Epithelioid Sarcoma Natural History Study

NCT03837678 | Completed

• 1 other identifier: EZH-1001
• Study Type: observational
• Target: 74
• Locations: 1 country
• Sites: 5

Brief Summary

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).

Conditions

Epithelioid Sarcoma

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (5)

• 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

  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)

  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 Outcomes (1)

• Safety Outcome: Occurrence of adverse events

  Initiation of treatment with any systemic anti-cancer therapy for the treatment between January 1, 2000 and December 31, 2017 treatment modification or discontinuation

Eligibility Criteria

• Age: 10 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

Sponsors & Collaborators

• Epizyme, Inc.: lead
• Analysis Group, Inc.: collaborator

Study Sites (5)

University of Colorado Cancer Center

Denver, Colorado, 80045, United States

Location

Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

University of Michigan Comprehensive Cancer Center

Ann Arbor, Michigan, 48109, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77054, United States

Location

MeSH Terms

Conditions

Sarcoma

Condition Hierarchy (Ancestors)

Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms

Study Officials

• Ipsen Medical Director

  Ipsen

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 5, 2019
• First Posted: February 12, 2019
• Study Start: June 4, 2018
• Primary Completion: September 2, 2021
• Study Completion: September 2, 2021
• Last Updated: March 7, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will share
• 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.
• Access Criteria: Further details on Ipsen's sharing criteria, eligible studies and process for sharing are available here (https://vivli.org/members/ourmembers/).

Locations

US (5)