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

September 23, 2022 updated by: University Hospital, Montpellier

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

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.

Study Overview

Status

Completed

Conditions

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 Type

Observational

Enrollment (Actual)

6

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Montpellier, France, 34295
        • Hôpital Arnaud de Villeneuve - CHU de Montpellier

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

Yes

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Three cystic fibrosis patients who are homozygous for the p.Phe508del mutation and 3 healthy subjects without cystic fibrosis.

Description

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

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Cystic fibrosis
Three cystic fibrosis patients who are homozygous for the p.Phe508del mutation.
Healthy subjects
Three healthy subjects.

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Principal Investigator: Arnaud Bourdin, MD, PhD, Montpellier University Hospitals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

November 5, 2018

Primary Completion (ACTUAL)

April 25, 2019

Study Completion (ACTUAL)

April 25, 2019

Study Registration Dates

First Submitted

November 22, 2018

First Submitted That Met QC Criteria

November 26, 2018

First Posted (ACTUAL)

November 27, 2018

Study Record Updates

Last Update Posted (ACTUAL)

September 26, 2022

Last Update Submitted That Met QC Criteria

September 23, 2022

Last Verified

December 1, 2021

More Information

Terms related to this study

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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