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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

91 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-12-18

Study Completion Date

2022-05-12

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Cystic Fibrosis Homozygous F508del Mutation

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

OTHER

Blinding Strategy

NONE

Interventions

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Nasal brushing

Nasal scrapping at visit V0

Intervention Type DIAGNOSTIC_TEST

Sputum sample

Visit V0 : 1 Aliquot for Sputum biobank

Visit V1 : 2 Aliquots

* for Sputum biobank
* for pharmacokinetic (PK) study

Visit V2 : 2 Aliquots

* for Sputum biobank
* for pharmacokinetic (PK) study

Intervention Type OTHER

blood sample

Visit V0 : Additional 14 mL

* 5 mL in ethylenediaminetetraacetic acid (EDTA) tube for DNA Analysis
* 3 mL in Dry tube for Serum biobank
* 6 mL in acid citrate dextrose (ACD) tube for peripheral blood mononuclear cell (PBMC) biobank

Visit V1 : Additional 9 mL

* 2x3 mL in dry tube for pharmacokinetic (PK) study
* 3 mL in Dry tube for Serum biobank

Visit V2 : Additional 6 mL

* 3 mL in dry tube for pharmacokinetic (PK) study
* 3 mL in Dry tube for Serum biobank

Intervention Type OTHER

Orkambi

Study the predictive value for improvement of the respiratory function after 24 weeks of Orkambi treatment.

Orkambi treatment is part of usual care.

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No