<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/NCT01700673</url>
  </required_header>
  <id_info>
    <org_study_id>J1240</org_study_id>
    <secondary_id>P01CA015396</secondary_id>
    <secondary_id>NA_00072223</secondary_id>
    <nct_id>NCT01700673</nct_id>
  </id_info>
  <brief_title>Phase II Study of Azacitidine and Sargramostim as Maintenance Treatment for Poor-Risk AML or MDS</brief_title>
  <official_title>A Phase II Study of 5-Azacitidine (5AC) in Combination With Sargramostim (GM-CSF) as Maintenance Treatment, After Definitive Therapy With Either Stem Cell Transplant (SCT) or Cytarabine-based Chemotherapy, in Patients With Poor-risk Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>National Cancer Institute (NCI)</agency>
      <agency_class>NIH</agency_class>
    </collaborator>
  </sponsors>
  <source>Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      To determine the impact of maintenance therapy in patients with MDS/AML in remission.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      We propose a phase II study to determine the impact of maintenance therapy with 5-azacytidine&#xD;
      and GM-CSF in patients with poor-risk AML or MDS, who are in remission after definitive&#xD;
      treatment with either stem cell transplant or cytarabine-based consolidation chemotherapy.&#xD;
&#xD;
      In order to precede relapse and to avoid lead time bias, treatment would need to commence&#xD;
      within 185 days of definitive therapy. Furthermore, approximately 50% of relapses occur&#xD;
      within the first year and up to 80% within two years after SCT, therefore we would limit the&#xD;
      duration of maintenance therapy to one year, followed by two years of follow-up.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">June 2013</start_date>
  <completion_date type="Actual">June 2020</completion_date>
  <primary_completion_date type="Actual">June 2020</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Two-year Relapse Free Survival of Patients</measure>
    <time_frame>2 year</time_frame>
    <description>To evaluate the two-year relapse-free survival (RFS) of patients with poor-risk Acute Myeloid Leukemia (AML) or Myelodysplasia (MDS), who receive maintenance treatment with 5-Azacytidine (5AC) in combination with sargramostim (GM-CSF) during remission, following definitive therapy with either a stem cell transplant (SCT) or cytarabine-based consolidation chemotherapy.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Hematologic Toxicity as Determined by Anemia</measure>
    <time_frame>1 year</time_frame>
    <description>Percentage of patients with anemia, the most commonly reported hematologic toxicity, after receiving the combination of Azacitidine and sargramostim</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>One-year RFS</measure>
    <time_frame>1 year</time_frame>
    <description>We will report the number of participants with one year RFS</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Overall Survival</measure>
    <time_frame>2 years</time_frame>
    <description>Percentage of participants with overall survival at 2 years.</description>
  </secondary_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">25</enrollment>
  <condition>Acute Myeloid Leukemia</condition>
  <condition>Myelodysplastic Syndrome</condition>
  <arm_group>
    <arm_group_label>Myeloablative BMT</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Azacitidine and sargramostim after myeloablative stem cell transplant</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Non-myeloablative BMT</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Azacitidine and sargramostim after non-myeloablative stem cell transplant</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Standard consolidation</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Azacitidine and sargramostim after standard consolidation</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Azacitidine</intervention_name>
    <description>Azacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles.</description>
    <arm_group_label>Myeloablative BMT</arm_group_label>
    <arm_group_label>Non-myeloablative BMT</arm_group_label>
    <arm_group_label>Standard consolidation</arm_group_label>
    <other_name>Vidaza</other_name>
    <other_name>5-azacytidine</other_name>
    <other_name>Azacytidine</other_name>
  </intervention>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Sargramostim</intervention_name>
    <description>Sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.</description>
    <arm_group_label>Myeloablative BMT</arm_group_label>
    <arm_group_label>Non-myeloablative BMT</arm_group_label>
    <arm_group_label>Standard consolidation</arm_group_label>
    <other_name>GM-CSF</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Age &gt; 6 months&#xD;
&#xD;
          2. Initial diagnosis of poor -risk AML or MDS (defined in section 3.2), treated with&#xD;
             either stem cell transplant or cytarabine-based consolidation chemotherapy, within the&#xD;
             past 60-185 days&#xD;
&#xD;
          3. ECOG performance status 0-2&#xD;
&#xD;
          4. No morphologic evidence of leukemia or active MDS as determined by JHH&#xD;
             Hematopathologist independent review of a bone marrow aspirate and biopsy done&#xD;
             following the completion of therapy and within 14 days prior to enrollment&#xD;
&#xD;
          5. Peripheral blood count recovery: Neutrophil count ≥ 1000 /µL, platelet count ≥ 50x 109&#xD;
             /µL without platelet transfusions, and adequate hematocrit independent of red cell&#xD;
             transfusions .&#xD;
&#xD;
          6. No evidence of extramedullary leukemia, such as CNS or soft tissue involvement&#xD;
&#xD;
          7. Adequate end organ function as measured by the following: AST and ALT &lt; 4 x normal,&#xD;
             total serum bilirubin &lt; 2 x upper limit normal (unless due to hemolysis, Gilbert's&#xD;
             syndrome, or ineffective erythropoiesis), creatinine &lt; 2 x upper limit of normal&#xD;
&#xD;
          8. Ability to give informed consent&#xD;
&#xD;
          9. In agreement to use an effective barrier method of birth control to avoid pregnancy&#xD;
             during the study and for a minimum of 30 days after study treatment, for all male and&#xD;
             female patients who are fertile&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Patients with untreated or uncontrolled infections&#xD;
&#xD;
          2. Patients with untreated or uncontrolled grade 3 or 4 GVHD&#xD;
&#xD;
          3. Pregnancy and lactation&#xD;
&#xD;
          4. Concurrent use of any other investigational agents.&#xD;
&#xD;
          5. Known HIV-positive patients.&#xD;
&#xD;
          6. Known hypersensitivity to 5AC or GM-CSF&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Months</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Margaret Showel, MD</last_name>
    <role>Study Chair</role>
    <affiliation>JHU</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins</name>
      <address>
        <city>Baltimore</city>
        <state>Maryland</state>
        <zip>21287</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>August 2021</verification_date>
  <study_first_submitted>October 2, 2012</study_first_submitted>
  <study_first_submitted_qc>October 3, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 4, 2012</study_first_posted>
  <results_first_submitted>August 6, 2021</results_first_submitted>
  <results_first_submitted_qc>August 6, 2021</results_first_submitted_qc>
  <results_first_posted type="Actual">September 2, 2021</results_first_posted>
  <last_update_submitted>September 23, 2022</last_update_submitted>
  <last_update_submitted_qc>September 23, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">October 18, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>maintenance treatment</keyword>
  <keyword>stem cell transplant</keyword>
  <keyword>cytarabine-based chemotherapy</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Leukemia</mesh_term>
    <mesh_term>Leukemia, Myeloid</mesh_term>
    <mesh_term>Leukemia, Myeloid, Acute</mesh_term>
    <mesh_term>Preleukemia</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>Azacitidine</mesh_term>
    <mesh_term>Sargramostim</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol and Statistical Analysis Plan</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>May 27, 2016</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/73/NCT01700673/Prot_SAP_000.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>Myeloablative BMT</title>
          <description>Azacitidine and sargramostim after myeloablative stem cell transplant&#xD;
Azacitidine: Azacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles.&#xD;
Sargramostim: Sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.</description>
        </group>
        <group group_id="P2">
          <title>Non-myeloablative BMT</title>
          <description>Azacitidine and sargramostim after non-myeloablative stem cell transplant&#xD;
Azacitidine: Azacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles.&#xD;
Sargramostim: Sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.</description>
        </group>
        <group group_id="P3">
          <title>Standard Consolidation</title>
          <description>Azacitidine and sargramostim after standard consolidation&#xD;
Azacitidine: Azacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles.&#xD;
Sargramostim: Sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="23"/>
                <participants group_id="P3" count="1"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="17"/>
                <participants group_id="P3" count="1"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="6"/>
                <participants group_id="P3" count="0"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Myeloablative BMT</title>
          <description>Azacitidine and sargramostim after myeloablative stem cell transplant&#xD;
Azacitidine: Azacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles.&#xD;
Sargramostim: Sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.</description>
        </group>
        <group group_id="B2">
          <title>Non-myeloablative BMT</title>
          <description>Azacitidine and sargramostim after non-myeloablative stem cell transplant&#xD;
Azacitidine: Azacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles.&#xD;
Sargramostim: Sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.</description>
        </group>
        <group group_id="B3">
          <title>Standard Consolidation</title>
          <description>Azacitidine and sargramostim after standard consolidation&#xD;
Azacitidine: Azacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles.&#xD;
Sargramostim: Sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.</description>
        </group>
        <group group_id="B4">
          <title>Total</title>
          <description>Total of all reporting groups</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="1"/>
            <count group_id="B2" value="23"/>
            <count group_id="B3" value="1"/>
            <count group_id="B4" value="25"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age, Customized</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <title>&lt;=18 years</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Between 18 and 72 years</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="23"/>
                    <measurement group_id="B3" value="1"/>
                    <measurement group_id="B4" value="25"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>&gt;=73 years</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="11"/>
                    <measurement group_id="B3" value="1"/>
                    <measurement group_id="B4" value="12"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="12"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="13"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>American Indian or Alaska Native</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="2"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="18"/>
                    <measurement group_id="B3" value="1"/>
                    <measurement group_id="B4" value="20"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Two-year Relapse Free Survival of Patients</title>
        <description>To evaluate the two-year relapse-free survival (RFS) of patients with poor-risk Acute Myeloid Leukemia (AML) or Myelodysplasia (MDS), who receive maintenance treatment with 5-Azacytidine (5AC) in combination with sargramostim (GM-CSF) during remission, following definitive therapy with either a stem cell transplant (SCT) or cytarabine-based consolidation chemotherapy.</description>
        <time_frame>2 year</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Non-myeloablative BMT</title>
            <description>Azacitidine and sargramostim after non-myeloablative stem cell transplant</description>
          </group>
          <group group_id="O2">
            <title>Myeloablative BMT</title>
            <description>Azacitidine and sargramostim after myeloablative stem cell transplant</description>
          </group>
          <group group_id="O3">
            <title>Standard Consolidation</title>
            <description>Azacitidine and sargramostim after standard consolidation</description>
          </group>
        </group_list>
        <measure>
          <title>Two-year Relapse Free Survival of Patients</title>
          <description>To evaluate the two-year relapse-free survival (RFS) of patients with poor-risk Acute Myeloid Leukemia (AML) or Myelodysplasia (MDS), who receive maintenance treatment with 5-Azacytidine (5AC) in combination with sargramostim (GM-CSF) during remission, following definitive therapy with either a stem cell transplant (SCT) or cytarabine-based consolidation chemotherapy.</description>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="17"/>
                <count group_id="O2" value="1"/>
                <count group_id="O3" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="8"/>
                    <measurement group_id="O2" value="1"/>
                    <measurement group_id="O3" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Hematologic Toxicity as Determined by Anemia</title>
        <description>Percentage of patients with anemia, the most commonly reported hematologic toxicity, after receiving the combination of Azacitidine and sargramostim</description>
        <time_frame>1 year</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Non-myeloablative BMT</title>
            <description>Azacitidine and sargramostim after non-myeloablative stem cell transplant</description>
          </group>
          <group group_id="O2">
            <title>Myeloablative BMT</title>
            <description>Azacitidine and sargramostim after myeloablative stem cell transplant</description>
          </group>
          <group group_id="O3">
            <title>Standard Consolidation</title>
            <description>Azacitidine and sargramostim after standard consolidation</description>
          </group>
        </group_list>
        <measure>
          <title>Hematologic Toxicity as Determined by Anemia</title>
          <description>Percentage of patients with anemia, the most commonly reported hematologic toxicity, after receiving the combination of Azacitidine and sargramostim</description>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="17"/>
                <count group_id="O2" value="1"/>
                <count group_id="O3" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="8"/>
                    <measurement group_id="O2" value="1"/>
                    <measurement group_id="O3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>One-year RFS</title>
        <description>We will report the number of participants with one year RFS</description>
        <time_frame>1 year</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Non-myeloablative BMT</title>
            <description>Azacitidine and sargramostim after non-myeloablative stem cell transplant</description>
          </group>
          <group group_id="O2">
            <title>Myeloablative BMT</title>
            <description>Azacitidine and sargramostim after myeloablative stem cell transplant</description>
          </group>
          <group group_id="O3">
            <title>Standard Consolidation</title>
            <description>Azacitidine and sargramostim after standard consolidation</description>
          </group>
        </group_list>
        <measure>
          <title>One-year RFS</title>
          <description>We will report the number of participants with one year RFS</description>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="17"/>
                <count group_id="O2" value="1"/>
                <count group_id="O3" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="12"/>
                    <measurement group_id="O2" value="1"/>
                    <measurement group_id="O3" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Overall Survival</title>
        <description>Percentage of participants with overall survival at 2 years.</description>
        <time_frame>2 years</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Non-myeloablative</title>
            <description>Azacitidine and sargramostim after non-myeloablative stem cell transplant</description>
          </group>
          <group group_id="O2">
            <title>Myeloablative BMT</title>
            <description>Azacitidine and sargramostim after myeloablative stem cell transplant</description>
          </group>
          <group group_id="O3">
            <title>Standard Consolidation</title>
            <description>Azacitidine and sargramostim after standard consolidation</description>
          </group>
        </group_list>
        <measure>
          <title>Overall Survival</title>
          <description>Percentage of participants with overall survival at 2 years.</description>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="11"/>
                <count group_id="O2" value="1"/>
                <count group_id="O3" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="6"/>
                    <measurement group_id="O2" value="1"/>
                    <measurement group_id="O3" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>2 years</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Non-myeloablative BMT</title>
          <description>Azacitidine and sargramostim after non-myeloablative stem cell transplant</description>
        </group>
        <group group_id="E2">
          <title>Myeloablative BMT</title>
          <description>Azacitidine and sargramostim after myeloablative stem cell transplant</description>
        </group>
        <group group_id="E3">
          <title>Standard Consolidation</title>
          <description>Azacitidine and sargramostim after standard consolidation</description>
        </group>
      </group_list>
      <serious_events>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="8" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="4" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>anemia</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>eosinophilia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>neutropenia</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>leukopenia</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>thrombocytopenia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>infections</sub_title>
                <counts group_id="E1" subjects_affected="4" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Graft-vs-host disease</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>rash</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="17" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>ALT increase</sub_title>
                <counts group_id="E1" events="12" subjects_affected="3" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>anemia</sub_title>
                <counts group_id="E1" events="21" subjects_affected="8" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>edema</sub_title>
                <counts group_id="E1" events="5" subjects_affected="2" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" events="2" subjects_affected="1" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>creatinine increase</sub_title>
                <counts group_id="E1" events="5" subjects_affected="2" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>eosiniphilia</sub_title>
                <counts group_id="E1" events="3" subjects_affected="2" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>neutropenia</sub_title>
                <counts group_id="E1" events="13" subjects_affected="5" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>leukopenia</sub_title>
                <counts group_id="E1" events="12" subjects_affected="4" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>thrombocytopenia</sub_title>
                <counts group_id="E1" events="23" subjects_affected="8" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Ear and labyrinth disorders</title>
            <event_list>
              <event>
                <sub_title>dizziness</sub_title>
                <counts group_id="E1" events="6" subjects_affected="4" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>nausea</sub_title>
                <counts group_id="E1" events="13" subjects_affected="8" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="1"/>
                <counts group_id="E3" events="2" subjects_affected="1" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>anorexia</sub_title>
                <counts group_id="E1" events="6" subjects_affected="3" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>constipation</sub_title>
                <counts group_id="E1" events="16" subjects_affected="4" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>diarrhea</sub_title>
                <counts group_id="E1" events="15" subjects_affected="8" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>dyspepsia</sub_title>
                <counts group_id="E1" events="4" subjects_affected="4" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>fatigue</sub_title>
                <counts group_id="E1" events="27" subjects_affected="9" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>infections</sub_title>
                <counts group_id="E1" events="44" subjects_affected="14" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>myalgia</sub_title>
                <counts group_id="E1" events="6" subjects_affected="4" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>bone pain</sub_title>
                <counts group_id="E1" events="8" subjects_affected="7" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>chills</sub_title>
                <counts group_id="E1" events="3" subjects_affected="2" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>headache</sub_title>
                <counts group_id="E1" events="8" subjects_affected="6" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>insomnia</sub_title>
                <counts group_id="E1" events="9" subjects_affected="4" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>cough</sub_title>
                <counts group_id="E1" events="14" subjects_affected="8" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>dyspnea</sub_title>
                <counts group_id="E1" events="12" subjects_affected="4" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>injection site erythema</sub_title>
                <counts group_id="E1" events="17" subjects_affected="12" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>graft-vs-host disease</sub_title>
                <counts group_id="E1" events="9" subjects_affected="4" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>rash</sub_title>
                <counts group_id="E1" events="19" subjects_affected="8" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" events="3" subjects_affected="1" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>pruritis</sub_title>
                <counts group_id="E1" events="13" subjects_affected="6" subjects_at_risk="17"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" events="4" subjects_affected="1" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>All Principal Investigators ARE employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. </restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Jonathan Webster, MD</name_or_title>
      <organization>Johns Hopkins University School of Medicine, Division of Hematologic Malignancies</organization>
      <phone>4106149106</phone>
      <email>jwebst17@jhmi.edu</email>
    </point_of_contact>
  </clinical_results>
</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/NCT01700673
org study id: J1240
secondary id: P01CA015396
secondary id: NA_00072223
nct id: NCT01700673
lead sponsor: 
collaborator: 
has dmc: No
To determine the impact of maintenance therapy in patients with MDS/AML in remission.
We propose a phase II study to determine the impact of maintenance therapy with 5-azacytidine
      and GM-CSF in patients with poor-risk AML or MDS, who are in remission after definitive
      treatment with either stem cell transplant or cytarabine-based consolidation chemotherapy.

      In order to precede relapse and to avoid lead time bias, treatment would need to commence
      within 185 days of definitive therapy. Furthermore, approximately 50% of relapses occur
      within the first year and up to 80% within two years after SCT, therefore we would limit the
      duration of maintenance therapy to one year, followed by two years of follow-up.
allocation: Non-Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Two-year Relapse Free Survival of Patients
time frame: 2 year
description: To evaluate the two-year relapse-free survival (RFS) of patients with poor-risk Acute Myeloid Leukemia (AML) or Myelodysplasia (MDS), who receive maintenance treatment with 5-Azacytidine (5AC) in combination with sargramostim (GM-CSF) during remission, following definitive therapy with either a stem cell transplant (SCT) or cytarabine-based consolidation chemotherapy.
measure: Hematologic Toxicity as Determined by Anemia
time frame: 1 year
description: Percentage of patients with anemia, the most commonly reported hematologic toxicity, after receiving the combination of Azacitidine and sargramostim
measure: One-year RFS
time frame: 1 year
description: We will report the number of participants with one year RFS
measure: Overall Survival
time frame: 2 years
description: Percentage of participants with overall survival at 2 years.
arm group label: Myeloablative BMT
arm group type: Experimental
description: Azacitidine and sargramostim after myeloablative stem cell transplant
arm group label: Non-myeloablative BMT
arm group type: Experimental
description: Azacitidine and sargramostim after non-myeloablative stem cell transplant
arm group label: Standard consolidation
arm group type: Experimental
description: Azacitidine and sargramostim after standard consolidation
intervention type: Drug
intervention name: Azacitidine
description: Azacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
arm group label: Myeloablative BMT
arm group label: Non-myeloablative BMT
arm group label: Standard consolidation
other name: Vidaza
other name: 5-azacytidine
other name: Azacytidine
intervention type: Biological
intervention name: Sargramostim
description: Sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
arm group label: Myeloablative BMT
arm group label: Non-myeloablative BMT
arm group label: Standard consolidation
other name: GM-CSF
criteria: 
gender: All
minimum age: 6 Months
maximum age: N/A
healthy volunteers: No
last name: Margaret Showel, MD
role: Study Chair
affiliation: JHU
facility: 
country: United States
responsible party type: Sponsor
mesh term: Leukemia
mesh term: Leukemia, Myeloid
mesh term: Leukemia, Myeloid, Acute
mesh term: Preleukemia
mesh term: Myelodysplastic Syndromes
mesh term: Azacitidine
mesh term: Sargramostim
sharing ipd: No
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 