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

NACompletedINTERVENTIONAL
Enrollment

91

Participants

Timeline

Start Date

December 18, 2019

Primary Completion Date

November 25, 2021

Study Completion Date

May 12, 2022

Conditions
Cystic FibrosisHomozygous F508del Mutation
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."

Trial Locations (1)

75015

Hôpital Necker-Enfants Malades, Paris

All Listed Sponsors
lead

Assistance Publique - Hôpitaux de Paris

OTHER

NCT03894657 - Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®. | Biotech Hunter | Biotech Hunter