<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/NCT02981784</url>
  </required_header>
  <id_info>
    <org_study_id>69HCL16_0716</org_study_id>
    <nct_id>NCT02981784</nct_id>
  </id_info>
  <brief_title>Comparative Evaluation of Results of Allogeneic Hematopoietic Stem Cells Versus Ponatinib in CML Patients Carrying a Mutation T315I</brief_title>
  <official_title>Overall Survival With Ponatinib Versus Allogeneic Stem Cell Transplant in Philadelphia-positive Leukemias With the T315I Mutation</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Hospices Civils de Lyon</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Hospices Civils de Lyon</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Effective treatment options for chronic myeloid leukemia (CML) or Philadelphia-positive (Ph+)&#xD;
      acute lymphoblastic leukemia (ALL) patients with the T315I mutation are few. This study&#xD;
      compared overall survival (OS) between CML and Ph+ ALL patients treated with ponatinib versus&#xD;
      allogeneic stem cell transplantation (allo-SCT).&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date>January 2000</start_date>
  <completion_date type="Actual">December 2015</completion_date>
  <primary_completion_date type="Actual">December 2011</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>Overall survival after either allogeneic stem cell transplantation or Ponatinib initiation. (Time between therapeutic intervention and death, in months)</measure>
    <time_frame>From date of inclusion until the date of death, assessed up to 180 months</time_frame>
  </primary_outcome>
  <number_of_groups>2</number_of_groups>
  <enrollment type="Actual">184</enrollment>
  <condition>Leukemia</condition>
  <arm_group>
    <arm_group_label>Ponatinib (PACE trial)</arm_group_label>
    <description>PACE trial : &quot;Ponatinib for Chronic Myeloid Leukemia (CML) Evaluation and Ph+ Acute Lymphoblastic Leukemia (ALL)&quot;, NCT01207440</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Allogenis stem cell transplantation (EBMT registry)</arm_group_label>
    <description>EBMT : European Group for Blood and Marrow Transplantation</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Ponatinib (Iclusig®)</intervention_name>
    <description>Novel tyrosine kinase inhibitor active in patients with an ABL T315I mutation compared to conventional or reduced intensity allogeneic stem cell transplantation.</description>
    <arm_group_label>Ponatinib (PACE trial)</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Procedure</intervention_type>
    <intervention_name>allogeneic stem cell transplantation</intervention_name>
    <arm_group_label>Allogenis stem cell transplantation (EBMT registry)</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        All 128 T315I+ patients from PACE comprised the ponatinib group of this study. Fifty-six&#xD;
        patients from the EBMT database comprised the allo-SCT group&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  CML any phase with T315I mutation&#xD;
&#xD;
          -  Ph+ ALL with a T315I mutation&#xD;
&#xD;
          -  Being treated in the PACE trial according to the criteria of this phase II trial&#xD;
&#xD;
          -  Or being allogeneic stem cell transplanted with any source of allogeneic cells and&#xD;
             after any conditioning regimen.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Ph negative patients&#xD;
&#xD;
          -  Patients under 18 years&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Centre Hospitalier Lyon-Sud</name>
      <address>
        <city>Pierre-Bénite</city>
        <zip>69310</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
  </location_countries>
  <verification_date>November 2016</verification_date>
  <study_first_submitted>November 21, 2016</study_first_submitted>
  <study_first_submitted_qc>December 1, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 5, 2016</study_first_posted>
  <last_update_submitted>December 1, 2016</last_update_submitted>
  <last_update_submitted_qc>December 1, 2016</last_update_submitted_qc>
  <last_update_posted type="Estimate">December 5, 2016</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>chronic myeloid leukemia (CML)</keyword>
  <keyword>Philadelphia-positive (Ph+)</keyword>
  <keyword>Acute Lymphoblastic Leukemia (ALL)</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Leukemia</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Ponatinib</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- 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/NCT02981784
org study id: 69HCL16_0716
nct id: NCT02981784
lead sponsor: 
has dmc: No
Effective treatment options for chronic myeloid leukemia (CML) or Philadelphia-positive (Ph+)
      acute lymphoblastic leukemia (ALL) patients with the T315I mutation are few. This study
      compared overall survival (OS) between CML and Ph+ ALL patients treated with ponatinib versus
      allogeneic stem cell transplantation (allo-SCT).
observational model: Cohort
time perspective: Retrospective
measure: Overall survival after either allogeneic stem cell transplantation or Ponatinib initiation. (Time between therapeutic intervention and death, in months)
time frame: From date of inclusion until the date of death, assessed up to 180 months
arm group label: Ponatinib (PACE trial)
description: PACE trial : "Ponatinib for Chronic Myeloid Leukemia (CML) Evaluation and Ph+ Acute Lymphoblastic Leukemia (ALL)", NCT01207440
arm group label: Allogenis stem cell transplantation (EBMT registry)
description: EBMT : European Group for Blood and Marrow Transplantation
intervention type: Drug
intervention name: Ponatinib (Iclusig®)
description: Novel tyrosine kinase inhibitor active in patients with an ABL T315I mutation compared to conventional or reduced intensity allogeneic stem cell transplantation.
arm group label: Ponatinib (PACE trial)
intervention type: Procedure
intervention name: allogeneic stem cell transplantation
arm group label: Allogenis stem cell transplantation (EBMT registry)
study pop: 
sampling method: Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
facility: 
country: France
responsible party type: Sponsor
mesh term: Leukemia
mesh term: Ponatinib
sharing ipd: No