<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/NCT02204085</url>
  </required_header>
  <id_info>
    <org_study_id>14-222</org_study_id>
    <nct_id>NCT02204085</nct_id>
  </id_info>
  <brief_title>A Phase I/II Trial of the MUC1 Inhibitor, GO-203-2C in Patients With Relapsed or Refractory Acute Myeloid Leukemia</brief_title>
  <official_title>A Phase I/II Trial of the MUC1 Inhibitor, GO-203-2C in Patients With Relapsed or Refractory Acute Myeloid Leukemia</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Beth Israel Deaconess Medical Center</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Dana-Farber Cancer Institute</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Beth Israel Deaconess Medical Center</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This research study is studying a targeted therapy known as GO-203-2C as a possible treatment&#xD;
      for with acute myeloid leukemia (AML) both alone and in combination with decitabine.&#xD;
      GO-203-2c targets cancer cells, while leaving healthy cells unaffected.This is a Phase I/II&#xD;
      clinical trial. A Phase I clinical trial tests the safety of an investigational intervention&#xD;
      and also tries to define the appropriate dose of the investigational intervention to use for&#xD;
      further studies.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      -  Phase I&#xD;
&#xD;
             -  The maximum tolerated dose (MTD) will be determined in the phase I section of the&#xD;
                trial.&#xD;
&#xD;
             -  Patients who fulfill eligibility criteria will be entered into the trial to&#xD;
                GO-203-2c.&#xD;
&#xD;
             -  After the screening procedures confirm participation in the research study. The&#xD;
                investigators are looking for the highest dose of the combination of study drugs&#xD;
                that can be administered safely without severe or unmanageable side effects in&#xD;
                participants that have acute myeloid leukemia (AML) not everyone who participates&#xD;
                in this research study will receive the same dose of the study drug. The dose given&#xD;
                will depend on the number of participants who have been enrolled in the study prior&#xD;
                and how well the dose was tolerated.&#xD;
&#xD;
             -  A subsequent dose escalation will evaluate the combination of GO-203-2c and&#xD;
                decitabine.&#xD;
&#xD;
        -  Phase II&#xD;
&#xD;
             -  The primary goal is to determine if the combination of the two drugs results in&#xD;
                clinical response&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date>September 2014</start_date>
  <completion_date type="Anticipated">December 2023</completion_date>
  <primary_completion_date type="Anticipated">July 2023</primary_completion_date>
  <phase>Phase 1/Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Maximum Tolerated Dose of GO-203-2c</measure>
    <time_frame>28 days</time_frame>
    <description>Phase I</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Maximum Tolerated Dose of GO-203-2c in combination with decitabine</measure>
    <time_frame>28 days</time_frame>
    <description>Phase 1</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Number of Participants with Adverse Events as a Measure of Safety and Tolerability</measure>
    <time_frame>2 years</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>To investigate whether GO-203-2c alone and in combination with decitabine is effective in targeting MUC1-C overexpressing AML progenitor cells in the lab</measure>
    <time_frame>2 Years</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>To assess whether in vitro response to GO-203-2c alone and in combination with decitabine is associated with clinical response</measure>
    <time_frame>2 Years</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>To determine if therapy with GO-203-2c alone and in combination with decitabine results in decreased engraftment potential of AML progenitor cells in an NSG mouse model</measure>
    <time_frame>2 Years</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>To determine if therapy GO-203-2c in combination with decitabine results in at least 20% of patients achieving a clinical response</measure>
    <time_frame>2 years</time_frame>
    <description>To determine if therapy GO-203-2c in combination with decitabine results in at least 20% of patients achieving a clinical response (blast response, minor response, partial response, or complete response).</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">33</enrollment>
  <condition>Acute Myeloid Leukemia, in Relapse</condition>
  <condition>Recurrent Adult Acute Myeloid Leukemia</condition>
  <arm_group>
    <arm_group_label>GO-203-2c</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Dose escalation will occur using a standard 3+3 dose escalation approach, beginning in dose level I with dose cohorts and rules for escalation and de-escalation.&#xD;
GO-203-2c given daily on predetermined schedule of a 28-day treatment cycle</description>
  </arm_group>
  <arm_group>
    <arm_group_label>GO-203-2c + Decitabine</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Dose escalation will occur for GO-203-2c using a standard 3+3 dose escalation approach, beginning in dose level I with dose cohorts and rules for escalation and de-escalation.&#xD;
GO-203-2c given daily on predetermined schedule of a 28-day treatment cycle. Decitabine will be administered at a dose of 20mg/m2 on days 8-12 of a 28-day treatment cycle.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>GO-203-2c</intervention_name>
    <arm_group_label>GO-203-2c</arm_group_label>
    <arm_group_label>GO-203-2c + Decitabine</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>GO-203-2c + Decitabine</intervention_name>
    <arm_group_label>GO-203-2c + Decitabine</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  To be considered eligible for enrollment into this study, all of the following&#xD;
             inclusion criteria must be met during the screening period:&#xD;
&#xD;
          -  Documented AML by peripheral blood and bone marrow analyses meeting WHO criteria,&#xD;
             excluding patients with acute promyelocytic leukemia (APL)&#xD;
&#xD;
          -  Patients with AML refractory to primary induction chemotherapy, relapsed disease, or&#xD;
             age ≥ 60 and not appropriate for standard cytotoxic therapy due to age, performance&#xD;
             status, and/or adverse risk factors according to the treating physician&#xD;
&#xD;
          -  Age ≥ 18 years&#xD;
&#xD;
          -  Karnofsky performance status ≥ 50% or ECOG performance status 0-2&#xD;
&#xD;
          -  Life expectancy ≥ 6 weeks&#xD;
&#xD;
          -  Able to understand the investigational nature of this study and to provide written&#xD;
             consent to participate in it&#xD;
&#xD;
          -  Signed written IRB-approved Informed Consent document&#xD;
&#xD;
          -  Adequate hepatic and renal function:&#xD;
&#xD;
               -  serum bilirubin ≤ 1.5 X institutional ULN OR serum direct bilirubin ≤ 2 X&#xD;
                  institutional ULN&#xD;
&#xD;
               -  serum ALT and AST ≤ 2.5 X institutional ULN&#xD;
&#xD;
               -  serum alkaline phosphatase &lt; 5 X institutional ULN&#xD;
&#xD;
               -  serum creatinine ≤ 2.0 mg/dL&#xD;
&#xD;
               -  corrected calcium level ≥ institutional LLN&#xD;
&#xD;
          -  Negative pregnancy test in women of child-bearing potential&#xD;
&#xD;
          -  Women and men of child-producing potential must agree to use effective contraceptive&#xD;
             methods during the study period (including post-treatment observation period)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  A patient will be considered not eligible for enrollment into this study if any of the&#xD;
             following criteria are met during the screening period:&#xD;
&#xD;
          -  Evidence of leukemic meningitis or other CNS involvement by leukemia&#xD;
&#xD;
          -  Uncontrolled or poorly controlled hypertension (systolic BP ≥ 160 mmHg or diastolic BP&#xD;
             ≥ 100 mmHg) Note: an isolated reading that is not sustained will be permitted.&#xD;
&#xD;
          -  Evidence of NYHA Class III or IV cardiac disease, or presence of unstable&#xD;
             life-threatening arrhythmia, or history of myocardial infarction during the past 6&#xD;
             months&#xD;
&#xD;
          -  Active bacterial, fungal, or viral infection requiring systemic treatment&#xD;
&#xD;
          -  Known infection with HIV&#xD;
&#xD;
          -  History or major surgery within 4 weeks before the first dose of study treatment, or&#xD;
             not recovered from prior surgery&#xD;
&#xD;
          -  Exposure to any other investigational agent at any time within 4 weeks before the&#xD;
             first dose of study treatment&#xD;
&#xD;
          -  Exposure to any other anti-leukemic therapy (except hydroxyurea, see Section 5.5.1)&#xD;
             within 2 weeks before the first dose of study treatment&#xD;
&#xD;
          -  Pregnant or lactating female&#xD;
&#xD;
          -  Unwilling or unable to comply with the requirements of the study protocol&#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>David Avigan, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Beth Israel Deaconess Medical Center</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Dana-Farber Cancer Institute</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Beth Israel Deaconess Medical Center</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02215</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>June 2023</verification_date>
  <study_first_submitted>July 9, 2014</study_first_submitted>
  <study_first_submitted_qc>July 29, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">July 30, 2014</study_first_posted>
  <last_update_submitted>June 16, 2023</last_update_submitted>
  <last_update_submitted_qc>June 16, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">June 22, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Beth Israel Deaconess Medical Center</investigator_affiliation>
    <investigator_full_name>David Avigan</investigator_full_name>
    <investigator_title>Principal Investigator</investigator_title>
  </responsible_party>
  <keyword>Refractory acute myeloid leukemia</keyword>
  <keyword>Relapsed acute myeloid leukemia</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>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Decitabine</mesh_term>
  </intervention_browse>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT02204085
org study id: 14-222
nct id: NCT02204085
lead sponsor: 
collaborator: 
has dmc: Yes
This research study is studying a targeted therapy known as GO-203-2C as a possible treatment
      for with acute myeloid leukemia (AML) both alone and in combination with decitabine.
      GO-203-2c targets cancer cells, while leaving healthy cells unaffected.This is a Phase I/II
      clinical trial. A Phase I clinical trial tests the safety of an investigational intervention
      and also tries to define the appropriate dose of the investigational intervention to use for
      further studies.
-  Phase I

             -  The maximum tolerated dose (MTD) will be determined in the phase I section of the
                trial.

             -  Patients who fulfill eligibility criteria will be entered into the trial to
                GO-203-2c.

             -  After the screening procedures confirm participation in the research study. The
                investigators are looking for the highest dose of the combination of study drugs
                that can be administered safely without severe or unmanageable side effects in
                participants that have acute myeloid leukemia (AML) not everyone who participates
                in this research study will receive the same dose of the study drug. The dose given
                will depend on the number of participants who have been enrolled in the study prior
                and how well the dose was tolerated.

             -  A subsequent dose escalation will evaluate the combination of GO-203-2c and
                decitabine.

        -  Phase II

             -  The primary goal is to determine if the combination of the two drugs results in
                clinical response
allocation: Non-Randomized
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Maximum Tolerated Dose of GO-203-2c
time frame: 28 days
description: Phase I
measure: Maximum Tolerated Dose of GO-203-2c in combination with decitabine
time frame: 28 days
description: Phase 1
measure: Number of Participants with Adverse Events as a Measure of Safety and Tolerability
time frame: 2 years
measure: To investigate whether GO-203-2c alone and in combination with decitabine is effective in targeting MUC1-C overexpressing AML progenitor cells in the lab
time frame: 2 Years
measure: To assess whether in vitro response to GO-203-2c alone and in combination with decitabine is associated with clinical response
time frame: 2 Years
measure: To determine if therapy with GO-203-2c alone and in combination with decitabine results in decreased engraftment potential of AML progenitor cells in an NSG mouse model
time frame: 2 Years
measure: To determine if therapy GO-203-2c in combination with decitabine results in at least 20% of patients achieving a clinical response
time frame: 2 years
description: To determine if therapy GO-203-2c in combination with decitabine results in at least 20% of patients achieving a clinical response (blast response, minor response, partial response, or complete response).
arm group label: GO-203-2c
arm group type: Experimental
description: Dose escalation will occur using a standard 3+3 dose escalation approach, beginning in dose level I with dose cohorts and rules for escalation and de-escalation.
GO-203-2c given daily on predetermined schedule of a 28-day treatment cycle
arm group label: GO-203-2c + Decitabine
arm group type: Experimental
description: Dose escalation will occur for GO-203-2c using a standard 3+3 dose escalation approach, beginning in dose level I with dose cohorts and rules for escalation and de-escalation.
GO-203-2c given daily on predetermined schedule of a 28-day treatment cycle. Decitabine will be administered at a dose of 20mg/m2 on days 8-12 of a 28-day treatment cycle.
intervention type: Drug
intervention name: GO-203-2c
arm group label: GO-203-2c
arm group label: GO-203-2c + Decitabine
intervention type: Drug
intervention name: GO-203-2c + Decitabine
arm group label: GO-203-2c + Decitabine
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: David Avigan, MD
role: Principal Investigator
affiliation: Beth Israel Deaconess Medical Center
facility: 
facility: 
country: United States
responsible party type: Principal Investigator
investigator affiliation: Beth Israel Deaconess Medical Center
investigator full name: David Avigan
investigator title: Principal Investigator
mesh term: Leukemia
mesh term: Leukemia, Myeloid
mesh term: Leukemia, Myeloid, Acute
mesh term: Decitabine