<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT03754088</url>
  </required_header>
  <id_info>
    <org_study_id>RECHMPL17_0254</org_study_id>
    <secondary_id>2017-A02341-52</secondary_id>
    <nct_id>NCT03754088</nct_id>
  </id_info>
  <brief_title>In Vitro Model of the Cystic Fibrosis Bronchial Epithelium Via iPS Technology</brief_title>
  <acronym>PaCyFIC</acronym>
  <official_title>Initiation of an in Vitro Model of the Cystic Fibrosis Bronchial Epithelium Via Induced Pluripotent Stem Cell Technology</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>University Hospital, Montpellier</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>University Hospital, Montpellier</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      In order to further enable physiopathology studies, the aim of this project is to validate an&#xD;
      in vitro model of cystic fibrosis created using induced pluripotent stemcell (iPS)&#xD;
      differentiated bronchial epithelium from cystic fibrosis (CF) patients homozygous for the&#xD;
      p.Phe508del mutation of the cystic fibrosis transmembrane conductance regulator (CFTR) gene.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The investigator's primary objective is to generate iPS lines from 3 CF patients and from 3&#xD;
      healthy subjects.&#xD;
&#xD;
      Secondary objectives include verification that cell lines express the CFTR gene according to&#xD;
      their genotype, verification or relative production of the CFTR protein for each iPS line,&#xD;
      and amplification of obtain iPS lines for aliquot creation to facilitate sharing.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">November 5, 2018</start_date>
  <completion_date type="Actual">April 25, 2019</completion_date>
  <primary_completion_date type="Actual">April 25, 2019</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Obtention of induced pluripotent stem cell line (iPS): yes/no</measure>
    <time_frame>28 days</time_frame>
    <description>Was a pluripotent stem cell line obtained? yes/no</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Functional bronchial epithelium present for the iPS? yes/no</measure>
    <time_frame>28 days</time_frame>
    <description>Was a functional bronchial epithelium present for the iPS? yes/no</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Cystic fibrosis transmembrane conductance regulator (CFTR) channel function: yes/no</measure>
    <time_frame>28 days</time_frame>
    <description>CFTR channel function demonstrated via response following exposure to modified oligonucleotides targeting the mutated CFTR transcript.</description>
  </secondary_outcome>
  <number_of_groups>2</number_of_groups>
  <enrollment type="Actual">6</enrollment>
  <condition>Cystic Fibrosis</condition>
  <arm_group>
    <arm_group_label>Cystic fibrosis</arm_group_label>
    <description>Three cystic fibrosis patients who are homozygous for the p.Phe508del mutation.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Healthy subjects</arm_group_label>
    <description>Three healthy subjects.</description>
  </arm_group>
  <biospec_retention>Samples With DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      Blood sample, RNA and induced pluripotent stem cell lines.&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Three cystic fibrosis patients who are homozygous for the p.Phe508del mutation and 3&#xD;
        healthy subjects without cystic fibrosis.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria for subjects with Cystic Fibrosis:&#xD;
&#xD;
          -  Homozygote for the p.Phe508del mutation&#xD;
&#xD;
          -  Signed informed consent given by the subject&#xD;
&#xD;
        Inclusion Criteria for subjects without Cystic Fibrosis:&#xD;
&#xD;
          -  Signed informed consent given by the subject&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Pregnancy, breastfeeding&#xD;
&#xD;
          -  Participant in an exclusion period determined by a previous study&#xD;
&#xD;
          -  Participant under any kind of guardianship&#xD;
&#xD;
          -  Unaffiliated with or not a beneficiary of a social security program (health insurance)&#xD;
&#xD;
          -  Subject deprived of liberty (e.g. prisoners)&#xD;
&#xD;
          -  Subject with positive infectious markers for HIV1, HIV2, HBC or HBV&#xD;
&#xD;
        Exclusion Criteria for subjects with Cystic Fibrosis:&#xD;
&#xD;
          -  Any pathology requiring a treatment or a pathology not requiring treatment but with&#xD;
             clinical significance according to the investigator&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Arnaud Bourdin, MD, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Montpellier University Hospitals</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Hôpital Arnaud de Villeneuve - CHU de Montpellier</name>
      <address>
        <city>Montpellier</city>
        <zip>34295</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
  </location_countries>
  <verification_date>December 2021</verification_date>
  <study_first_submitted>November 22, 2018</study_first_submitted>
  <study_first_submitted_qc>November 26, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">November 27, 2018</study_first_posted>
  <last_update_submitted>September 23, 2022</last_update_submitted>
  <last_update_submitted_qc>September 23, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">September 26, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cystic Fibrosis</mesh_term>
    <mesh_term>Fibrosis</mesh_term>
  </condition_browse>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03754088
org study id: RECHMPL17_0254
secondary id: 2017-A02341-52
nct id: NCT03754088
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
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.
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.
observational model: Cohort
time perspective: Prospective
measure: Obtention of induced pluripotent stem cell line (iPS): yes/no
time frame: 28 days
description: Was a pluripotent stem cell line obtained? yes/no
measure: Functional bronchial epithelium present for the iPS? yes/no
time frame: 28 days
description: Was a functional bronchial epithelium present for the iPS? yes/no
measure: Cystic fibrosis transmembrane conductance regulator (CFTR) channel function: yes/no
time frame: 28 days
description: CFTR channel function demonstrated via response following exposure to modified oligonucleotides targeting the mutated CFTR transcript.
arm group label: Cystic fibrosis
description: Three cystic fibrosis patients who are homozygous for the p.Phe508del mutation.
arm group label: Healthy subjects
description: Three healthy subjects.
Blood sample, RNA and induced pluripotent stem cell lines.
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: Accepts Healthy Volunteers
last name: Arnaud Bourdin, MD, PhD
role: Principal Investigator
affiliation: Montpellier University Hospitals
facility: 
country: France
responsible party type: Sponsor
mesh term: Cystic Fibrosis
mesh term: Fibrosis