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

NCT03894657 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 91

Last updated 2026-03-10

No results posted yet for this study

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.

Conditions

Interventions

DIAGNOSTIC_TEST

Nasal brushing

Nasal scrapping at visit V0

OTHER

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

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

DRUG

Orkambi

Study the predictive value for improvement of the respiratory function after 24 weeks of Orkambi treatment. Orkambi treatment is part of usual care.

Sponsors & Collaborators

  • URC-CIC Paris Descartes Necker Cochin

    collaborator OTHER

  • Assistance Publique - Hôpitaux de Paris

    lead OTHER

Principal Investigators

  • ISABELLE SERMET, PhD · Hospital Necker Enfants Malades

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-12-18
Primary Completion
2021-11-25
Completion
2022-05-12

Countries

  • France

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03894657 on ClinicalTrials.gov