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

Sponsor
Hackensack Meridian Health (Other)
Overall Status
Recruiting
CT.gov ID
NCT04794686
Collaborator
(none)
100
1
120
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Study Details

Study Description

Brief Summary

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.

Condition or Disease Intervention/Treatment Phase

    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 Design

    Study Type:
    Observational [Patient Registry]
    Anticipated Enrollment :
    100 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    ETMR One: An International Registry and Research Platform for Children With Embryonal Tumor With Multilayer Rosettes
    Actual Study Start Date :
    Jul 12, 2020
    Anticipated Primary Completion Date :
    Jul 12, 2030
    Anticipated Study Completion Date :
    Jul 12, 2030

    Outcome Measures

    Primary Outcome Measures

    1. ETMR One Registry [10 years]

      To create and maintain a comprehensive registry containing the epidemiologic, clinical, and molecular data of patients with embryonal tumor with multilayer rosettes.

    2. Efficacy of consensus therapy on median event-free survival [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.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    All
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Hackensack University Medical Center Hackensack New Jersey United States 07601

    Sponsors and Collaborators

    • Hackensack Meridian Health

    Investigators

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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    Responsible Party:
    Hackensack Meridian Health
    ClinicalTrials.gov Identifier:
    NCT04794686
    Other Study ID Numbers:
    • Pro2020-0391
    First Posted:
    Mar 12, 2021
    Last Update Posted:
    Aug 15, 2022
    Last Verified:
    Aug 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Hackensack Meridian Health
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Aug 15, 2022