Epithelioid Sarcoma Natural History Study

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).

Study Overview

• Status: Completed
• Conditions: Epithelioid Sarcoma

Detailed Description

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.

• Study Type: Observational
• Enrollment (Actual): 74

Contacts and Locations

Study Locations

• United States
  • Colorado
    • Denver, Colorado, United States, 80045
      • University of Colorado Cancer Center
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Dana Farber Cancer Institute
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan Comprehensive Cancer Center
  • New York
    • New York, New York, United States, 10065
      • Memorial Sloan Kettering Cancer Center
  • Texas
    • Houston, Texas, United States, 77054
      • MD Anderson Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 10 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

The study population will consist of Epithelioid Sarcoma (ES) patients (approximately 70 to 100) from approximately five (5) academic centers (i.e., study sites) in the US. The sampling period will span from January 1, 2000 to December 31, 2017.

Description

Inclusion Criteria:

• Diagnosed with histologically confirmed, locally advanced unresectable or metastatic Epithelioid Sarcoma (ES) requiring systemic therapy during between January 1, 2000 and December 31, 2017. The date of the confirmed locally advanced unresectable or metastatic ES diagnosis is designated as the index date.
• Patients may have a date of ES diagnosis at an earlier stage prior to 2000 and still be eligible for the study
• Initiation of treatment with any systemic anti-cancer therapy for the treatment of their locally advanced unresectable or metastatic ES between January 1, 2000 and December 31, 2017
• At least 10 years of age at the index date

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Real-word overall response rate (rwORR)	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	Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to treatment failure
Real-world duration of response (rwDOR)	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.	Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to treatment failure

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time from treatment initiation to treatment discontinuation		Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to treatment failure
Time from treatment initiation to treatment failure	Treatment failure is defined as discontinuation of treatment for any reason, including disease progression, treatment toxicity, and death	Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to treatment failure
Real-world disease control rate (rwDCR)	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	Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to treatment failure
Overall survival (OS)		Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to death
Real-world time to tumor progression (rwTTP)		Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 to tumor progression

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety Outcome: Occurrence of adverse events	Occurrence of adverse events resulting in treatment modification or discontinuation, or patient hospitalization, while on systemic therapy	Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 treatment modification or discontinuation

Collaborators and Investigators

• Sponsor: Epizyme, Inc.
• Collaborators: Analysis Group, Inc.
• Investigators: Study Director: Ipsen Medical Director, Ipsen

Study record dates

Study Major Dates

• Study Start (Actual): June 4, 2018
• Primary Completion (Actual): September 2, 2021
• Study Completion (Actual): September 2, 2021

Study Registration Dates

• First Submitted: February 5, 2019
• First Submitted That Met QC Criteria: February 8, 2019
• First Posted (Actual): February 12, 2019

Study Record Updates

• Last Update Posted (Actual): March 7, 2024
• Last Update Submitted That Met QC Criteria: March 6, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Sarcoma
• Other Study ID Numbers: EZH-1001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications.

Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants. Any requests should be submitted to www.vivli.org for assessment by an independent scientific review board.

• IPD Sharing Time Frame: Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and EU or after the primary manuscript describing the results has been accepted for publication, whichever is later.
• IPD Sharing Access Criteria: Further details on Ipsen's sharing criteria, eligible studies and process for sharing are available here (https://vivli.org/members/ourmembers/).