<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/NCT03837678</url>
  </required_header>
  <id_info>
    <org_study_id>EZH-1001</org_study_id>
    <nct_id>NCT03837678</nct_id>
  </id_info>
  <brief_title>Epithelioid Sarcoma Natural History Study</brief_title>
  <official_title>Epithelioid Sarcoma Natural History Study</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Epizyme, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Analysis Group, Inc.</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Epizyme, Inc.</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This study is a multi-center, non-interventional retrospective medical records review. The&#xD;
      study will involve identification of medical records of patients with confirmed locally&#xD;
      advanced unresectable or metastatic ES, who initiated systemic therapy between January 1,&#xD;
      2000 and December 31, 2017.&#xD;
&#xD;
      Data for the chart review will be extracted retrospectively from eligible subjects' charts&#xD;
      (electronic or paper). Information on prior surgical treatment and neoadjuvant/adjuvant&#xD;
      therapies for ES will also be collected for ineligible subjects with a locally advanced or&#xD;
      metastatic ES diagnosis who did not initiated systemic therapy. Data collected will be&#xD;
      anonymized by the investigators and will not be traceable back to individual subjects by the&#xD;
      sponsor (i.e., no protected health information [PHI] will be collected).&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The sampling period will span from January 1, 2000 to December 31, 2017. As the chart&#xD;
      abstraction dates may be different across institutions due to contracting and other&#xD;
      logistical issues, to avoid seeing biased outcomes that may be related to this type of&#xD;
      heterogeneity, a consistent study end date will be imposed for all patients when conducting&#xD;
      the analysis. Information before this study end date will be analyzed and used to conduct the&#xD;
      analysis.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">June 4, 2018</start_date>
  <completion_date type="Actual">September 2, 2021</completion_date>
  <primary_completion_date type="Actual">September 2, 2021</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Other</observational_model>
    <time_perspective>Retrospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Real-word overall response rate (rwORR)</measure>
    <time_frame>Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to treatment failure</time_frame>
    <description>Proportion of patients who have a documented radiological scan showing clinician-assessed complete response or less-than-complete response, of any duration, defined for each regimen and by line</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Real-world duration of response (rwDOR)</measure>
    <time_frame>Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to treatment failure</time_frame>
    <description>Duration of time between the first radiological scan showing documented tumor response (any time of response excluding SD) to first noted disease progression, or treatment discontinuation if no progression occurred. This is defined for each line of therapy and by regimen.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Time from treatment initiation to treatment discontinuation</measure>
    <time_frame>Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to treatment failure</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time from treatment initiation to treatment failure</measure>
    <time_frame>Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to treatment failure</time_frame>
    <description>Treatment failure is defined as discontinuation of treatment for any reason, including disease progression, treatment toxicity, and death</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Real-world disease control rate (rwDCR)</measure>
    <time_frame>Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to treatment failure</time_frame>
    <description>Proportion of patients who have documented radiological scans showing clinician-assessed complete response or less-than-complete response, or stable disease lasting at least 32 weeks</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Overall survival (OS)</measure>
    <time_frame>Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to death</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Real-world time to tumor progression (rwTTP)</measure>
    <time_frame>Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to tumor progression</time_frame>
  </secondary_outcome>
  <other_outcome>
    <measure>Safety Outcome: Occurrence of adverse events</measure>
    <time_frame>Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 treatment modification or discontinuation</time_frame>
    <description>Occurrence of adverse events resulting in treatment modification or discontinuation, or patient hospitalization, while on systemic therapy</description>
  </other_outcome>
  <enrollment type="Actual">74</enrollment>
  <condition>Epithelioid Sarcoma</condition>
  <eligibility>
    <study_pop>
      <textblock>
        The study population will consist of Epithelioid Sarcoma (ES) patients (approximately 70 to&#xD;
        100) from approximately five (5) academic centers (i.e., study sites) in the US. The&#xD;
        sampling period will span from January 1, 2000 to December 31, 2017.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Diagnosed with histologically confirmed, locally advanced unresectable or metastatic&#xD;
             Epithelioid Sarcoma (ES) requiring systemic therapy during between January 1, 2000 and&#xD;
             December 31, 2017. The date of the confirmed locally advanced unresectable or&#xD;
             metastatic ES diagnosis is designated as the index date.&#xD;
&#xD;
          -  Patients may have a date of ES diagnosis at an earlier stage prior to 2000 and still&#xD;
             be eligible for the study&#xD;
&#xD;
          -  Initiation of treatment with any systemic anti-cancer therapy for the treatment of&#xD;
             their locally advanced unresectable or metastatic ES between January 1, 2000 and&#xD;
             December 31, 2017&#xD;
&#xD;
          -  At least 10 years of age at the index date&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>10 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>University of Colorado Cancer Center</name>
      <address>
        <city>Denver</city>
        <state>Colorado</state>
        <zip>80045</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Dana Farber Cancer Institute</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02215</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Michigan Comprehensive Cancer Center</name>
      <address>
        <city>Ann Arbor</city>
        <state>Michigan</state>
        <zip>48109</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Memorial Sloan Kettering Cancer Center</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10065</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>MD Anderson Cancer Center</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77054</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>December 2021</verification_date>
  <study_first_submitted>February 5, 2019</study_first_submitted>
  <study_first_submitted_qc>February 8, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">February 12, 2019</study_first_posted>
  <last_update_submitted>December 16, 2021</last_update_submitted>
  <last_update_submitted_qc>December 16, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">December 17, 2021</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>Sarcoma</mesh_term>
  </condition_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/NCT03837678
org study id: EZH-1001
nct id: NCT03837678
lead sponsor: 
collaborator: 
has dmc: No
This study is a multi-center, non-interventional retrospective medical records review. The
      study will involve identification of medical records of patients with confirmed locally
      advanced unresectable or metastatic ES, who initiated systemic therapy between January 1,
      2000 and December 31, 2017.

      Data for the chart review will be extracted retrospectively from eligible subjects' charts
      (electronic or paper). Information on prior surgical treatment and neoadjuvant/adjuvant
      therapies for ES will also be collected for ineligible subjects with a locally advanced or
      metastatic ES diagnosis who did not initiated systemic therapy. Data collected will be
      anonymized by the investigators and will not be traceable back to individual subjects by the
      sponsor (i.e., no protected health information [PHI] will be collected).
The sampling period will span from January 1, 2000 to December 31, 2017. As the chart
      abstraction dates may be different across institutions due to contracting and other
      logistical issues, to avoid seeing biased outcomes that may be related to this type of
      heterogeneity, a consistent study end date will be imposed for all patients when conducting
      the analysis. Information before this study end date will be analyzed and used to conduct the
      analysis.
observational model: Other
time perspective: Retrospective
measure: Real-word overall response rate (rwORR)
time frame: Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to treatment failure
description: Proportion of patients who have a documented radiological scan showing clinician-assessed complete response or less-than-complete response, of any duration, defined for each regimen and by line
measure: Real-world duration of response (rwDOR)
time frame: Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to treatment failure
description: Duration of time between the first radiological scan showing documented tumor response (any time of response excluding SD) to first noted disease progression, or treatment discontinuation if no progression occurred. This is defined for each line of therapy and by regimen.
measure: Time from treatment initiation to treatment discontinuation
time frame: Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to treatment failure
measure: Time from treatment initiation to treatment failure
time frame: Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to treatment failure
description: Treatment failure is defined as discontinuation of treatment for any reason, including disease progression, treatment toxicity, and death
measure: Real-world disease control rate (rwDCR)
time frame: Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to treatment failure
description: Proportion of patients who have documented radiological scans showing clinician-assessed complete response or less-than-complete response, or stable disease lasting at least 32 weeks
measure: Overall survival (OS)
time frame: Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to death
measure: Real-world time to tumor progression (rwTTP)
time frame: Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to tumor progression
measure: Safety Outcome: Occurrence of adverse events
time frame: Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 treatment modification or discontinuation
description: Occurrence of adverse events resulting in treatment modification or discontinuation, or patient hospitalization, while on systemic therapy
study pop: 
sampling method: Probability Sample
criteria: 
gender: All
minimum age: 10 Years
maximum age: N/A
healthy volunteers: No
facility: 
facility: 
facility: 
facility: 
facility: 
country: United States
responsible party type: Sponsor
mesh term: Sarcoma