In Vitro Model of the Cystic Fibrosis Bronchial Epithelium Via iPS Technology (PaCyFIC)
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|ClinicalTrials.gov Identifier: NCT03754088|
Recruitment Status : Completed
First Posted : November 27, 2018
Last Update Posted : June 17, 2019
|Condition or disease|
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|
|Actual Enrollment :||6 participants|
|Official Title:||Initiation of an in Vitro Model of the Cystic Fibrosis Bronchial Epithelium Via Induced Pluripotent Stem Cell Technology|
|Actual Study Start Date :||February 14, 2019|
|Actual Primary Completion Date :||April 25, 2019|
|Actual Study Completion Date :||April 25, 2019|
Three cystic fibrosis patients who are homozygous for the p.Phe508del mutation.
Three healthy subjects.
- Obtention of induced pluripotent stem cell line (iPS): yes/no [ Time Frame: 28 days ]Was a pluripotent stem cell line obtained? yes/no
- Functional bronchial epithelium present for the iPS? yes/no [ Time Frame: 28 days ]Was a functional bronchial epithelium present for the iPS? yes/no
- 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.
Biospecimen Retention: Samples With DNA
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Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03754088
|Hôpital Arnaud de Villeneuve - CHU de Montpellier|
|Montpellier, France, 34295|
|Principal Investigator:||Arnaud Bourdin, MD, PhD||Montpellier University Hospitals|