<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/NCT02194881</url>
  </required_header>
  <id_info>
    <org_study_id>CCTIRS 13.652</org_study_id>
    <nct_id>NCT02194881</nct_id>
  </id_info>
  <brief_title>Ivacaftor in French Patients With Cystic Fibrosis and a G551D Mutation</brief_title>
  <acronym>IVACAFTOR1</acronym>
  <official_title>Ivacaftor in French Patients With Cystic Fibrosis and a G551D Mutation - Efficacy and Safety Results After the First Year of Treatment in the Real Life Setting.</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Assistance Publique - Hôpitaux de Paris</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Vaincre la Mucoviscidose</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Assistance Publique - Hôpitaux de Paris</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to determine whether the treatment with Ivacaftor remains&#xD;
      effective and safe in the patients with cystic fibrosis (and at least one G551D CFTR&#xD;
      mutation) in the real life setting, after the drug has been approved by the Health&#xD;
      authorities.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The aims of our study are:&#xD;
&#xD;
        1. to describe the treated population at initiation of treatment,&#xD;
&#xD;
        2. to evaluate clinical parameters during the year before Ivacaftor was started, at&#xD;
           initiation of treatment and during at least one year of treatment, until June 2014.&#xD;
&#xD;
        3. to evaluate the tolerance and safety of this treatment.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>October 2014</start_date>
  <completion_date type="Actual">May 2016</completion_date>
  <primary_completion_date type="Actual">February 2016</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>Retrospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>FEV1 (in liters and in % predicted)</measure>
    <time_frame>until one years after initiation of treatment</time_frame>
    <description>pulmonary function</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>pulmonary exacerbations</measure>
    <time_frame>until one years after initiation of treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>number of hospitalizations and number of days of hospitalization per year</measure>
    <time_frame>until one year after initiation of treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>number of antibiotic treatments and number of days of antibiotic treatments</measure>
    <time_frame>until one year after initiation of treatment</time_frame>
    <description>number of oral antibiotic treatments and number of days of oral antibiotic treatments, number of IV courses and days of IV antibiotics per year</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>respiratory colonization</measure>
    <time_frame>until least one year after initiation of treatment</time_frame>
    <description>Evolution of bacteria and fungi in sputum</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>nutritional status</measure>
    <time_frame>until one year after initiation of treatment</time_frame>
    <description>Weight (and BMI-Zscore for children)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Adverse events</measure>
    <time_frame>until one year after initiation of treatment</time_frame>
    <description>Dates and reasons for interruption and discontinuation of treatment with Ivacaftor Adverse events, indicating what in the physician's opinion might be due to Ivacaftor</description>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">57</enrollment>
  <condition>Cystic Fibrosis</condition>
  <arm_group>
    <arm_group_label>Ivacaftor 1</arm_group_label>
    <description>patients with CF who are homozygous or heterozygous for the G551D mutation and treated with Ivacaftor</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>CF patients with a G551D mutation and treated with Ivacaftor</intervention_name>
    <arm_group_label>Ivacaftor 1</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Patients with CF aged 6 or older and cared in French CF centres&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  French patients with CF aged 6 or older who are homozygous or heterozygous for the&#xD;
             G551D mutation&#xD;
&#xD;
          -  Treated with Ivacaftor&#xD;
&#xD;
          -  First prescription of Ivacaftor before June 1st 2013 (including patients randomized in&#xD;
             the VX770 clinical trials)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  CF patients younger than 6 years old&#xD;
&#xD;
          -  CF patients who have received lung transplantation&#xD;
&#xD;
          -  CF patients without a G551D mutation.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Dominique Hubert, MD</last_name>
    <role>Study Chair</role>
    <affiliation>Assistance Publique - Hôpitaux de Paris</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Hôpital Cochin</name>
      <address>
        <city>Paris</city>
        <zip>75014</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
  </location_countries>
  <verification_date>July 2016</verification_date>
  <study_first_submitted>July 17, 2014</study_first_submitted>
  <study_first_submitted_qc>July 17, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">July 18, 2014</study_first_posted>
  <last_update_submitted>July 31, 2017</last_update_submitted>
  <last_update_submitted_qc>July 31, 2017</last_update_submitted_qc>
  <last_update_posted type="Actual">August 1, 2017</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Cystic fibrosis</keyword>
  <keyword>CFTR</keyword>
  <keyword>G551D mutation</keyword>
  <keyword>Novel treatments</keyword>
  <keyword>Potentiator</keyword>
  <keyword>Ivacaftor</keyword>
  <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>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Ivacaftor</mesh_term>
  </intervention_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/NCT02194881
org study id: CCTIRS 13.652
nct id: NCT02194881
lead sponsor: 
collaborator: 
has dmc: No
The purpose of this study is to determine whether the treatment with Ivacaftor remains
      effective and safe in the patients with cystic fibrosis (and at least one G551D CFTR
      mutation) in the real life setting, after the drug has been approved by the Health
      authorities.
The aims of our study are:

        1. to describe the treated population at initiation of treatment,

        2. to evaluate clinical parameters during the year before Ivacaftor was started, at
           initiation of treatment and during at least one year of treatment, until June 2014.

        3. to evaluate the tolerance and safety of this treatment.
observational model: Cohort
time perspective: Retrospective
measure: FEV1 (in liters and in % predicted)
time frame: until one years after initiation of treatment
description: pulmonary function
measure: pulmonary exacerbations
time frame: until one years after initiation of treatment
measure: number of hospitalizations and number of days of hospitalization per year
time frame: until one year after initiation of treatment
measure: number of antibiotic treatments and number of days of antibiotic treatments
time frame: until one year after initiation of treatment
description: number of oral antibiotic treatments and number of days of oral antibiotic treatments, number of IV courses and days of IV antibiotics per year
measure: respiratory colonization
time frame: until least one year after initiation of treatment
description: Evolution of bacteria and fungi in sputum
measure: nutritional status
time frame: until one year after initiation of treatment
description: Weight (and BMI-Zscore for children)
measure: Adverse events
time frame: until one year after initiation of treatment
description: Dates and reasons for interruption and discontinuation of treatment with Ivacaftor Adverse events, indicating what in the physician's opinion might be due to Ivacaftor
arm group label: Ivacaftor 1
description: patients with CF who are homozygous or heterozygous for the G551D mutation and treated with Ivacaftor
intervention type: Drug
intervention name: CF patients with a G551D mutation and treated with Ivacaftor
arm group label: Ivacaftor 1
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 6 Years
maximum age: N/A
healthy volunteers: No
last name: Dominique Hubert, MD
role: Study Chair
affiliation: Assistance Publique - Hôpitaux de Paris
facility: 
country: France
responsible party type: Sponsor
mesh term: Cystic Fibrosis
mesh term: Fibrosis
mesh term: Ivacaftor