ETMR One: An International Registry and Research Platform for Children With Embryonal Tumor With Multilayer Rosettes

March 12, 2024 updated by: Hackensack Meridian Health

This research is being done to find out more information about a brain tumor called Embryonal Tumor with Multilayer Rosettes (ETMR) by collecting medical information from children who have this disease.

The purpose of this research study is to create and maintain a research database for patients with ETMR. The database will include information about occurrence rates, patient information, tumor tissue information, and response to treatment. This will help advance our understanding of this rare disease.

In addition, this study will include obtaining survival data and evaluating therapeutic response to expert consensus therapy, and procuring patient tumor tissue.

Study Overview

Status

Recruiting

Detailed Description

All patients with ETMR are eligible for inclusion within the registry, regardless of their disease status or treatment plan. Epidemiologic, clinical, and molecular data from each patient will be obtained following enrollment. Patients will then be followed longitudinally to obtain clinical outcome data. In an effort to best achieve the registry's secondary objectives, it is preferable, but not required, for patients to be registered soon after their diagnosis, prior to the initiation of any tumor-directed therapy.

This registry protocol contains recommendations for a consensus therapy derived from the ETMR medical literature, the investigators' own experience, and clinical data from other infant brain tumor protocols. The recommended therapy can thus not be viewed as investigational, but rather as a consensus recommendation derived from available data. While not mandated, it is strongly encouraged that patients enrolled in the registry be treated according to the recommended consensus therapy, as this will facilitate the prospective evaluation of ETMR patients using a uniform treatment approach.

Study Type

Observational

Enrollment (Estimated)

100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

    • New Jersey
      • Hackensack, New Jersey, United States, 07601
        • Recruiting
        • Hackensack University Medical Center
        • Contact:
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Sampling Method

Non-Probability Sample

Study Population

This registry will encompass a population of patients of any age with histologically confirmed primary intracranial CNS Embryonal Tumor with Multilayer Rosettes OR tumor tissue that possesses C19MC amplification. Patients may be enrolled at any point after diagnosis. In an effort to best achieve the registry's secondary objectives, it is preferable, but not required, for patients to be registered soon after their diagnosis, prior to the initiation of any tumor-directed therapy. Deceased patients may be enrolled to aid in the collection of historical data.

Description

Inclusion Criteria:

  1. Patients of any age
  2. Patients must have either a histologically confirmed primary intracranial CNS Embryonal Tumor with Multilayer Rosettes (as agreed upon by central review or local pathologist OR
  3. Patients must have tumor tissue that possesses C19MC amplification . Central Review is not required (but is strongly recommended) if the patient's tumor does not demonstrate C19MC amplification.
  4. Patients may be enrolled at point following diagnosis

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
ETMR One Registry
Time Frame: 10 years
To create and maintain a comprehensive registry containing the epidemiologic, clinical, and molecular data of patients with embryonal tumor with multilayer rosettes.
10 years
Efficacy of consensus therapy on median event-free survival
Time Frame: 10 years
To investigate the efficacy of the consensus regimen of chemotherapy, high-dose chemotherapy with autologous stem cell rescue, and radiotherapy (as indicated) following maximal feasible surgical resection for patients with newly-diagnosed ETMR as measured by median event-free and overall survival in comparison to historical controls.
10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Principal Investigator: Derek Hanson, MD, Hackensack Meridian Health

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 12, 2020

Primary Completion (Estimated)

July 12, 2030

Study Completion (Estimated)

July 12, 2030

Study Registration Dates

First Submitted

March 9, 2021

First Submitted That Met QC Criteria

March 9, 2021

First Posted (Actual)

March 12, 2021

Study Record Updates

Last Update Posted (Actual)

March 13, 2024

Last Update Submitted That Met QC Criteria

March 12, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Other Study ID Numbers

  • Pro2020-0391

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Embryonal Tumor With Multilayered Rosettes

Subscribe