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OpenTrials
Completed

NCT Number: NCT03754088

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

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Key information

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Hôpital Arnaud de Villeneuve - CHU de Montpellier

Montpellier, 34295, France

About this study

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.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. Obtention of induced pluripotent stem cell line (iPS): yes/no

    Time frame: 28 days

    Was a pluripotent stem cell line obtained? yes/no

Secondary outcomes

  1. Functional bronchial epithelium present for the iPS? yes/no

    Time frame: 28 days

    Was a functional bronchial epithelium present for the iPS? yes/no

  2. 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.

Sponsors and collaborators

Lead sponsor

University Hospital, Montpellier

Other

Registry information

Official study title

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

Acronym: PaCyFIC

Important dates

Study start
2018
Primary completion
2019
Study completion
2019
First posted
Nov 27, 2018
Registry last updated
Sep 26, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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