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

Initiation of an in Vitro Model of the Cystic Fibrosis Bronchial Epithelium Via Induced Pluripotent Stem Cell Technology

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT03754088

Acronym

PaCyFIC

Enrollment

Registered

2018-11-27

Start date

2018-11-05

Completion date

2019-04-25

Last updated

2022-09-26

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cystic Fibrosis

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Yes

Inclusion criteria

for subjects with Cystic Fibrosis: * Homozygote for the p.Phe508del mutation * Signed informed consent given by the subject Inclusion Criteria for subjects without Cystic Fibrosis: * Signed informed consent given by the subject

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

Design outcomes

Primary

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

Secondary

Measure	Time frame	Description
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.
Functional bronchial epithelium present for the iPS? yes/no	28 days	Was a functional bronchial epithelium present for the iPS? yes/no

Countries

France

Outcome results