<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/NCT03821987</url>
  </required_header>
  <id_info>
    <org_study_id>PUIH-SAA</org_study_id>
    <nct_id>NCT03821987</nct_id>
  </id_info>
  <brief_title>Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA</brief_title>
  <official_title>Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA</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>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The haplotype HSCT system including Bu(0.8mg/kg Q6hx2d)CTX(50mg/kgx4d)rATG(2.5mg/kgx4d) ,&#xD;
      established in Institute of Hematology of Peking University ,has been evaluated to be&#xD;
      effective for acquired SAA.But some patients with high risk factors may not tolerate CTX&#xD;
      200mg/kg,alternative conditioning regimen including Bu/Fludarabine/dercreased CTX was studied&#xD;
      in this trial.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Patients enrolled in this study would receive Bu (IV)0.8mg/kg Q6hx2d,Fludarabine 30mg/m2x5d&#xD;
      ,CTX(cyclophosphamide) 25mg/kg/dx4d,rATG (thymoglobulin,Sang Stat,France) 2.5mg/kg/dx4d.&#xD;
&#xD;
      BM or Blood samples from patients were obtained to assess engraftment and chimerism after&#xD;
      HSCT. The time point that investigators monitor BM or blood samples at 1 month,2 months, 3&#xD;
      months,6 months, 9 months and 1year,2years,3years 5 years after HSCT.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Recruiting</last_known_status>
  <start_date type="Actual">December 17, 2018</start_date>
  <completion_date type="Anticipated">March 30, 2022</completion_date>
  <primary_completion_date type="Anticipated">December 30, 2021</primary_completion_date>
  <phase>N/A</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>
    <intervention_model_description>Patients enrolled in this study would receive Bu (IV)0.8mg/kg Q6hx2d,Fludarabine 30mg/m2x5d ,CTX(cyclophosphamide) 25mg/kg/dx4d,rATG (thymoglobulin,Sang Stat,France) 2.5mg/kg/dx4d.&#xD;
BM or Blood samples from patients were obtained to assess engraftment and chimerism after HSCT. The time point that we monitored BM or blood samples included at 1 month,2 months, 3 months,6 months, 9 months and 1year,2years,3years 5 years after HSCT.</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>1 year cumulative incidence overall survival</measure>
    <time_frame>1 year post HSCT</time_frame>
    <description>Tne cumulative incidence of overall survival at 1 year post HSCT</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>one month Transplantation related mortality</measure>
    <time_frame>1 month post HSCT</time_frame>
    <description>Tne cumulative incidence of transplantation related motality at 1 month post HSCT</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Engraftment</measure>
    <time_frame>1 month post HSCT</time_frame>
    <description>Tne cumulative incidence of engraftment at 1 moths post HSCT</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>0ne month regimen-related toxicity</measure>
    <time_frame>1 month post HSCT</time_frame>
    <description>Tne cumulative incidence of regimen related toxicity at 1 month post HSCT</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>aGVHD</measure>
    <time_frame>100 days post HSCT</time_frame>
    <description>Tne cumulative incidence of acute GVHD at Day 100 post HSCT</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>failure-free survival</measure>
    <time_frame>1 year post HSCT</time_frame>
    <description>The cumulative incidence of failure-free survival at 1year post HSCT</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">55</enrollment>
  <condition>Aplastic Anemia</condition>
  <condition>Stem Cell Transplant Complications</condition>
  <condition>Engraft Failure</condition>
  <arm_group>
    <arm_group_label>BFCA regimen</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Detail: Patients enrolled in this study would receive Busulfan(B) (IV)0.8mg/kg Q6hx2d,Fludarabine(F) 30mg/m2x5d ,cyclophosphamide(C) 25mg/kg/dx4d,thymoglobulin (A :rATG ,Sang Stat,France) 2.5mg/kg/dx4d.&#xD;
BM or Blood samples from patients were obtained to assess engraftment and chimerism after HSCT. The time point that we monitored BM or blood samples included at 1 month,2 months, 3 months,6 months, 9 months and 1year,2years,3years 5 years after HSCT.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Fludarabine</intervention_name>
    <description>Patients enrolled in this study would receive Bu (IV)0.8mg/kg Q6hx2d,Fludarabine 30mg/m2x5d ,CTX (cyclophosphamide) 25mg/kg/dx4d,rATG (thymoglobulin,Sang Stat,France) 2.5mg/kg/dx4d.</description>
    <arm_group_label>BFCA regimen</arm_group_label>
    <other_name>Busulfan</other_name>
    <other_name>Cyclophosphamide</other_name>
    <other_name>Thymoglobulin</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        A：inclusion criteria&#xD;
&#xD;
          1. Patients diagnosed as acquired severe aplastic anemia(SAA) /very vSAA&#xD;
&#xD;
          2. patients with age 3-55 years&#xD;
&#xD;
          3. patients have no matched sibling donor&#xD;
&#xD;
          4. Patients have no matched unrelated donor&#xD;
&#xD;
          5. patients have no severe infection&#xD;
&#xD;
          6. Patients have no severe organ dysfunction&#xD;
&#xD;
          7. patients have risk factors of potential intolerance to previous condition regimen&#xD;
             including BuCy(200mg/kg)and ATG&#xD;
&#xD;
          8. Consent form signed&#xD;
&#xD;
        B. Exclusion criteria ：&#xD;
&#xD;
          1. patients with congenital SAA/vSAA&#xD;
&#xD;
          2. patients with age&lt; 3years or &gt;55 years&#xD;
&#xD;
          3. patients with matched sibling donor&#xD;
&#xD;
          4. patients with matched URD&#xD;
&#xD;
          5. patients with severe infection&#xD;
&#xD;
          6. patients with severe organ dysfunction&#xD;
&#xD;
          7. pregnancy women&#xD;
&#xD;
          8. no Consent form signed&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>3 Years</minimum_age>
    <maximum_age>55 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Xiaojun Huang, Prof.</last_name>
    <role>Principal Investigator</role>
    <affiliation>Peking Universiy Institute of Hematology,People's Hospital Pekiking Universiyy</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Xiaojun Huang, Prof.</last_name>
    <phone>861088326006</phone>
    <email>lpxu_0415@sina.com</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Lanping Xu</last_name>
    <phone>861088326904</phone>
    <email>lpxu_0415@sina.com</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Peking University Institute of Hematology,People's hospital Peking University</name>
      <address>
        <city>Beijing</city>
        <state>Beijing</state>
        <zip>100044</zip>
        <country>China</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Lanping Xu, Prof.</last_name>
      <phone>8613641028627</phone>
      <email>lpxu_0415@sina.com</email>
    </contact>
    <contact_backup>
      <last_name>Yanru Ma</last_name>
      <phone>8613641134402</phone>
      <email>lpxu_0415@vip.sina.com</email>
    </contact_backup>
  </location>
  <location_countries>
    <country>China</country>
  </location_countries>
  <verification_date>March 2020</verification_date>
  <study_first_submitted>January 23, 2019</study_first_submitted>
  <study_first_submitted_qc>January 26, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">January 30, 2019</study_first_posted>
  <last_update_submitted>March 9, 2020</last_update_submitted>
  <last_update_submitted_qc>March 9, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">March 10, 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>Director of Institute Hematology,Peking University</investigator_title>
  </responsible_party>
  <keyword>haploidentical transplantation</keyword>
  <keyword>aplastic anemia</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Anemia, Aplastic</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cyclophosphamide</mesh_term>
    <mesh_term>Busulfan</mesh_term>
    <mesh_term>Fludarabine</mesh_term>
    <mesh_term>Thymoglobulin</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/NCT03821987
org study id: PUIH-SAA
nct id: NCT03821987
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
The haplotype HSCT system including Bu(0.8mg/kg Q6hx2d)CTX(50mg/kgx4d)rATG(2.5mg/kgx4d) ,
      established in Institute of Hematology of Peking University ,has been evaluated to be
      effective for acquired SAA.But some patients with high risk factors may not tolerate CTX
      200mg/kg,alternative conditioning regimen including Bu/Fludarabine/dercreased CTX was studied
      in this trial.
Patients enrolled in this study would receive Bu (IV)0.8mg/kg Q6hx2d,Fludarabine 30mg/m2x5d
      ,CTX(cyclophosphamide) 25mg/kg/dx4d,rATG (thymoglobulin,Sang Stat,France) 2.5mg/kg/dx4d.

      BM or Blood samples from patients were obtained to assess engraftment and chimerism after
      HSCT. The time point that investigators monitor BM or blood samples at 1 month,2 months, 3
      months,6 months, 9 months and 1year,2years,3years 5 years after HSCT.
allocation: N/A
intervention model: Single Group Assignment
intervention model description: Patients enrolled in this study would receive Bu (IV)0.8mg/kg Q6hx2d,Fludarabine 30mg/m2x5d ,CTX(cyclophosphamide) 25mg/kg/dx4d,rATG (thymoglobulin,Sang Stat,France) 2.5mg/kg/dx4d.
BM or Blood samples from patients were obtained to assess engraftment and chimerism after HSCT. The time point that we monitored BM or blood samples included at 1 month,2 months, 3 months,6 months, 9 months and 1year,2years,3years 5 years after HSCT.
primary purpose: Treatment
masking: None (Open Label)
measure: 1 year cumulative incidence overall survival
time frame: 1 year post HSCT
description: Tne cumulative incidence of overall survival at 1 year post HSCT
measure: one month Transplantation related mortality
time frame: 1 month post HSCT
description: Tne cumulative incidence of transplantation related motality at 1 month post HSCT
measure: Engraftment
time frame: 1 month post HSCT
description: Tne cumulative incidence of engraftment at 1 moths post HSCT
measure: 0ne month regimen-related toxicity
time frame: 1 month post HSCT
description: Tne cumulative incidence of regimen related toxicity at 1 month post HSCT
measure: aGVHD
time frame: 100 days post HSCT
description: Tne cumulative incidence of acute GVHD at Day 100 post HSCT
measure: failure-free survival
time frame: 1 year post HSCT
description: The cumulative incidence of failure-free survival at 1year post HSCT
arm group label: BFCA regimen
arm group type: Experimental
description: Detail: Patients enrolled in this study would receive Busulfan(B) (IV)0.8mg/kg Q6hx2d,Fludarabine(F) 30mg/m2x5d ,cyclophosphamide(C) 25mg/kg/dx4d,thymoglobulin (A :rATG ,Sang Stat,France) 2.5mg/kg/dx4d.
BM or Blood samples from patients were obtained to assess engraftment and chimerism after HSCT. The time point that we monitored BM or blood samples included at 1 month,2 months, 3 months,6 months, 9 months and 1year,2years,3years 5 years after HSCT.
intervention type: Drug
intervention name: Fludarabine
description: Patients enrolled in this study would receive Bu (IV)0.8mg/kg Q6hx2d,Fludarabine 30mg/m2x5d ,CTX (cyclophosphamide) 25mg/kg/dx4d,rATG (thymoglobulin,Sang Stat,France) 2.5mg/kg/dx4d.
arm group label: BFCA regimen
other name: Busulfan
other name: Cyclophosphamide
other name: Thymoglobulin
criteria: 
gender: All
minimum age: 3 Years
maximum age: 55 Years
healthy volunteers: No
last name: Xiaojun Huang, Prof.
role: Principal Investigator
affiliation: Peking Universiy Institute of Hematology,People's Hospital Pekiking Universiyy
last name: Xiaojun Huang, Prof.
phone: 861088326006
email: lpxu_0415@sina.com
last name: Lanping Xu
phone: 861088326904
email: lpxu_0415@sina.com
facility: 
status: Recruiting
contact: 
contact backup: 
country: China
responsible party type: Principal Investigator
investigator affiliation: Peking University People's Hospital
investigator full name: Xiaojun Huang,MD
investigator title: Director of Institute Hematology,Peking University
mesh term: Anemia, Aplastic
mesh term: Cyclophosphamide
mesh term: Busulfan
mesh term: Fludarabine
mesh term: Thymoglobulin