<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/NCT02969980</url>
  </required_header>
  <id_info>
    <org_study_id>MDS_PTCy_2016</org_study_id>
    <nct_id>NCT02969980</nct_id>
  </id_info>
  <brief_title>Hematopoietic Cell Transplantation With Post-transplantation Cyclophosphamide in MDS</brief_title>
  <official_title>Allogeneic Hematopoietic Cell Transplantation Using Post-transplantation Cyclophosphamide in Myelodysplastic Syndrome Patients</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Asan Medical Center</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Asan Medical Center</source>
  <brief_summary>
    <textblock>
      This study is conducted to evaluate the feasibility and efficacy of post-transplantation&#xD;
      cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic&#xD;
      hematopoietic cell transplantation (HCT) in patients with myelodysplastic syndrome (MDS).&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      -  Conditioning regimen A) Busulfan 3.2 mg/kg/day i.v. daily For 4 days (days -7 to -4) in&#xD;
           patients aged &lt; 55 years For 2 days (days -7 and -6) in patients aged ≥ 55 years B)&#xD;
           Fludarabine 30 mg/m2/day i.v. daily on days -7 to -2 (for 6 days)&#xD;
&#xD;
        -  Harvest and infusion of donor hematopoietic cells Harvested peripheral blood mononuclear&#xD;
           cells of donors via leukapheresis will be infused to recipients on day 0. Additional&#xD;
           infusion on day 1 can be made based on the judgement of attending physician.&#xD;
&#xD;
        -  GVHD prophylaxis A) Cyclophosphamide 50 mg/kg/day i.v. daily on days 3 and 4 (for 2&#xD;
           days) B) Cyclosporine: 1.5 mg/kg i.v. every 12 h hours beginning on day 5 and changed to&#xD;
           oral dosing when oral intake is possible. If there is no evidence of GVHD, the dosage&#xD;
           will be tapered beginning on day 90 and discontinued on day 180. C) MMF: 15 mg/kg p.o. 3&#xD;
           times daily with a maximum daily dose of 3.0 g beginning on day 5&#xD;
&#xD;
      If there is no active GVHD, Cyclosporine and MMF will be discontinued without taper at day&#xD;
      180 and 35, respectively.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Active, not recruiting</last_known_status>
  <start_date>November 2016</start_date>
  <completion_date type="Anticipated">December 31, 2021</completion_date>
  <primary_completion_date type="Anticipated">December 31, 2021</primary_completion_date>
  <phase>Phase 2</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 incidence of acute and chronic graft-versus-host disease</measure>
    <time_frame>100 days and 2 year, respectively</time_frame>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">30</enrollment>
  <condition>Myelodysplastic Syndromes</condition>
  <arm_group>
    <arm_group_label>PTCy</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Patients who receive post-transplantation cyclophosphamide</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Cyclophosphamide</intervention_name>
    <description>Cyclophosphamide 50 mg/kg/day i.v. daily on days 3 and 4 of hematopoietic cell transplantation</description>
    <arm_group_label>PTCy</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  MDS defined by WHO classification, chronic myelomonocytic leukemia (CMML), or acute&#xD;
             myeloid leukemia (AML) evolving from MDS A. IPSS &gt; 1.0 or bone marrow blast ≥ 5% at&#xD;
             any time points before HCT or B. AML progressed from MDS or C. CMML&#xD;
&#xD;
          -  Patients receiving first HCT&#xD;
&#xD;
          -  Patients with appropriate hematopoietic cell donors A. HLA-matched sibling donor B.&#xD;
             Unrelated donor C. HLA-mismatched familial donor&#xD;
&#xD;
          -  15 years old or older&#xD;
&#xD;
          -  Adequate performance status (Karnofsky score of 70 or more)&#xD;
&#xD;
          -  Adequate hepatic and renal function (AST, ALT, and bilirubin &lt; 3.0 x upper normal&#xD;
             limit, and creatinine &lt; 2.0 mg/dL).&#xD;
&#xD;
          -  Adequate cardiac function (left ventricular ejection fraction over 40% on heart scan&#xD;
             or echocardiogram&#xD;
&#xD;
          -  Signed and dated informed consent must be obtained from both recipient and donor.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Presence of significant active infection&#xD;
&#xD;
          -  Presence of uncontrolled bleeding&#xD;
&#xD;
          -  Any coexisting major illness or organ failure&#xD;
&#xD;
          -  Patients with psychiatric disorder or mental deficiency severe as to make compliance&#xD;
             with the treatment unlike, and making informed consent impossible&#xD;
&#xD;
          -  Nursing women, pregnant women, women of childbearing potential who do not want&#xD;
             adequate contraception&#xD;
&#xD;
          -  Patients with a diagnosis of prior malignancy unless disease-free for at least 5 years&#xD;
             following therapy with curative intent (except curatively treated nonmelanoma skin&#xD;
             cancer, in situ carcinoma, or cervical intraepithelial neoplasia)&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>15 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Asan Medical Center, University of Ulsan College of Medicine</name>
      <address>
        <city>Seoul</city>
        <zip>05505</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Korea, Republic of</country>
  </location_countries>
  <verification_date>January 2020</verification_date>
  <study_first_submitted>November 17, 2016</study_first_submitted>
  <study_first_submitted_qc>November 17, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">November 21, 2016</study_first_posted>
  <last_update_submitted>January 8, 2020</last_update_submitted>
  <last_update_submitted_qc>January 8, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">January 13, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Asan Medical Center</investigator_affiliation>
    <investigator_full_name>Je-Hwan Lee</investigator_full_name>
    <investigator_title>M.D. Professor</investigator_title>
  </responsible_party>
  <keyword>MDS</keyword>
  <keyword>AML progressed from MDS</keyword>
  <keyword>CMML</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Preleukemia</mesh_term>
    <mesh_term>Myelodysplastic Syndromes</mesh_term>
    <mesh_term>Syndrome</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cyclophosphamide</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/NCT02969980
org study id: MDS_PTCy_2016
nct id: NCT02969980
lead sponsor: 
This study is conducted to evaluate the feasibility and efficacy of post-transplantation
      cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic
      hematopoietic cell transplantation (HCT) in patients with myelodysplastic syndrome (MDS).
-  Conditioning regimen A) Busulfan 3.2 mg/kg/day i.v. daily For 4 days (days -7 to -4) in
           patients aged < 55 years For 2 days (days -7 and -6) in patients aged ≥ 55 years B)
           Fludarabine 30 mg/m2/day i.v. daily on days -7 to -2 (for 6 days)

        -  Harvest and infusion of donor hematopoietic cells Harvested peripheral blood mononuclear
           cells of donors via leukapheresis will be infused to recipients on day 0. Additional
           infusion on day 1 can be made based on the judgement of attending physician.

        -  GVHD prophylaxis A) Cyclophosphamide 50 mg/kg/day i.v. daily on days 3 and 4 (for 2
           days) B) Cyclosporine: 1.5 mg/kg i.v. every 12 h hours beginning on day 5 and changed to
           oral dosing when oral intake is possible. If there is no evidence of GVHD, the dosage
           will be tapered beginning on day 90 and discontinued on day 180. C) MMF: 15 mg/kg p.o. 3
           times daily with a maximum daily dose of 3.0 g beginning on day 5

      If there is no active GVHD, Cyclosporine and MMF will be discontinued without taper at day
      180 and 35, respectively.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: the incidence of acute and chronic graft-versus-host disease
time frame: 100 days and 2 year, respectively
arm group label: PTCy
arm group type: Experimental
description: Patients who receive post-transplantation cyclophosphamide
intervention type: Drug
intervention name: Cyclophosphamide
description: Cyclophosphamide 50 mg/kg/day i.v. daily on days 3 and 4 of hematopoietic cell transplantation
arm group label: PTCy
criteria: 
gender: All
minimum age: 15 Years
maximum age: N/A
healthy volunteers: No
facility: 
country: Korea, Republic of
responsible party type: Principal Investigator
investigator affiliation: Asan Medical Center
investigator full name: Je-Hwan Lee
investigator title: M.D. Professor
mesh term: Preleukemia
mesh term: Myelodysplastic Syndromes
mesh term: Syndrome
mesh term: Cyclophosphamide