The purpose of this study is to investigate the predictive value for improvement of the respiratory function after 24 weeks of Orkambi treatment of an in vitro test. This test quantifies the correction of CFTR activity as assessed by the change of cyclic AMP (cAMP) dependant chloride (Cl-) secretion in patient derived Human Nasal Epithelial (HNE) derived primary culture after Lumacaftor/Ivacaftor 48 hours incubation. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03894657
Study type Interventional
Source Assistance Publique - Hôpitaux de Paris
Contact
Status Completed
Phase N/A
Start date December 18, 2019
Completion date May 12, 2022
View Details
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