<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/NCT03756675</url>
  </required_header>
  <id_info>
    <org_study_id>Haplo-PBSCT for AL</org_study_id>
    <nct_id>NCT03756675</nct_id>
  </id_info>
  <brief_title>Haploidentical Peripheral Blood Stem Cell Transplantation for Acute Leukemia</brief_title>
  <official_title>Haploidentical Peripheral Blood Stem Cell Transplantation for Acute Leukemia</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Peking University People's Hospital</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Peking University People's Hospital</source>
  <oversight_info>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      Allogeneic stem cell transplantation (Allo-HSCT) is the effective and even the only treatment&#xD;
      for hematological malignancies. The &quot;GIAC&quot; protocol established by our center has&#xD;
      successfully crossed the HLA barrier in HLA-mismatched/haploidentical HSCT. The protocol&#xD;
      entails the following: treating donors with granulocyte colony-stimulating factor (G-CSF) to&#xD;
      induce donor immune tolerance, intensified immunologic suppression to both promote&#xD;
      engraftment and to prevent GVHD, antithymocyte globulin (ATG) was included for the&#xD;
      prophylaxis of GVHD and graft rejection, and combination of G-CSF-primed bone marrow harvest&#xD;
      (G-BM) and G-CSF-mobilized peripheral blood stem cell harvest (G-PB) as the source of stem&#xD;
      cell grafts. But peripheral blood transplantation is still prevalent. Compared with BM, G-PB&#xD;
      is more convenient to collect, and the number of T lymphocytes and CD34+ cells is higher. It&#xD;
      is reported that G-PB has a higher implantation rate and even a higher disease-free survival&#xD;
      rate in sibiling-identical transplantation compared with BM transplantation, whereas there&#xD;
      were also reports with different conclusions. This prospective, one-arm clinical cohort study&#xD;
      aims to evaluate the safety and efficacy of haplotype peripheral blood stem cell&#xD;
      transplantation (PBSCT) in the treatment of acute leukemia.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Allogeneic stem cell transplantation (Allo-HSCT) is the effective and even the only treatment&#xD;
      for hematological malignancies. The &quot;GIAC&quot; protocol established by our center has&#xD;
      successfully crossed the HLA barrier in HLA-mismatched/haploidentical HSCT. The protocol&#xD;
      entails the following: treating donors with granulocyte colony-stimulating factor (G-CSF) to&#xD;
      induce donor immune tolerance, intensified immunologic suppression to both promote&#xD;
      engraftment and to prevent GVHD, antithymocyte globulin (ATG) was included for the&#xD;
      prophylaxis of GVHD and graft rejection, and combination of G-CSF-primed bone marrow harvest&#xD;
      (G-BM) and G-CSF-mobilized peripheral blood stem cell harvest (G-PB) as the source of stem&#xD;
      cell grafts. But peripheral blood transplantation is still prevalent. Compared with BM, G-PB&#xD;
      is more convenient to collect, and the number of T lymphocytes and CD34+ cells is higher. It&#xD;
      is reported that G-PB has a higher implantation rate and even a higher disease-free survival&#xD;
      rate in sibiling-identical transplantation compared with BM transplantation, whereas there&#xD;
      were also reports with different conclusions. This prospective, one-arm clinical cohort study&#xD;
      aims to evaluate the safety and efficacy of haplotype peripheral blood stem cell&#xD;
      transplantation (PBSCT) in the treatment of acute leukemia.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">November 1, 2018</start_date>
  <completion_date type="Anticipated">November 1, 2025</completion_date>
  <primary_completion_date type="Anticipated">November 1, 2023</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>engraftment rate</measure>
    <time_frame>one year after transplantation</time_frame>
    <description>Neutrophil recovery was defined as an absolute neutrophil count(ANC) of 0.5×10^9/L or more for three consecutive days and platelet recovery, as 20×10^9/L or more for seven consecutive days without transfusion.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>cumulative incidence of acute graft-versus-host disease(GVHD)</measure>
    <time_frame>one year after transplantation</time_frame>
    <description>cumulative incidence of acute graft-versus-host disease(GVHD)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>cumulative incidence of chronic GVHD at one year</measure>
    <time_frame>one year after transplantation</time_frame>
    <description>cumulative incidence of chronic GVHD at one year</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>cumulative incidence of relapse at one year</measure>
    <time_frame>one year after transplantation</time_frame>
    <description>Cumulative incidence of relapse was defined as the cumulative incidences of presence of morphological evidence of disease in samples from peripheral blood, bone marrow, or extramedullary sites, or by the recurrence and sustained presence of pre-transplantation chromosomal abnormalities.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>cumulative incidence of non-relapse mortality (NRM) at one year</measure>
    <time_frame>one year after transplantation</time_frame>
    <description>NRM was defined as the death without disease progression or relapse.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>overall survival at one year</measure>
    <time_frame>one year after transplantation</time_frame>
    <description>OS was defined as the time from the date of first dose until death due to any cause.</description>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Anticipated">45</enrollment>
  <condition>Acute Leukemia</condition>
  <arm_group>
    <arm_group_label>haplotype PBSCT group</arm_group_label>
    <description>Subjects in this group will receive haplotype peripheral blood stem cell transplantation (PBSCT) of &quot;GIAC&quot; system in the treatment of acute leukemia.</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>haplotype PBSCT</intervention_name>
    <description>haplotype peripheral blood stem cell transplantation (PBSCT) of &quot;GIAC&quot; system</description>
    <arm_group_label>haplotype PBSCT group</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Patients aged 2-60, who plan to receive haplotype PBSCT in the first complete remission&#xD;
        phase (CR1) of acute leukemia, and with no uncontrolled current infections or organ&#xD;
        failure.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  2-60 years old, all genders;&#xD;
&#xD;
          -  the first complete remission phase (CR1) of acute leukemia;&#xD;
&#xD;
          -  planning to receive haplotype PBSCT;&#xD;
&#xD;
          -  no uncontrolled current infections (new infections, body temperature still above 38 ℃&#xD;
             after treatment with broad-spectrum antibiotics for 72h, except for other&#xD;
             non-infectious factors);&#xD;
&#xD;
          -  no organ failure.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  with poor compliance;&#xD;
&#xD;
          -  with uncontrolled current infections；&#xD;
&#xD;
          -  pregnancy;&#xD;
&#xD;
          -  donors with contraindications of mobilization and collection of peripheral blood stem&#xD;
             cells;&#xD;
&#xD;
          -  with mental sickness&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>2 Years</minimum_age>
    <maximum_age>60 Years</maximum_age>
  </eligibility>
  <overall_official>
    <last_name>Xiao-Jun Huang, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Peking University People's Hospital</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Yu Wang, MD</last_name>
    <phone>13552647384</phone>
    <email>ywyw3172@sina.com</email>
  </overall_contact>
  <location>
    <facility>
      <name>Peking University People's Hospital</name>
      <address>
        <city>Beijing</city>
        <state>Beijing</state>
        <zip>100044</zip>
        <country>China</country>
      </address>
    </facility>
    <status>Recruiting</status>
  </location>
  <location_countries>
    <country>China</country>
  </location_countries>
  <verification_date>September 2020</verification_date>
  <study_first_submitted>November 27, 2018</study_first_submitted>
  <study_first_submitted_qc>November 27, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">November 28, 2018</study_first_posted>
  <last_update_submitted>September 15, 2020</last_update_submitted>
  <last_update_submitted_qc>September 15, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">September 17, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Peking University People's Hospital</investigator_affiliation>
    <investigator_full_name>Xiaojun Huang,MD</investigator_full_name>
    <investigator_title>Supervisor of Hematology, Peking University People's Hospital</investigator_title>
  </responsible_party>
  <keyword>allogeneic stem cell transplantation</keyword>
  <keyword>G-CSF-mobilized peripheral blood stem cell</keyword>
  <keyword>haplotype</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Leukemia</mesh_term>
    <mesh_term>Acute Disease</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Undecided</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/NCT03756675
org study id: Haplo-PBSCT for AL
nct id: NCT03756675
lead sponsor: 
is fda regulated drug: No
is fda regulated device: No
Allogeneic stem cell transplantation (Allo-HSCT) is the effective and even the only treatment
      for hematological malignancies. The "GIAC" protocol established by our center has
      successfully crossed the HLA barrier in HLA-mismatched/haploidentical HSCT. The protocol
      entails the following: treating donors with granulocyte colony-stimulating factor (G-CSF) to
      induce donor immune tolerance, intensified immunologic suppression to both promote
      engraftment and to prevent GVHD, antithymocyte globulin (ATG) was included for the
      prophylaxis of GVHD and graft rejection, and combination of G-CSF-primed bone marrow harvest
      (G-BM) and G-CSF-mobilized peripheral blood stem cell harvest (G-PB) as the source of stem
      cell grafts. But peripheral blood transplantation is still prevalent. Compared with BM, G-PB
      is more convenient to collect, and the number of T lymphocytes and CD34+ cells is higher. It
      is reported that G-PB has a higher implantation rate and even a higher disease-free survival
      rate in sibiling-identical transplantation compared with BM transplantation, whereas there
      were also reports with different conclusions. This prospective, one-arm clinical cohort study
      aims to evaluate the safety and efficacy of haplotype peripheral blood stem cell
      transplantation (PBSCT) in the treatment of acute leukemia.
Allogeneic stem cell transplantation (Allo-HSCT) is the effective and even the only treatment
      for hematological malignancies. The "GIAC" protocol established by our center has
      successfully crossed the HLA barrier in HLA-mismatched/haploidentical HSCT. The protocol
      entails the following: treating donors with granulocyte colony-stimulating factor (G-CSF) to
      induce donor immune tolerance, intensified immunologic suppression to both promote
      engraftment and to prevent GVHD, antithymocyte globulin (ATG) was included for the
      prophylaxis of GVHD and graft rejection, and combination of G-CSF-primed bone marrow harvest
      (G-BM) and G-CSF-mobilized peripheral blood stem cell harvest (G-PB) as the source of stem
      cell grafts. But peripheral blood transplantation is still prevalent. Compared with BM, G-PB
      is more convenient to collect, and the number of T lymphocytes and CD34+ cells is higher. It
      is reported that G-PB has a higher implantation rate and even a higher disease-free survival
      rate in sibiling-identical transplantation compared with BM transplantation, whereas there
      were also reports with different conclusions. This prospective, one-arm clinical cohort study
      aims to evaluate the safety and efficacy of haplotype peripheral blood stem cell
      transplantation (PBSCT) in the treatment of acute leukemia.
observational model: Cohort
time perspective: Prospective
measure: engraftment rate
time frame: one year after transplantation
description: Neutrophil recovery was defined as an absolute neutrophil count(ANC) of 0.5×10^9/L or more for three consecutive days and platelet recovery, as 20×10^9/L or more for seven consecutive days without transfusion.
measure: cumulative incidence of acute graft-versus-host disease(GVHD)
time frame: one year after transplantation
description: cumulative incidence of acute graft-versus-host disease(GVHD)
measure: cumulative incidence of chronic GVHD at one year
time frame: one year after transplantation
description: cumulative incidence of chronic GVHD at one year
measure: cumulative incidence of relapse at one year
time frame: one year after transplantation
description: Cumulative incidence of relapse was defined as the cumulative incidences of presence of morphological evidence of disease in samples from peripheral blood, bone marrow, or extramedullary sites, or by the recurrence and sustained presence of pre-transplantation chromosomal abnormalities.
measure: cumulative incidence of non-relapse mortality (NRM) at one year
time frame: one year after transplantation
description: NRM was defined as the death without disease progression or relapse.
measure: overall survival at one year
time frame: one year after transplantation
description: OS was defined as the time from the date of first dose until death due to any cause.
arm group label: haplotype PBSCT group
description: Subjects in this group will receive haplotype peripheral blood stem cell transplantation (PBSCT) of "GIAC" system in the treatment of acute leukemia.
intervention type: Other
intervention name: haplotype PBSCT
description: haplotype peripheral blood stem cell transplantation (PBSCT) of "GIAC" system
arm group label: haplotype PBSCT group
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 2 Years
maximum age: 60 Years
last name: Xiao-Jun Huang, MD
role: Principal Investigator
affiliation: Peking University People's Hospital
last name: Yu Wang, MD
phone: 13552647384
email: ywyw3172@sina.com
facility: 
status: Recruiting
country: China
responsible party type: Principal Investigator
investigator affiliation: Peking University People's Hospital
investigator full name: Xiaojun Huang,MD
investigator title: Supervisor of Hematology, Peking University People's Hospital
mesh term: Leukemia
mesh term: Acute Disease
sharing ipd: Undecided