<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/NCT01720225</url>
  </required_header>
  <id_info>
    <org_study_id>2012-0507</org_study_id>
    <secondary_id>NCI-2012-02215</secondary_id>
    <nct_id>NCT01720225</nct_id>
  </id_info>
  <brief_title>Decitabine Versus Azacitidine in Myelodysplastic Syndrome Patients With Low and Intermediate-1 Risk</brief_title>
  <official_title>Phase II Randomized Study of Lower Doses of Decitabine (DAC; 20 mg/m2 IV Daily for 3 Days Every Month) Versus Azacitidine (AZA; 75 mg/m2 SC/IV Daily for 3 Days Every Month) in Myelodysplastic Syndrome (MDS) Patients With Low and Intermediate-1 Risk Disease</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>M.D. Anderson Cancer Center</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>M.D. Anderson Cancer Center</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The goal of this clinical research study is to compare how two different drugs, decitabine&#xD;
      and azacitidine, when given on a shorter than standard dosing schedule can help to control&#xD;
      MDS. The safety of the drugs will also be studied.&#xD;
&#xD;
      Decitabine is designed to damage the DNA (the genetic material) of cells, which may cause&#xD;
      cancer cells to die.&#xD;
&#xD;
      Azacitidine is designed to block certain proteins in cancer cells whose job is to stop the&#xD;
      function of the tumor-fighting proteins. By blocking the &quot;bad&quot; proteins, the tumor-fighting&#xD;
      genes may be able to work better. This could cause the cancer cells to die.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Study Groups:&#xD;
&#xD;
      If you are found to be eligible to take part in this study, you will be randomly assigned (as&#xD;
      in the flip of a coin) to 1 of 2 groups:&#xD;
&#xD;
        -  If you are in Group 1, you will receive decitabine by vein over about 1 hour.&#xD;
&#xD;
        -  If you are in Group 2, you will receive azacitidine either as an injection under your&#xD;
           skin or through a vein. If by vein, the infusion will take about an hour.&#xD;
&#xD;
      At first, there will be an equal chance of being assigned to either group. However, as the&#xD;
      study goes on and more information becomes available, the chance of being assigned to the&#xD;
      group that has shown the most effectiveness will increase. However, once you are already&#xD;
      enrolled and assigned to a group, you will not be eligible to change groups.&#xD;
&#xD;
      Study Drug Administration:&#xD;
&#xD;
      Each cycle is 28 days.&#xD;
&#xD;
      You will receive the study drug on Days 1-3 of every cycle and you will receive at least 2&#xD;
      cycles of study drug.&#xD;
&#xD;
      Study Visits:&#xD;
&#xD;
      Every 7-14 days, blood (about 2 tablespoons) will be drawn for routine tests.&#xD;
&#xD;
      At the end of Cycle 2, you will have a bone marrow biopsy and/or aspirate to check the status&#xD;
      of the disease. This will then be repeated every 2-4 cycles until any point that the disease&#xD;
      appears to have responded to the study drug, then as often as the study doctor thinks is&#xD;
      necessary. To collect a bone marrow biopsy/aspirate, an area of the hip bone is numbed with&#xD;
      anesthetic, and a small amount of bone and/or bone marrow is withdrawn through a large&#xD;
      needle.&#xD;
&#xD;
      After Cycle 3:&#xD;
&#xD;
      If the study doctor decides it is acceptable, you may be allowed to receive treatment from&#xD;
      your local cancer doctor. However, you have to return to Houston at least every 3 months for&#xD;
      your study visits.&#xD;
&#xD;
      The frequency of the visits will depend on what the doctor thinks is in your best interest.&#xD;
&#xD;
      Length of Study:&#xD;
&#xD;
      You may continue taking the study drug for as long as the doctor thinks it is in your best&#xD;
      interest. You will no longer be able to take the study drug if the disease gets worse, if&#xD;
      intolerable side effects occur, or if you are unable to follow study directions.&#xD;
&#xD;
      Follow-Up Visits:&#xD;
&#xD;
      One (1) time every 3 months after your last dose of study drug, you will return to the clinic&#xD;
      for a bone marrow aspiration to check the status of the disease.&#xD;
&#xD;
      Other Information:&#xD;
&#xD;
      You may be given other drugs to help prevent side effects. The study staff will tell you&#xD;
      about these drugs, how they will be given, and the possible risks.&#xD;
&#xD;
      This is an investigational study. Decitabine and Azacitidine are both FDA approved and&#xD;
      commercially available for use in patients with MDS.&#xD;
&#xD;
      Up to 120 patients will take part in this study. All will be enrolled at MD Anderson.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">November 6, 2012</start_date>
  <completion_date type="Actual">January 8, 2020</completion_date>
  <primary_completion_date type="Actual">January 8, 2020</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Participants With a Response</measure>
    <time_frame>56 days</time_frame>
    <description>Overall Response = complete remission (CR) + partial remission (PR) + marrow CR (mCR) + hematologic improvement (HI). CR is normalization of peripheral blood and bone marrow with &lt;5% bone marrow blasts, a peripheral blood granulocyte count &gt; (1.0 x 10^9/L, and platelet count &gt; 100 x 10^9/L). PR is same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if &lt;15% at start of treatment. Marrow CR is blasts &lt;/= 5% and decreased by &gt;/=50% from baseline. HI is platelets increase by 50% and to above 30 x 10^9/L untransfused (if lower than that pre-therapy; or hemoglobin increase by 2 g/dl; or transfusion independent; or splenomegaly reduction by &gt; 50%; or monocytosis reduction by &gt; 50% if pretreatment &gt; 5 X1 0^9/L.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Number of Participants Who Became Transfusion Independent</measure>
    <time_frame>8 weeks</time_frame>
    <description>Participants who were transfusion dependent at baseline prior to starting therapy on the Decitabine or Azacitidine arm will be analyzed for transfusion independence. Transfusion independence defined as being transfusion-free for ≥8 consecutive weeks between the first dose and study treatment discontinuation.</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">113</enrollment>
  <condition>Leukemia</condition>
  <arm_group>
    <arm_group_label>Decitabine</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Azacitidine</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Decitabine</intervention_name>
    <description>20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.</description>
    <arm_group_label>Decitabine</arm_group_label>
    <other_name>DAC</other_name>
    <other_name>Dacogen</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Azacitidine</intervention_name>
    <description>75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.</description>
    <arm_group_label>Azacitidine</arm_group_label>
    <other_name>AZA</other_name>
    <other_name>5-azacytidine</other_name>
    <other_name>5-aza</other_name>
    <other_name>Vidaza</other_name>
    <other_name>5-AZC</other_name>
    <other_name>AZA-CR</other_name>
    <other_name>Ladakamycin</other_name>
    <other_name>NSC-102816</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Sign an Institutional Review Board (IRB)-approved informed consent document.&#xD;
&#xD;
          2. Age &gt;/= than18 years&#xD;
&#xD;
          3. de novo or secondary International Prostate Symptom Score (IPSS) low- or&#xD;
             intermediate-1 - risk MDS, including CMML&#xD;
&#xD;
          4. Eastern Cooperative Oncology Group (ECOG) performance status of &lt;/= 3 at study entry.&#xD;
&#xD;
          5. Organ function as defined: Serum creatinine &lt;/= 3 x Upper Limit of Normal (ULN), Total&#xD;
             bilirubin &lt;/= 2 x ULN, Alanine transaminase (ALT) (SGPT) &lt;/= 2 x ULN&#xD;
&#xD;
          6. Women of childbearing potential must have a negative serum or urine pregnancy test&#xD;
             within 7 days and will also need to use contraceptives. Men must agree not to father a&#xD;
             child and agree to use a condom if his partner is of child bearing potential.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Breast feeding females&#xD;
&#xD;
          2. Prior therapy with decitabine or azacitidine&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Elias Jabbour, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>M.D. Anderson Cancer Center</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>University of Texas MD Anderson Cancer Center</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>http://www.mdanderson.org</url>
    <description>University of Texas MD Anderson Cancer Center Website</description>
  </link>
  <verification_date>December 2020</verification_date>
  <study_first_submitted>October 31, 2012</study_first_submitted>
  <study_first_submitted_qc>October 31, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">November 2, 2012</study_first_posted>
  <results_first_submitted>December 1, 2020</results_first_submitted>
  <results_first_submitted_qc>December 1, 2020</results_first_submitted_qc>
  <results_first_posted type="Actual">December 24, 2020</results_first_posted>
  <last_update_submitted>December 1, 2020</last_update_submitted>
  <last_update_submitted_qc>December 1, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">December 24, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Leukemia</keyword>
  <keyword>Myelodysplastic syndromes</keyword>
  <keyword>MDS</keyword>
  <keyword>Low and Intermediate-1 Risk Disease</keyword>
  <keyword>Decitabine</keyword>
  <keyword>DAC</keyword>
  <keyword>Dacogen</keyword>
  <keyword>Azacitidine</keyword>
  <keyword>AZA</keyword>
  <keyword>5-azacytidine</keyword>
  <keyword>5-aza</keyword>
  <keyword>Vidaza</keyword>
  <keyword>5-AZC</keyword>
  <keyword>AZA-CR</keyword>
  <keyword>Ladakamycin</keyword>
  <keyword>NSC-102816</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>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Azacitidine</mesh_term>
    <mesh_term>Decitabine</mesh_term>
  </intervention_browse>
  <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 13, 2015</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/25/NCT01720225/Prot_SAP_000.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <recruitment_details>Recruitment Period: November 2012 to February 2016</recruitment_details>
      <group_list>
        <group group_id="P1">
          <title>Decitabine</title>
          <description>Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.&#xD;
Decitabine: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.</description>
        </group>
        <group group_id="P2">
          <title>Azacitidine</title>
          <description>Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.&#xD;
Azacitidine: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="73"/>
                <participants group_id="P2" count="40"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="70"/>
                <participants group_id="P2" count="39"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
                <participants group_id="P2" count="1"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Withdrawal by Subject</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Did not complete 2 cycles of therapy</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Decitabine</title>
          <description>Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.&#xD;
Decitabine: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.</description>
        </group>
        <group group_id="B2">
          <title>Azacitidine</title>
          <description>Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.&#xD;
Azacitidine: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.</description>
        </group>
        <group group_id="B3">
          <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="73"/>
            <count group_id="B2" value="40"/>
            <count group_id="B3" value="113"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>&lt;=18 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Between 18 and 65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="26"/>
                    <measurement group_id="B2" value="12"/>
                    <measurement group_id="B3" value="38"/>
                  </measurement_list>
                </category>
                <category>
                  <title>&gt;=65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="47"/>
                    <measurement group_id="B2" value="28"/>
                    <measurement group_id="B3" value="75"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Median</param>
          <dispersion>Full Range</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="70" lower_limit="44" upper_limit="88"/>
                    <measurement group_id="B2" value="70" lower_limit="53" upper_limit="84"/>
                    <measurement group_id="B3" value="70" lower_limit="44" upper_limit="88"/>
                  </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="24"/>
                    <measurement group_id="B2" value="16"/>
                    <measurement group_id="B3" value="40"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="49"/>
                    <measurement group_id="B2" value="24"/>
                    <measurement group_id="B3" value="73"/>
                  </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_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </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_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="3"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="71"/>
                    <measurement group_id="B2" value="39"/>
                    <measurement group_id="B3" value="110"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>United States</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="73"/>
                    <measurement group_id="B2" value="40"/>
                    <measurement group_id="B3" value="113"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Participants With a Response</title>
        <description>Overall Response = complete remission (CR) + partial remission (PR) + marrow CR (mCR) + hematologic improvement (HI). CR is normalization of peripheral blood and bone marrow with &lt;5% bone marrow blasts, a peripheral blood granulocyte count &gt; (1.0 x 10^9/L, and platelet count &gt; 100 x 10^9/L). PR is same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if &lt;15% at start of treatment. Marrow CR is blasts &lt;/= 5% and decreased by &gt;/=50% from baseline. HI is platelets increase by 50% and to above 30 x 10^9/L untransfused (if lower than that pre-therapy; or hemoglobin increase by 2 g/dl; or transfusion independent; or splenomegaly reduction by &gt; 50%; or monocytosis reduction by &gt; 50% if pretreatment &gt; 5 X1 0^9/L.</description>
        <time_frame>56 days</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Decitabine</title>
            <description>Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.&#xD;
Decitabine: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.</description>
          </group>
          <group group_id="O2">
            <title>Azacitidine</title>
            <description>Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.&#xD;
Azacitidine: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.</description>
          </group>
        </group_list>
        <measure>
          <title>Participants With a Response</title>
          <description>Overall Response = complete remission (CR) + partial remission (PR) + marrow CR (mCR) + hematologic improvement (HI). CR is normalization of peripheral blood and bone marrow with &lt;5% bone marrow blasts, a peripheral blood granulocyte count &gt; (1.0 x 10^9/L, and platelet count &gt; 100 x 10^9/L). PR is same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if &lt;15% at start of treatment. Marrow CR is blasts &lt;/= 5% and decreased by &gt;/=50% from baseline. HI is platelets increase by 50% and to above 30 x 10^9/L untransfused (if lower than that pre-therapy; or hemoglobin increase by 2 g/dl; or transfusion independent; or splenomegaly reduction by &gt; 50%; or monocytosis reduction by &gt; 50% if pretreatment &gt; 5 X1 0^9/L.</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="70"/>
                <count group_id="O2" value="39"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="49"/>
                    <measurement group_id="O2" value="19"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Participants Who Became Transfusion Independent</title>
        <description>Participants who were transfusion dependent at baseline prior to starting therapy on the Decitabine or Azacitidine arm will be analyzed for transfusion independence. Transfusion independence defined as being transfusion-free for ≥8 consecutive weeks between the first dose and study treatment discontinuation.</description>
        <time_frame>8 weeks</time_frame>
        <population>Participants analyzed for Transfusion Independence must have been transfusion dependent at baseline. Thirty-seven participants in the Decitabine arm and nineteen participants in the Azacitidine arm where transfusion dependent prior to treatment on this study.</population>
        <group_list>
          <group group_id="O1">
            <title>Decitabine</title>
            <description>Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.&#xD;
Decitabine: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.</description>
          </group>
          <group group_id="O2">
            <title>Azacitidine</title>
            <description>Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.&#xD;
Azacitidine: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants Who Became Transfusion Independent</title>
          <description>Participants who were transfusion dependent at baseline prior to starting therapy on the Decitabine or Azacitidine arm will be analyzed for transfusion independence. Transfusion independence defined as being transfusion-free for ≥8 consecutive weeks between the first dose and study treatment discontinuation.</description>
          <population>Participants analyzed for Transfusion Independence must have been transfusion dependent at baseline. Thirty-seven participants in the Decitabine arm and nineteen participants in the Azacitidine arm where transfusion dependent prior to treatment on this study.</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="37"/>
                <count group_id="O2" value="19"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="12"/>
                    <measurement group_id="O2" value="3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>3 years, 3 months</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Decitabine</title>
          <description>Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.&#xD;
Decitabine: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.</description>
        </group>
        <group group_id="E2">
          <title>Azacitidine</title>
          <description>Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.&#xD;
Azacitidine: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>CTCAE (4.0)</default_vocab>
        <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="7" subjects_at_risk="73"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="32" subjects_at_risk="73"/>
                <counts group_id="E2" subjects_affected="14" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>Neutropenic Fever</sub_title>
                <counts group_id="E1" events="17" subjects_affected="9" subjects_at_risk="73"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Cardiac disorders</title>
            <event_list>
              <event>
                <sub_title>Heart Failure</sub_title>
                <counts group_id="E1" events="3" subjects_affected="2" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Myocardial Infarction</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Pericarditis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Ear and labyrinth disorders</title>
            <event_list>
              <event>
                <sub_title>Otitis Externa</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Ascites</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Diverticulitis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="73"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Gastroenteritis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Gastrointestinal Bleed</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Nausea</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Rectal Hermorrhage</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Sialadenitis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="73"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Small Intestine Obstruction</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="73"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Back Pain</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Chest Pain</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Edema Limbs</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="73"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Fever</sub_title>
                <counts group_id="E1" events="3" subjects_affected="2" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Pain</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Pain Abdominal Abcess</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Pain Right Extremity</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="73"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Right Hip Pain</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="73"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Appendicitis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Cellulitis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="73"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Ear Infection</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Tooth Infection</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Joint Infection</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Leg Infection</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Lung Infection</sub_title>
                <counts group_id="E1" events="3" subjects_affected="3" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Pneumonia</sub_title>
                <counts group_id="E1" events="7" subjects_affected="6" subjects_at_risk="73"/>
                <counts group_id="E2" events="4" subjects_affected="1" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Salmonella Bactremia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="73"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Sepsis</sub_title>
                <counts group_id="E1" events="4" subjects_affected="4" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Submandibular Cellulitis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Urinary Tract Infection</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="73"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Wound Infection</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Fall</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="73"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Food Poisoning</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="73"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Altered Mental Status</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="73"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Intracranial Hemorrhage</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Syncopal episode</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Renal and urinary disorders</title>
            <event_list>
              <event>
                <sub_title>Acute Kidney Injury</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Renal Failure</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Reproductive system and breast disorders</title>
            <event_list>
              <event>
                <sub_title>Vaginal Fistual</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="73"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Acute Respiratory Distress Syndrome</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Bronchopulmonary Hemorrhage</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="73"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Hemoptysis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Left plerual effusion</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="73"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Right atelectasis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Right Pleural Effusion</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Respiratory Failure</sub_title>
                <counts group_id="E1" events="3" subjects_affected="3" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Shortness of Breath</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Groin Abcess</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Skin Ulceration</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Surgical and medical procedures</title>
            <event_list>
              <event>
                <sub_title>Cholecystectomy</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Vascular disorders</title>
            <event_list>
              <event>
                <sub_title>Hypertension</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="73"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_vocab>CTCAE (4.0)</default_vocab>
        <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="20" subjects_at_risk="73"/>
                <counts group_id="E2" subjects_affected="16" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Nausea</sub_title>
                <counts group_id="E1" events="11" subjects_affected="11" subjects_at_risk="73"/>
                <counts group_id="E2" events="6" subjects_affected="6" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Constipation</sub_title>
                <counts group_id="E1" events="3" subjects_affected="3" subjects_at_risk="73"/>
                <counts group_id="E2" events="6" subjects_affected="6" subjects_at_risk="40"/>
              </event>
              <event>
                <sub_title>Diarrhea</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="73"/>
                <counts group_id="E2" events="3" subjects_affected="3" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" events="6" subjects_affected="6" subjects_at_risk="73"/>
                <counts group_id="E2" events="4" subjects_affected="4" subjects_at_risk="40"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT 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>Elias Joseph MD./Professor</name_or_title>
      <organization>The University of Texas MD Anderson Cancer Center</organization>
      <phone>713-792-4764</phone>
      <email>ejabbour@mdanderson.org</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/NCT01720225
org study id: 2012-0507
secondary id: NCI-2012-02215
nct id: NCT01720225
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
The goal of this clinical research study is to compare how two different drugs, decitabine
      and azacitidine, when given on a shorter than standard dosing schedule can help to control
      MDS. The safety of the drugs will also be studied.

      Decitabine is designed to damage the DNA (the genetic material) of cells, which may cause
      cancer cells to die.

      Azacitidine is designed to block certain proteins in cancer cells whose job is to stop the
      function of the tumor-fighting proteins. By blocking the "bad" proteins, the tumor-fighting
      genes may be able to work better. This could cause the cancer cells to die.
Study Groups:

      If you are found to be eligible to take part in this study, you will be randomly assigned (as
      in the flip of a coin) to 1 of 2 groups:

        -  If you are in Group 1, you will receive decitabine by vein over about 1 hour.

        -  If you are in Group 2, you will receive azacitidine either as an injection under your
           skin or through a vein. If by vein, the infusion will take about an hour.

      At first, there will be an equal chance of being assigned to either group. However, as the
      study goes on and more information becomes available, the chance of being assigned to the
      group that has shown the most effectiveness will increase. However, once you are already
      enrolled and assigned to a group, you will not be eligible to change groups.

      Study Drug Administration:

      Each cycle is 28 days.

      You will receive the study drug on Days 1-3 of every cycle and you will receive at least 2
      cycles of study drug.

      Study Visits:

      Every 7-14 days, blood (about 2 tablespoons) will be drawn for routine tests.

      At the end of Cycle 2, you will have a bone marrow biopsy and/or aspirate to check the status
      of the disease. This will then be repeated every 2-4 cycles until any point that the disease
      appears to have responded to the study drug, then as often as the study doctor thinks is
      necessary. To collect a bone marrow biopsy/aspirate, an area of the hip bone is numbed with
      anesthetic, and a small amount of bone and/or bone marrow is withdrawn through a large
      needle.

      After Cycle 3:

      If the study doctor decides it is acceptable, you may be allowed to receive treatment from
      your local cancer doctor. However, you have to return to Houston at least every 3 months for
      your study visits.

      The frequency of the visits will depend on what the doctor thinks is in your best interest.

      Length of Study:

      You may continue taking the study drug for as long as the doctor thinks it is in your best
      interest. You will no longer be able to take the study drug if the disease gets worse, if
      intolerable side effects occur, or if you are unable to follow study directions.

      Follow-Up Visits:

      One (1) time every 3 months after your last dose of study drug, you will return to the clinic
      for a bone marrow aspiration to check the status of the disease.

      Other Information:

      You may be given other drugs to help prevent side effects. The study staff will tell you
      about these drugs, how they will be given, and the possible risks.

      This is an investigational study. Decitabine and Azacitidine are both FDA approved and
      commercially available for use in patients with MDS.

      Up to 120 patients will take part in this study. All will be enrolled at MD Anderson.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Participants With a Response
time frame: 56 days
description: Overall Response = complete remission (CR) + partial remission (PR) + marrow CR (mCR) + hematologic improvement (HI). CR is normalization of peripheral blood and bone marrow with <5% bone marrow blasts, a peripheral blood granulocyte count > (1.0 x 10^9/L, and platelet count > 100 x 10^9/L). PR is same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if <15% at start of treatment. Marrow CR is blasts </= 5% and decreased by >/=50% from baseline. HI is platelets increase by 50% and to above 30 x 10^9/L untransfused (if lower than that pre-therapy; or hemoglobin increase by 2 g/dl; or transfusion independent; or splenomegaly reduction by > 50%; or monocytosis reduction by > 50% if pretreatment > 5 X1 0^9/L.
measure: Number of Participants Who Became Transfusion Independent
time frame: 8 weeks
description: Participants who were transfusion dependent at baseline prior to starting therapy on the Decitabine or Azacitidine arm will be analyzed for transfusion independence. Transfusion independence defined as being transfusion-free for ≥8 consecutive weeks between the first dose and study treatment discontinuation.
arm group label: Decitabine
arm group type: Experimental
description: Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.
arm group label: Azacitidine
arm group type: Experimental
description: Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
intervention type: Drug
intervention name: Decitabine
description: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.
arm group label: Decitabine
other name: DAC
other name: Dacogen
intervention type: Drug
intervention name: Azacitidine
description: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
arm group label: Azacitidine
other name: AZA
other name: 5-azacytidine
other name: 5-aza
other name: Vidaza
other name: 5-AZC
other name: AZA-CR
other name: Ladakamycin
other name: NSC-102816
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Elias Jabbour, MD
role: Principal Investigator
affiliation: M.D. Anderson Cancer Center
facility: 
country: United States
url: http://www.mdanderson.org
description: University of Texas MD Anderson Cancer Center Website
responsible party type: Sponsor
mesh term: Preleukemia
mesh term: Myelodysplastic Syndromes
mesh term: Azacitidine
mesh term: Decitabine
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 