<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/NCT02233504</url>
  </required_header>
  <id_info>
    <org_study_id>UPCC 40413</org_study_id>
    <nct_id>NCT02233504</nct_id>
  </id_info>
  <brief_title>Pilot Study to Assess Hematologic Response in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndromes Undergoing Monotherapy With Exjade (Deferasirox)</brief_title>
  <official_title>Pilot Study to Assess Hematologic Response in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndromes Undergoing Monotherapy With Exjade (Deferasirox)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Abramson Cancer Center at Penn Medicine</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Abramson Cancer Center at Penn Medicine</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this trial is to examine the hematologic response rate of Exjade® in patients&#xD;
      with AML and high risk MDS and chronic iron overload from blood transfusions. Deferasirox has&#xD;
      been developed as an iron-chelating agent, and unlike deferoxamine, a previously developed&#xD;
      iron chelator, deferasirox has the advantage of oral administration.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">August 2014</start_date>
  <completion_date type="Actual">October 2, 2017</completion_date>
  <primary_completion_date type="Actual">October 2017</primary_completion_date>
  <phase>Early Phase 1</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
  </study_design_info>
  <primary_outcome>
    <measure>Number of Adverse Events</measure>
    <time_frame>2 years</time_frame>
  </primary_outcome>
  <enrollment type="Anticipated">25</enrollment>
  <condition>High Risk MDS or AML Patients</condition>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Exjade® (deferasirox, ICL670)</intervention_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Patients with high risk MDS or AML Exjade Pilot Assessing Hematologic Response in&#xD;
             AML/MDS UPCC# 40413&#xD;
&#xD;
          -  Newly diagnosed acute myeloid leukemia, not a candidate for intensive induction&#xD;
             chemotherapy in the judgement of the investigator or unwilling to undergo intensive&#xD;
             induction chemotherapy OR&#xD;
&#xD;
          -  AML that is refractory or relapsed after treatment with a non-intensive regimen, and&#xD;
             not a candidate for allogeneic transplant at this time OR&#xD;
&#xD;
          -  MDS, IPSS 1.5 or greater, intolerant or with disease progression/lack of response to&#xD;
             hypomethylating agents.&#xD;
&#xD;
          -  Age ≥ 18 years and able to provide independent informed consent&#xD;
&#xD;
          -  Serum Ferritin: For enrollment on the study: serum ferritin ≥ 500 ng/mL at screening.&#xD;
             Samples must be obtained in the absence of concomitant infection. If transfusion is&#xD;
             scheduled, draw serum ferritin PRIOR to transfusion.&#xD;
&#xD;
          -  ECOG performance status 0-2.&#xD;
&#xD;
          -  Sexually active women must use an effective method of contraception, or must have&#xD;
             undergone clinically documented total hysterectomy and/or oophorectomy, or tubal&#xD;
             ligation or be postmenopausal (defined as amenorrhea for at least 12 months)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Prior therapy with intensive chemotherapy for AML&#xD;
&#xD;
          -  Prior therapy with iron chelating agents within the last 6 months.&#xD;
&#xD;
          -  Serum creatinine levels greater than 1.5x above the upper limit of normal&#xD;
&#xD;
          -  AST or ALT levels greater than 5x the upper limit of normal&#xD;
&#xD;
          -  Current therapy for AML or MDS (a ≥ 4 week washout period for any agent used to treat&#xD;
             AML or MDS prior to first dose of study drug is required).&#xD;
&#xD;
          -  Current therapy with hydrea to control leukocytosis.&#xD;
&#xD;
          -  Clinical or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the&#xD;
             absence of HBsAb OR HCV Ab positive with HCV RNA positive and ALT above the normal&#xD;
             range) infection. In the absence of clinical suspicion for active infection,&#xD;
             laboratory testing to assess Hepatitis B or C status will not be required at&#xD;
             screening.&#xD;
&#xD;
          -  Clinical history of HIV positive test result (ELISA or Western blot). Laboratory&#xD;
             testing to assess HIV status will not be required at screening.&#xD;
&#xD;
          -  Clinical or self reported history of drug or alcohol abuse within the 12 months prior&#xD;
             to enrollment&#xD;
&#xD;
          -  ECOG Performance Status &gt; 2&#xD;
&#xD;
          -  Uncontrolled systemic hypertension&#xD;
&#xD;
          -  Severe cardiac insufficiency (NYHA III or IV), with uncontrolled and/or unstable&#xD;
             cardiac or coronary artery disease not controlled by standard medical therapy&#xD;
&#xD;
          -  Clinical diagnosis of or history of clinically relevant ocular toxicity related to&#xD;
             iron chelation&#xD;
&#xD;
          -  Systemic diseases (cardiovascular, renal, hepatic, etc.) which in the opinion of the&#xD;
             investigator would prevent study treatment&#xD;
&#xD;
          -  Pregnant or breast feeding women&#xD;
&#xD;
          -  Treatment with systemic investigational drug within the past 4 weeks or topical&#xD;
             investigational drug within the past 7 days or plan to receive other investigational&#xD;
             drugs while participating in the study&#xD;
&#xD;
          -  Other surgical or medical condition which might significantly alter the absorption,&#xD;
             distribution, metabolism or excretion of study drug&#xD;
&#xD;
          -  History of non-compliance to medical regimens or patients who are considered&#xD;
             potentially unreliable and/or not cooperative&#xD;
&#xD;
          -  Hematopoietic stem cell transplant for MDS or AML.&#xD;
&#xD;
          -  Active CNS leukemia Patients who are found to be ineligible after screening procedures&#xD;
             will have the reason for ineligibility documented on the screening log. No further&#xD;
             data will be collected in the CRF for these patients.&#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>James Mangan, MD, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Abramson Cancer Center at Penn Medicine</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Abramson Cancer Center of the University of Pennsylvania</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>April 2020</verification_date>
  <study_first_submitted>September 4, 2014</study_first_submitted>
  <study_first_submitted_qc>September 4, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 8, 2014</study_first_posted>
  <last_update_submitted>April 7, 2020</last_update_submitted>
  <last_update_submitted_qc>April 7, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">April 8, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <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>Deferasirox</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/NCT02233504
org study id: UPCC 40413
nct id: NCT02233504
lead sponsor: 
has dmc: Yes
The purpose of this trial is to examine the hematologic response rate of Exjade® in patients
      with AML and high risk MDS and chronic iron overload from blood transfusions. Deferasirox has
      been developed as an iron-chelating agent, and unlike deferoxamine, a previously developed
      iron chelator, deferasirox has the advantage of oral administration.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
measure: Number of Adverse Events
time frame: 2 years
intervention type: Drug
intervention name: Exjade® (deferasirox, ICL670)
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: James Mangan, MD, PhD
role: Principal Investigator
affiliation: Abramson Cancer Center at Penn Medicine
facility: 
country: United States
responsible party type: Sponsor
mesh term: Leukemia, Myeloid, Acute
mesh term: Preleukemia
mesh term: Myelodysplastic Syndromes
mesh term: Deferasirox