In Vitro Model of the Cystic Fibrosis Bronchial Epithelium Via iPS Technology

Completed

• Conditions: Cystic Fibrosis

• Registration Number: NCT03754088
• Lead Sponsor: University Hospital, Montpellier

Brief Summary

In order to further enable physiopathology studies, the aim of this project is to validate an in vitro model of cystic fibrosis created using induced pluripotent stemcell (iPS) differentiated bronchial epithelium from cystic fibrosis (CF) patients homozygous for the p.Phe508del mutation of the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Detailed Description

The investigator's primary objective is to generate iPS lines from 3 CF patients and from 3 healthy subjects.

Secondary objectives include verification that cell lines express the CFTR gene according to their genotype, verification or relative production of the CFTR protein for each iPS line, and amplification of obtain iPS lines for aliquot creation to facilitate sharing.

Study Timeline

• Study Start: 2018-11-05
• Study First Submitted: 2018-11-22
• Study First Submitted QC: 2018-11-26
• Study First Posted: 2018-11-27
• Primary Completion: 2019-04-25
• Study Completion: 2019-04-25
• Status Verified: 2021-12
• Last Update Submitted: 2022-09-23
• Last Update Posted: 2022-09-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

Not provided

Exclusion Criteria

• Pregnancy, breastfeeding
• Participant in an exclusion period determined by a previous study
• Participant under any kind of guardianship
• Unaffiliated with or not a beneficiary of a social security program (health insurance)
• Subject deprived of liberty (e.g. prisoners)
• Subject with positive infectious markers for HIV1, HIV2, HBC or HBV

Exclusion Criteria for subjects with Cystic Fibrosis:

• Any pathology requiring a treatment or a pathology not requiring treatment but with clinical significance according to the investigator

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Obtention of induced pluripotent stem cell line (iPS): yes/no	28 days	Was a pluripotent stem cell line obtained? yes/no

Secondary Outcome Measures

Name	Time	Method
Functional bronchial epithelium present for the iPS? yes/no	28 days	Was a functional bronchial epithelium present for the iPS? yes/no
Cystic fibrosis transmembrane conductance regulator (CFTR) channel function: yes/no	28 days	CFTR channel function demonstrated via response following exposure to modified oligonucleotides targeting the mutated CFTR transcript.

Trial Locations

Locations (1)

Hôpital Arnaud de Villeneuve - CHU de Montpellier; 🇫🇷 Montpellier, France