NCT03652090

Primary Nasal Cell Culture as a Tool for Personalized Therapy in Cystic Fibrosis

Evaluation of the Primary Human Nasal Epithelial Cell Culture Model in the Context of Personalized Therapy in Cystic Fibrosis

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 112 (actual)
• Sponsor: Institut National de la Santé Et de la Recherche Médicale, France · Other Government
• Sex: All
• Age: —
• Healthy volunteers: —

Summary

characterization of CFTR function and expression in nasal primary cells collected from patients with cystic fibrosis in comparison to their parents, healthy heterozygotes and healthy controls

Detailed description

3 groups of subjects are enrolled CF subjects according to their genotypes (aiming to enroll patients carrying 2 CF causing mutations with no CFTR expression/function, and patients carrying at least 1 mutation with residual function, such R117H) Parents or siblings of the CF subjects, as healthy hétérozygotes healthy controls All these subjects experience nasal brushings. From these nasal brushings,nasal cells are expanded, and cultured in air liquid interface to obtain polarized epithelium. This epithelium is then studied in Ussing chamber experiments to characterize the level of cAMP dependant Chloride transport and Sodium reabsorption. Apical expression of CFTR is assessed by immunofluorescence. Results will allow to define the variability of CFTR function and expression criteria in subjects with the same genotype. Such data are crucial for interpretation of the effect of CFTR modulators.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
PROCEDURE	cell sampling	nasal brushing to collect cells

Timeline

• Start date: 2010-09-01
• Primary completion: 2016-03-03
• Completion: 2016-03-03
• First posted: 2018-08-29
• Last updated: 2019-03-12