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

Cystic Fibrosis Clinical Trial

— PaCyFIC  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03754088
• Other study ID #: RECHMPL17_0254
• Secondary ID: 2017-A02341-52
• Status: Completed
• Start date: November 5, 2018
• Est. completion date: April 25, 2019

Study information

• Verified date: December 2021
• Source: University Hospital, Montpellier
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 6
• Est. completion date: April 25, 2019
• Est. primary completion date: April 25, 2019
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Locations

Country	Name	City	State
France	Hôpital Arnaud de Villeneuve - CHU de Montpellier	Montpellier

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Montpellier

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Obtention of induced pluripotent stem cell line (iPS): yes/no	Was a pluripotent stem cell line obtained? yes/no	28 days
Secondary	Functional bronchial epithelium present for the iPS? yes/no	Was a functional bronchial epithelium present for the iPS? yes/no	28 days
Secondary	Cystic fibrosis transmembrane conductance regulator (CFTR) channel function: yes/no	CFTR channel function demonstrated via response following exposure to modified oligonucleotides targeting the mutated CFTR transcript.	28 days