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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
Sponsor:
Information provided by (Responsible Party):
University Hospital, Montpellier

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.

Condition or disease
Cystic Fibrosis

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.


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Study Type : Observational
Actual Enrollment : 6 participants
Observational Model: Cohort
Time Perspective: Prospective
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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

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



Primary Outcome Measures :
  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 Outcome Measures :
  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.


Biospecimen Retention:   Samples With DNA
Blood sample, RNA and induced pluripotent stem cell lines.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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.
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03754088


Locations
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France
Hôpital Arnaud de Villeneuve - CHU de Montpellier
Montpellier, France, 34295
Sponsors and Collaborators
University Hospital, Montpellier
Investigators
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Principal Investigator: Arnaud Bourdin, MD, PhD Montpellier University Hospitals

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Responsible Party: University Hospital, Montpellier
ClinicalTrials.gov Identifier: NCT03754088     History of Changes
Other Study ID Numbers: RECHMPL17_0254
2017-A02341-52 ( Other Identifier: RCB number )
First Posted: November 27, 2018    Key Record Dates
Last Update Posted: June 17, 2019
Last Verified: June 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases