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RecruitingNCT04794686Updated Jun 2, 2026

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

An observational study in Embryonal Tumor With Multilayered Rosettes, sponsored by Hackensack Meridian Health. Recruiting at 1 site in United States. Per ClinicalTrials.gov, last updated 2026-06-02.

Sponsored by Hackensack Meridian Health · Observational

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
100
Sex
All
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Study 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.

Read the 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.

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Conditions studied

  • Embryonal Tumor With Multilayered Rosettes

Keywords

  • Embryonal
  • Multilayer Rosettes
  • CNS tumor
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Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
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.

Eligibility criteria

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
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Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
100 participants (estimated)
Target follow-up
10 Years
Patient registry
Yes
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What researchers measure

Primary outcomes

  1. ETMR One Registry

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

    Time frame: 10 years

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

    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.

    Time frame: 10 years

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Study locations

1 of 1 sites recruiting
  • Hackensack University Medical Center
    Hackensack, New Jersey 07601, United States
    Recruiting
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References and documents

Publications

  • Picard D, Miller S, Hawkins CE, Bouffet E, Rogers HA, Chan TS, Kim SK, Ra YS, Fangusaro J, Korshunov A, Toledano H, Nakamura H, Hayden JT, Chan J, Lafay-Cousin L, Hu P, Fan X, Muraszko KM, Pomeroy SL, Lau CC, Ng HK, Jones C, Van Meter T, Clifford SC, Eberhart C, Gajjar A, Pfister SM, Grundy RG, Huang A. Markers of survival and metastatic potential in childhood CNS primitive neuro-ectodermal brain tumours: an integrative genomic analysis. Lancet Oncol. 2012 Aug;13(8):838-48. doi: 10.1016/S1470-2045(12)70257-7. Epub 2012 Jun 11. PubMed 22691720 ↗
  • Korshunov A, Sturm D, Ryzhova M, Hovestadt V, Gessi M, Jones DT, Remke M, Northcott P, Perry A, Picard D, Rosenblum M, Antonelli M, Aronica E, Schuller U, Hasselblatt M, Woehrer A, Zheludkova O, Kumirova E, Puget S, Taylor MD, Giangaspero F, Peter Collins V, von Deimling A, Lichter P, Huang A, Pietsch T, Pfister SM, Kool M. Embryonal tumor with abundant neuropil and true rosettes (ETANTR), ependymoblastoma, and medulloepithelioma share molecular similarity and comprise a single clinicopathological entity. Acta Neuropathol. 2014 Aug;128(2):279-89. doi: 10.1007/s00401-013-1228-0. Epub 2013 Dec 14. PubMed 24337497 ↗
  • Korshunov A, Ryzhova M, Jones DT, Northcott PA, van Sluis P, Volckmann R, Koster J, Versteeg R, Cowdrey C, Perry A, Picard D, Rosenblum M, Giangaspero F, Aronica E, Schuller U, Hasselblatt M, Collins VP, von Deimling A, Lichter P, Huang A, Pfister SM, Kool M. LIN28A immunoreactivity is a potent diagnostic marker of embryonal tumor with multilayered rosettes (ETMR). Acta Neuropathol. 2012 Dec;124(6):875-81. doi: 10.1007/s00401-012-1068-3. Epub 2012 Nov 16. PubMed 23161096 ↗
  • Zhu H, Shyh-Chang N, Segre AV, Shinoda G, Shah SP, Einhorn WS, Takeuchi A, Engreitz JM, Hagan JP, Kharas MG, Urbach A, Thornton JE, Triboulet R, Gregory RI; DIAGRAM Consortium; MAGIC Investigators; Altshuler D, Daley GQ. The Lin28/let-7 axis regulates glucose metabolism. Cell. 2011 Sep 30;147(1):81-94. doi: 10.1016/j.cell.2011.08.033. PubMed 21962509 ↗
  • Li M, Lee KF, Lu Y, Clarke I, Shih D, Eberhart C, Collins VP, Van Meter T, Picard D, Zhou L, Boutros PC, Modena P, Liang ML, Scherer SW, Bouffet E, Rutka JT, Pomeroy SL, Lau CC, Taylor MD, Gajjar A, Dirks PB, Hawkins CE, Huang A. Frequent amplification of a chr19q13.41 microRNA polycistron in aggressive primitive neuroectodermal brain tumors. Cancer Cell. 2009 Dec 8;16(6):533-46. doi: 10.1016/j.ccr.2009.10.025. PubMed 19962671 ↗

Individual participant data

Plan to share: No

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Registry details

Key details

Study ID
NCT04794686
Lead sponsor
Hackensack Meridian Health
Responsible party
Sponsor
First posted
Mar 12, 2021
Start date
Jul 12, 2020
Primary completion
Jul 12, 2030 (estimated)
Completion
Jul 12, 2030 (estimated)
Last update
Jun 2, 2026

Study contacts

Derek Hanson, MD
Contact
Derek.Hanson@HMHN.org
551-996-8437
Kellie Daniel, BA
Contact
Kellie.daniel@HMHN.org
551-996-3654
Derek Hanson, MD
principal investigator · Hackensack Meridian Health

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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