<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/NCT02241031</url>
  </required_header>
  <id_info>
    <org_study_id>MPs-AL-2014</org_study_id>
    <nct_id>NCT02241031</nct_id>
  </id_info>
  <brief_title>Megakaryocytic Progenitor Cells for Prophylaxis and Treatment of Thrombocytopenia</brief_title>
  <official_title>Megakaryocytic Progenitor Cells for Prophylaxis and Treatment of Thrombocytopenia in Patients With Acute Leukemia Receiving Chemotherapy</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Nanfang Hospital, Southern Medical University</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Guangdong Provincial People's Hospital</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Third Affiliated Hospital, Sun Yat-Sen University</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Guangzhou General Hospital of Guangzhou Military Command</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Guangzhou First People's Hospital</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Southern Medical University, China</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Peking University People's Hospital</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Huazhong University of Science and Technology</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Sun Yat-sen University</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Academy Military Medical Science, China</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Nanfang Hospital, Southern Medical University</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to evaluate the efficacy of ex vivo generated megakaryocytic&#xD;
      progenitor cells (MPs) in prophylaxis and treatment of thrombocytopenia caused by&#xD;
      chemotherapy in patients with acute leukemia (AL).&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Thrombocytopenia is a common and potentially fatal complication of chemotherapy and&#xD;
      hematopoietic stem cell transplantation. Owing to the short storage time and increased demand&#xD;
      of platelets from unrelated donors, a constant shortage in the supply of platelets has become&#xD;
      an important medical and society challenge. Therefore, investigation of alternative sources&#xD;
      of platelets would be beneficial.&#xD;
&#xD;
      Hematopoietic stem cells (HSCs) can be used to generate functional megakaryocytic progenitors&#xD;
      (MPs), megakaryocytes, and platelets on a large scale. Functional MPs and platelets have&#xD;
      successfully been produced in vitro from CD34+ hematopoietic cells from bone marrow, cord&#xD;
      blood, and peripheral blood. Several studies have reported that transplantation of in vitro&#xD;
      auto-producing MPs can promote platelet recovery after high-dose therapy and HSC&#xD;
      transplantation.&#xD;
&#xD;
      Umbilical cord blood is an abundant source of HSCs. In vitro large scale production of MPs&#xD;
      from cord blood could represent an effective platelet substitute. Theoretically, the&#xD;
      additional transplantation of ex vivo generated progenitor and post-progenitor cells might&#xD;
      lead to the production of sufficient numbers of mature functional cells within a few days&#xD;
      after transplantation.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Recruiting</last_known_status>
  <start_date>September 2014</start_date>
  <completion_date type="Anticipated">December 2016</completion_date>
  <primary_completion_date type="Anticipated">September 2016</primary_completion_date>
  <phase>Phase 2/Phase 3</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Platelet recovery after infusion of MPs</measure>
    <time_frame>3 months</time_frame>
    <description>Platelet recovery after infusion of MPs includes the time from infusion to platelet count≥20×10^9/L,50×10^9/L,100×10^9/L.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>frequency of platelet infusion</measure>
    <time_frame>3 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>incidence of acute toxicity</measure>
    <time_frame>3 month</time_frame>
    <description>Acute toxicity mainly involves the heart,live and kidney.</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Anticipated">250</enrollment>
  <condition>Hematological Diseases</condition>
  <condition>Thrombocytopenia</condition>
  <arm_group>
    <arm_group_label>MPs</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>MPs will be intravenously infused within 48 hours after chemotherapy. During the study period, patients can receive platelet infusion but can not receive platelet stimulating factors.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Non-MPs</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>Patients can receive platelet infusion but can not receive platelet stimulating factors.</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>MPs</intervention_name>
    <description>MPs are generated from cord blood using a combination of cytokines.</description>
    <arm_group_label>MPs</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>thrombopoietin (TPO) and interleukin-11</intervention_name>
    <description>Platelet stimulating factors include thrombopoietin (TPO) and interleukin-11 and so on.</description>
    <arm_group_label>Non-MPs</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  age：14-65 years&#xD;
&#xD;
          -  achieve complete remission of acute leukemia&#xD;
&#xD;
          -  the first course of consolidation chemotherapy&#xD;
&#xD;
          -  ECOG grades 0 or 1&#xD;
&#xD;
          -  expected survival time ≥ three months&#xD;
&#xD;
          -  Subjects (or their legally acceptable representatives) must have signed an informed&#xD;
             consent document.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  cardiac dysfunction (particularly congestive heart failure), hepatic abnormalities&#xD;
             (bilirubin ≥ 3 mg/dL, aminotransferase&gt; 2 times the upper limit of normal), renal&#xD;
             dysfunction (creatinine clearance rate &lt; 30 mL/min)&#xD;
&#xD;
          -  Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood&#xD;
             pressure)&#xD;
&#xD;
          -  Patients with any conditions not suitable for the trial (investigators' decision)&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>14 Years</minimum_age>
    <maximum_age>65 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Qifa Liu, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Nanfang Hospital, Southern Medical University</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Ren Lin, MD</last_name>
    <phone>+86-020-62787883</phone>
    <email>lansinglinren@hotmail.com</email>
  </overall_contact>
  <location>
    <facility>
      <name>Department of Hematology,Nanfang Hospital, Southern Medical University</name>
      <address>
        <city>Guangzhou</city>
        <state>Guangdong</state>
        <zip>510515</zip>
        <country>China</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Ren Lin, MD</last_name>
      <phone>+86-020-62787883</phone>
      <email>lansinglinren@hotmail.com</email>
    </contact>
    <investigator>
      <last_name>Qifa Liu, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>China</country>
  </location_countries>
  <reference>
    <citation>Xi J, Zhu H, Liu D, Nan X, Zheng W, Liu K, Shi W, Chen L, Lv Y, Yan F, Li Y, Xie X, Wang Y, Yue W, Xu X, Wei X, Zhu J, Huang X, Pei X. Infusion of megakaryocytic progenitor products generated from cord blood hematopoietic stem/progenitor cells: results of the phase 1 study. PLoS One. 2013;8(2):e54941. doi: 10.1371/journal.pone.0054941. Epub 2013 Feb 4.</citation>
    <PMID>23390507</PMID>
  </reference>
  <verification_date>September 2014</verification_date>
  <study_first_submitted>September 13, 2014</study_first_submitted>
  <study_first_submitted_qc>September 15, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 16, 2014</study_first_posted>
  <last_update_submitted>September 15, 2014</last_update_submitted>
  <last_update_submitted_qc>September 15, 2014</last_update_submitted_qc>
  <last_update_posted type="Estimate">September 16, 2014</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Nanfang Hospital, Southern Medical University</investigator_affiliation>
    <investigator_full_name>Qifa Liu</investigator_full_name>
    <investigator_title>Professor</investigator_title>
  </responsible_party>
  <keyword>Megakaryocytic Progenitor Products</keyword>
  <keyword>Hematological Diseases</keyword>
  <keyword>Thrombocytopenia</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Thrombocytopenia</mesh_term>
    <mesh_term>Hematologic Diseases</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Oprelvekin</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/NCT02241031
org study id: MPs-AL-2014
nct id: NCT02241031
lead sponsor: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
has dmc: Yes
The purpose of this study is to evaluate the efficacy of ex vivo generated megakaryocytic
      progenitor cells (MPs) in prophylaxis and treatment of thrombocytopenia caused by
      chemotherapy in patients with acute leukemia (AL).
Thrombocytopenia is a common and potentially fatal complication of chemotherapy and
      hematopoietic stem cell transplantation. Owing to the short storage time and increased demand
      of platelets from unrelated donors, a constant shortage in the supply of platelets has become
      an important medical and society challenge. Therefore, investigation of alternative sources
      of platelets would be beneficial.

      Hematopoietic stem cells (HSCs) can be used to generate functional megakaryocytic progenitors
      (MPs), megakaryocytes, and platelets on a large scale. Functional MPs and platelets have
      successfully been produced in vitro from CD34+ hematopoietic cells from bone marrow, cord
      blood, and peripheral blood. Several studies have reported that transplantation of in vitro
      auto-producing MPs can promote platelet recovery after high-dose therapy and HSC
      transplantation.

      Umbilical cord blood is an abundant source of HSCs. In vitro large scale production of MPs
      from cord blood could represent an effective platelet substitute. Theoretically, the
      additional transplantation of ex vivo generated progenitor and post-progenitor cells might
      lead to the production of sufficient numbers of mature functional cells within a few days
      after transplantation.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Platelet recovery after infusion of MPs
time frame: 3 months
description: Platelet recovery after infusion of MPs includes the time from infusion to platelet count≥20×10^9/L,50×10^9/L,100×10^9/L.
measure: frequency of platelet infusion
time frame: 3 months
measure: incidence of acute toxicity
time frame: 3 month
description: Acute toxicity mainly involves the heart,live and kidney.
arm group label: MPs
arm group type: Experimental
description: MPs will be intravenously infused within 48 hours after chemotherapy. During the study period, patients can receive platelet infusion but can not receive platelet stimulating factors.
arm group label: Non-MPs
arm group type: Active Comparator
description: Patients can receive platelet infusion but can not receive platelet stimulating factors.
intervention type: Biological
intervention name: MPs
description: MPs are generated from cord blood using a combination of cytokines.
arm group label: MPs
intervention type: Drug
intervention name: thrombopoietin (TPO) and interleukin-11
description: Platelet stimulating factors include thrombopoietin (TPO) and interleukin-11 and so on.
arm group label: Non-MPs
criteria: 
gender: All
minimum age: 14 Years
maximum age: 65 Years
healthy volunteers: No
last name: Qifa Liu, MD
role: Principal Investigator
affiliation: Nanfang Hospital, Southern Medical University
last name: Ren Lin, MD
phone: +86-020-62787883
email: lansinglinren@hotmail.com
facility: 
status: Recruiting
contact: 
investigator: 
country: China
citation: Xi J, Zhu H, Liu D, Nan X, Zheng W, Liu K, Shi W, Chen L, Lv Y, Yan F, Li Y, Xie X, Wang Y, Yue W, Xu X, Wei X, Zhu J, Huang X, Pei X. Infusion of megakaryocytic progenitor products generated from cord blood hematopoietic stem/progenitor cells: results of the phase 1 study. PLoS One. 2013;8(2):e54941. doi: 10.1371/journal.pone.0054941. Epub 2013 Feb 4.
PMID: 23390507
responsible party type: Principal Investigator
investigator affiliation: Nanfang Hospital, Southern Medical University
investigator full name: Qifa Liu
investigator title: Professor
mesh term: Thrombocytopenia
mesh term: Hematologic Diseases
mesh term: Oprelvekin