<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/NCT02221310</url>
  </required_header>
  <id_info>
    <org_study_id>NYMC-515</org_study_id>
    <secondary_id>L-10,349</secondary_id>
    <nct_id>NCT02221310</nct_id>
  </id_info>
  <brief_title>Immunochemotherapy and AlloSCT in Patients With High Risk CD33+ AML/MDS</brief_title>
  <official_title>A Pilot Study of Gemtuzumab Ozogamicin in Combination With Busulfan and Cyclophosphamide (Immunochemotherapy) and Allogeneic Stem Cell Transplantation in Patients With High Risk Acute Myelogenous Leukemia and Myelodysplastic Syndrome</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>New York Medical College</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>New York Medical College</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Targeted immune therapy with gemtuzumab ozogamicin (Mylotarg) in combination with&#xD;
      chemotherapy followed by allogeneic stem cell transplantation will be given to patients with&#xD;
      high risk acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS).&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Gemtuzumab ozogamicin in combination with busulfan and cyclophosphamide (immunochemotherapy)&#xD;
      conditioning followed by allogeneic stem cell transplantation + anti-thymocyte globulin&#xD;
      (unrelated donors only) in patients with high risk CD33+AML or MDS meeting eligibility&#xD;
      criteria.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date>November 2011</start_date>
  <completion_date type="Anticipated">June 2024</completion_date>
  <primary_completion_date type="Anticipated">June 2023</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>Response rate</measure>
    <time_frame>1 year</time_frame>
    <description>overall response rate (CR + PR) in patients receiving GO, busulfan and cyclophosphamide and AlloSCT in patients with measurable disease (relapse/refractory) with high risk CD33+ AML/MDS</description>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">25</enrollment>
  <condition>Acute Myelogenous Leukemia</condition>
  <condition>Myelodysplastic Syndrome</condition>
  <arm_group>
    <arm_group_label>Gemtuzumab Ozogamicin</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Conditioning therapy with Gemtuzumab Ozogamicin in combination with busulfan and cyclophosphamide chemotherapy followed by allogeneic stem cell transplantation.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Gemtuzumab Ozogamicin</intervention_name>
    <description>Gemtuzumab Ozogamicin 7.5 mg/m^2/dose given IV over 2 hours once during conditioning</description>
    <arm_group_label>Gemtuzumab Ozogamicin</arm_group_label>
    <other_name>Mylotarg</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Disease Status:&#xD;
&#xD;
               -  History of AML Induction/Reinduction Failure&#xD;
&#xD;
               -  AML 1st Complete Remission (CR) with poor cytogenetics&#xD;
&#xD;
               -  AML 2nd CR with minimal residual disease (MRD)&#xD;
&#xD;
               -  AML 3rd CR&#xD;
&#xD;
               -  AML in refractory relapse but ≤25% bone marrow leukemia blasts&#xD;
&#xD;
               -  MDS with &gt;6% bone marrow blasts at diagnosis&#xD;
&#xD;
               -  Secondary MDS with ≤5% bone marrow myeloblasts at diagnosis&#xD;
&#xD;
        Disease Immunophenotype:&#xD;
&#xD;
        • Disease must express a minimum of &gt;/= 10% CD33+ for patients with AML&#xD;
&#xD;
        Organ Function:&#xD;
&#xD;
        • Adequate renal function, adequate liver function, adequate cardiac function, adequate&#xD;
        pulmonary function Age: ≤25 years of age Donor: matched family donor, unrelated cord blood&#xD;
        donor, unrelated adult donor&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Patients with active central nervous system (CNS) AML/MDS disease at time of&#xD;
             conditioning therapy&#xD;
&#xD;
          -  Female patients who are pregnant&#xD;
&#xD;
          -  Karnofsky &lt;50% or Lansky &lt;50% if 10 years or less&#xD;
&#xD;
          -  Age &gt;25 years&#xD;
&#xD;
          -  Has received gemtuzumab in the previous 30 days or has not recovered from prior&#xD;
             gemtuzumab therapy.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>25 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Mitchell S Cairo, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>New York Medical College</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>New York Medical College</name>
      <address>
        <city>Valhalla</city>
        <state>New York</state>
        <zip>10595</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <reference>
    <citation>Satwani P, Bhatia M, Garvin JH Jr, George D, Dela Cruz F, Le Gall J, Jin Z, Schwartz J, Duffy D, van de Ven C, Foley S, Hawks R, Morris E, Baxter-Lowe LA, Cairo MS. A Phase I study of gemtuzumab ozogamicin (GO) in combination with busulfan and cyclophosphamide (Bu/Cy) and allogeneic stem cell transplantation in children with poor-risk CD33+ AML: a new targeted immunochemotherapy myeloablative conditioning (MAC) regimen. Biol Blood Marrow Transplant. 2012 Feb;18(2):324-9. doi: 10.1016/j.bbmt.2011.11.007. Epub 2011 Nov 9.</citation>
    <PMID>22079471</PMID>
  </reference>
  <verification_date>September 2022</verification_date>
  <study_first_submitted>July 31, 2014</study_first_submitted>
  <study_first_submitted_qc>August 18, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">August 20, 2014</study_first_posted>
  <last_update_submitted>September 26, 2022</last_update_submitted>
  <last_update_submitted_qc>September 26, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">September 27, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>New York Medical College</investigator_affiliation>
    <investigator_full_name>Mitchell Cairo</investigator_full_name>
    <investigator_title>Principal Investigator</investigator_title>
  </responsible_party>
  <keyword>targeted therapy</keyword>
  <keyword>leukemia</keyword>
  <keyword>MDS</keyword>
  <keyword>immune therapy</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Leukemia</mesh_term>
    <mesh_term>Preleukemia</mesh_term>
    <mesh_term>Leukemia, Myeloid</mesh_term>
    <mesh_term>Leukemia, Myeloid, Acute</mesh_term>
    <mesh_term>Myelodysplastic Syndromes</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Gemtuzumab</mesh_term>
  </intervention_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/NCT02221310
org study id: NYMC-515
secondary id: L-10,349
nct id: NCT02221310
lead sponsor: 
has dmc: No
Targeted immune therapy with gemtuzumab ozogamicin (Mylotarg) in combination with
      chemotherapy followed by allogeneic stem cell transplantation will be given to patients with
      high risk acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS).
Gemtuzumab ozogamicin in combination with busulfan and cyclophosphamide (immunochemotherapy)
      conditioning followed by allogeneic stem cell transplantation + anti-thymocyte globulin
      (unrelated donors only) in patients with high risk CD33+AML or MDS meeting eligibility
      criteria.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Response rate
time frame: 1 year
description: overall response rate (CR + PR) in patients receiving GO, busulfan and cyclophosphamide and AlloSCT in patients with measurable disease (relapse/refractory) with high risk CD33+ AML/MDS
arm group label: Gemtuzumab Ozogamicin
arm group type: Experimental
description: Conditioning therapy with Gemtuzumab Ozogamicin in combination with busulfan and cyclophosphamide chemotherapy followed by allogeneic stem cell transplantation.
intervention type: Drug
intervention name: Gemtuzumab Ozogamicin
description: Gemtuzumab Ozogamicin 7.5 mg/m^2/dose given IV over 2 hours once during conditioning
arm group label: Gemtuzumab Ozogamicin
other name: Mylotarg
criteria: 
gender: All
minimum age: N/A
maximum age: 25 Years
healthy volunteers: No
last name: Mitchell S Cairo, MD
role: Principal Investigator
affiliation: New York Medical College
facility: 
country: United States
citation: Satwani P, Bhatia M, Garvin JH Jr, George D, Dela Cruz F, Le Gall J, Jin Z, Schwartz J, Duffy D, van de Ven C, Foley S, Hawks R, Morris E, Baxter-Lowe LA, Cairo MS. A Phase I study of gemtuzumab ozogamicin (GO) in combination with busulfan and cyclophosphamide (Bu/Cy) and allogeneic stem cell transplantation in children with poor-risk CD33+ AML: a new targeted immunochemotherapy myeloablative conditioning (MAC) regimen. Biol Blood Marrow Transplant. 2012 Feb;18(2):324-9. doi: 10.1016/j.bbmt.2011.11.007. Epub 2011 Nov 9.
PMID: 22079471
responsible party type: Principal Investigator
investigator affiliation: New York Medical College
investigator full name: Mitchell Cairo
investigator title: Principal Investigator
mesh term: Leukemia
mesh term: Preleukemia
mesh term: Leukemia, Myeloid
mesh term: Leukemia, Myeloid, Acute
mesh term: Myelodysplastic Syndromes
mesh term: Gemtuzumab
sharing ipd: Undecided