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

Cystic Fibrosis Clinical Trial

— Epiithelix  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03652090
• Other study ID #: 2010-05-03 A3
• Status: Completed
• Start date: September 1, 2010
• Est. completion date: March 3, 2016

Study information

• Verified date: March 2019
• Source: Institut National de la Santé Et de la Recherche Médicale, France
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 112
• Est. completion date: March 3, 2016
• Est. primary completion date: March 3, 2016
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• patients with Cystic Fibrosis with 2 mutations in CFTR

• healthy heterozygotes with 1 mutation in CFTR

• healthy subjects with no familial history of Cystic Fibrosis and no symptoms suggesting Cystic Fibrosis

Exclusion Criteria:

• smoking

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Procedure:
• cell sampling
nasal brushing to collect cells

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Institut National de la Santé Et de la Recherche Médicale, France	ABCF2

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	variation in the short-circuit-current (Isc) after Forskolin (Forskolin)/IBMx and VX-770 (?IscFsk/IBMx+VX-770)	The short-circuit-current (Isc) was measured under voltage clamp conditions. Inhibitors and activators were added after stabilization of baseline Isc. The sum of the change after Forskolin (Forskolin)/IBMx and VX-770 (?IscFsk/IBMx+VX-770) served as an index of CFTR function.	1 day
Secondary	percentage of cells displaying apical staining	CFTR immuno-detection was performed as previously described 31. Apical CFTR staining was assessed semi quantitatively as the percentage of cells displaying apical staining multiplied by the average corrected apical fluorescence 32.	1 day