Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®.

• Status: Recruiting
• Trial end date: 2023-02-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

Criteria

Inclusion Criteria:

• Homozygous F508del patient aged 5 years or older

• Patient with an indication for Orkambi® treatment according to the marketing authorization application

• Patient never received Orkambi® in the past

• Patient able to perform FEV1

• Signed Informed consent form by the patient (if aged ≥ 18 years), or by parents / legal guardian and patient's agreement (if aged < 18 years) Patient affiliated to the health insurance system

Exclusion Criteria:

• Homozygous F508del patients who do not meet the treatment indications according to the marketing authorization application

• Patients refusing Orkambi®

• CF patients not homozygous for the p.Phe508del mutation

• Active smoker

• Severe nasal mucosa disrepair

• Contraindications to xylocaine anesthesia,

• Participation with another interventional study with drug