<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/NCT02218437</url>
  </required_header>
  <id_info>
    <org_study_id>MSC-2014-SAA-1</org_study_id>
    <secondary_id>H0806-MSC-2014</secondary_id>
    <nct_id>NCT02218437</nct_id>
  </id_info>
  <brief_title>Treatment Protocol of Child SAA With the Injection of Mesenchymal Stem Cells（Umbilical Cord Derived）</brief_title>
  <acronym>MSC-SAA</acronym>
  <official_title>Child With Severe Aplastic Anemia (SAA) Therapy: the Injection of Umbilical Cord Derived Mesenchymal Stem Cells.</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Cancer Institute and Hospital, Chinese Academy of Medical Sciences</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Cancer Institute and Hospital, Chinese Academy of Medical Sciences</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Mesenchymal Stem Cells (MSC) is a non Hematopoietic Stem Cells (HSC) in adult bone marrow and&#xD;
      takes part in the bone marrow microenvironment. The response rate of early treatment on&#xD;
      Children's SAA application combined with anti-thymocyte globulin (ATG) (40-50 days after ATG&#xD;
      treatment) is associated with long-term effect. The injection of Umbilical Cord Derived&#xD;
      Mesenchymal Stem Cells combined with ATG improves the efficacy of children with SAA.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      1. Mesenchymal Stem Cells were prepared by in vitro separation, screening, and culture from&#xD;
           healthy human umbilical cord tissue; The &quot; injection of mesenchymal stem cells&#xD;
           (umbilical) manufacturing and verification regulation &quot; was also formulated.&#xD;
&#xD;
        2. The starting dose of Umbilical Cord Derived MSC was 0.5-1.0 * 106 cells /kg, based on&#xD;
           the previous human studies; And the maximum tolerated dose was 1 * 107 cells /kg&#xD;
&#xD;
        3. The response and complete remission rate, relapse rate of the injection of Umbilical&#xD;
           Cord Derived Mesenchymal Stem Cells (or combined with ATG ) for Child with SAA were&#xD;
           determined.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Recruiting</last_known_status>
  <start_date>October 2013</start_date>
  <completion_date type="Anticipated">October 2015</completion_date>
  <primary_completion_date type="Anticipated">October 2015</primary_completion_date>
  <phase>Phase 4</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>The response and complete remission rate with different doses of Umbilical Cord Derived MSC to treat child with SAA</measure>
    <time_frame>1 year</time_frame>
    <description>Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count &gt;100*109/L, and absolute neutrophil count (ANC) &gt;1.5*1.0 9/L. Partial response (PR) was defined as injection independence, reticulocyte count&gt;30*109/L, platelet count &gt;20*109/L, and ANC&gt;0.5*1.0 9/L above the baseline. Persistence of injection requirement or death was evidence of no response (NR).</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>The relapse rate with different doses of MSC to treat child with SAA</measure>
    <time_frame>3-10 year</time_frame>
    <description>The relapse was defined as injection dependence again; or progressed or paroxysmal nocturnal hemoglobinuria (PNH)/acute myeloid leukemia/myelodysplasia syndrome(MDS); or cyclosporin A (CsA) dependence.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">20</enrollment>
  <condition>Complications of Organ Transplant Stem Cells Umbilical Cord</condition>
  <arm_group>
    <arm_group_label>MSC+ATG</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>The first agent MSC injection, began two weeks after ATG application; Each patient was injected three times, one time per week; Study on single dose tolerance and efficacy index; Each subjects received three dose groups of treatment.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>MSC+ATG</intervention_name>
    <description>MSC+ATG:Starting dose was 0.5-1.0 × 106 cells /kg; And the maximum tolerated dose was 1 ×107 cells /kg after ATG application.</description>
    <arm_group_label>MSC+ATG</arm_group_label>
    <other_name>Mesenchymal Stem Cells (Umbilical Cord Derived)</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Non-Severe Aplastic Anemia (NSAA)atients&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Severe Aplastic Anemia (SAA) patients&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Month</minimum_age>
    <maximum_age>18 Years</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Xiaofan Zhu, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Cancer Institute and Hospital, Chinese Academy of Medical Sciences</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Xiaofan Zhu, MD</last_name>
    <phone>+86-22-23909001</phone>
    <email>zhuxf6465@gmail.com</email>
  </overall_contact>
  <location>
    <facility>
      <name>Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences and Peking Union Medical College</name>
      <address>
        <city>Tianjin</city>
        <zip>300020</zip>
        <country>China</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Xiaofan Zhu, MD</last_name>
      <phone>+86-22-23909001</phone>
      <email>zhuxf6465@gmail.com</email>
    </contact>
    <investigator>
      <last_name>Xiaofan Zhu, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>China</country>
  </location_countries>
  <verification_date>August 2014</verification_date>
  <study_first_submitted>August 6, 2014</study_first_submitted>
  <study_first_submitted_qc>August 14, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">August 18, 2014</study_first_posted>
  <last_update_submitted>August 14, 2014</last_update_submitted>
  <last_update_submitted_qc>August 14, 2014</last_update_submitted_qc>
  <last_update_posted type="Estimate">August 18, 2014</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Cancer Institute and Hospital, Chinese Academy of Medical Sciences</investigator_affiliation>
    <investigator_full_name>Xiaofan Zhu</investigator_full_name>
    <investigator_title>chief physician</investigator_title>
  </responsible_party>
  <keyword>Mesenchymal Stem Cells (MSC)</keyword>
  <keyword>Umbilical Cord</keyword>
  <keyword>thymocyte globulin (ATG)</keyword>
  <keyword>Severe Aplastic Anemia (SAA)</keyword>
  <!-- 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/NCT02218437
org study id: MSC-2014-SAA-1
secondary id: H0806-MSC-2014
nct id: NCT02218437
lead sponsor: 
has dmc: Yes
Mesenchymal Stem Cells (MSC) is a non Hematopoietic Stem Cells (HSC) in adult bone marrow and
      takes part in the bone marrow microenvironment. The response rate of early treatment on
      Children's SAA application combined with anti-thymocyte globulin (ATG) (40-50 days after ATG
      treatment) is associated with long-term effect. The injection of Umbilical Cord Derived
      Mesenchymal Stem Cells combined with ATG improves the efficacy of children with SAA.
1. Mesenchymal Stem Cells were prepared by in vitro separation, screening, and culture from
           healthy human umbilical cord tissue; The " injection of mesenchymal stem cells
           (umbilical) manufacturing and verification regulation " was also formulated.

        2. The starting dose of Umbilical Cord Derived MSC was 0.5-1.0 * 106 cells /kg, based on
           the previous human studies; And the maximum tolerated dose was 1 * 107 cells /kg

        3. The response and complete remission rate, relapse rate of the injection of Umbilical
           Cord Derived Mesenchymal Stem Cells (or combined with ATG ) for Child with SAA were
           determined.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: The response and complete remission rate with different doses of Umbilical Cord Derived MSC to treat child with SAA
time frame: 1 year
description: Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count >100*109/L, and absolute neutrophil count (ANC) >1.5*1.0 9/L. Partial response (PR) was defined as injection independence, reticulocyte count>30*109/L, platelet count >20*109/L, and ANC>0.5*1.0 9/L above the baseline. Persistence of injection requirement or death was evidence of no response (NR).
measure: The relapse rate with different doses of MSC to treat child with SAA
time frame: 3-10 year
description: The relapse was defined as injection dependence again; or progressed or paroxysmal nocturnal hemoglobinuria (PNH)/acute myeloid leukemia/myelodysplasia syndrome(MDS); or cyclosporin A (CsA) dependence.
arm group label: MSC+ATG
arm group type: Experimental
description: The first agent MSC injection, began two weeks after ATG application; Each patient was injected three times, one time per week; Study on single dose tolerance and efficacy index; Each subjects received three dose groups of treatment.
intervention type: Drug
intervention name: MSC+ATG
description: MSC+ATG:Starting dose was 0.5-1.0 × 106 cells /kg; And the maximum tolerated dose was 1 ×107 cells /kg after ATG application.
arm group label: MSC+ATG
other name: Mesenchymal Stem Cells (Umbilical Cord Derived)
criteria: 
gender: All
minimum age: 1 Month
maximum age: 18 Years
healthy volunteers: Accepts Healthy Volunteers
last name: Xiaofan Zhu, MD
role: Principal Investigator
affiliation: Cancer Institute and Hospital, Chinese Academy of Medical Sciences
last name: Xiaofan Zhu, MD
phone: +86-22-23909001
email: zhuxf6465@gmail.com
facility: 
status: Recruiting
contact: 
investigator: 
country: China
responsible party type: Principal Investigator
investigator affiliation: Cancer Institute and Hospital, Chinese Academy of Medical Sciences
investigator full name: Xiaofan Zhu
investigator title: chief physician