Embryonal TumorNCT05711992
Rare Embryonal Tumors of the Central Nervous System: International Registry
An observational study of Embryonal Tumor, run by Immune Oncology Research Institute at 11 sites in 9 countries.
Trial summary
Central nervous system (CNS) tumors are the most common solid malignancies among children. Although some types of CNS tumors like medulloblastomas and low-grade gliomas are widespread and well-studied, there is a huge number of rare diseases that need further research. Read more about this study
- Who is it for?
- Ages 1 day to 25 years
- Conditions the study is for
- Embryonal Tumor
- How many people
- 300 participants
- Where
- 11 sitesin 9 countries
- Study dates
- 1 Feb 2023to Feb 2033
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 day to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: All patients diagnosed with rare embryonal tumors
Who may be able to join
- Patients diagnosed with rare embryonal tumors of CNS since 01.01.2010:
- ETMR (including embryonal tumor with abundant neuropil and true rosettes (ETANTR), ependymoblastoma (EBL) and medulloepithelioma (MEPL) which were previously classified as CNS-PNETs)
- FOXR2-activated CNS neuroblastoma
- cribriform neuroepithelial tumor
- CNS tumor with BCOR internal tandem duplication
- all patients diagnosed with neuroblastoma and ganglioneuroblastoma with no molecular genetic tests available
Show all 9Show fewer
- Patients ≤ 25 years of age
- Signed informed consent form for prospective patients ≥ 18 years of age
- Signed parental permission and child assent forms for prospective patients < 18 years of age
Who cannot join
- CNS metastases of extracranial embryonal tumors
Trial locations and contacts
11 sites in 9 countries. Contact the study team or a site near you to ask about taking part.
11 sites in 9 countries
Mexico2 sites, 2 recruiting
- Recruiting
Hospital Civil de Guadalajara "Dr. Juan I. Menchaca"
Guadalajara, 44340
Regina Mallinalli Navarro Martin Del Campo, MD, reginamallinalli@hotmail.com
- Recruiting
Centro Médico ABC
Mexico City, 01120
Alejandra Jimena García Velázquez, MD, ale.garciavelazquez@gmail.com
Peru2 sites, 2 recruiting
- Recruiting
Hospital Nacional Alberto Sabogal Sologuren - EsSalud
Bellavista, 07011
Omar Reyes Cruzado, MD, omarreyescruzado@hotmail.com
Sofia Aguilar Moreno, MD
- Recruiting
Hospital Nacional Edgardo Rebagliati Martins
Lima, 15072
Ana Maria Glória Paredes Guerra, MD, gloriaparedes2000@yahoo.com
Armenia1 site, 1 recruiting
- Recruiting
Hematology Center named after prof. R. Yeolyan
Yerevan, 0014
Julieta Hoveyan, MD, +374 10 28 38 00, julia.hoveyan95@gmail.com
Ruzanna Papyan, MD, +374 10 28 38 00, ruzannapapyan92@gmail.com
Canada1 site, 1 recruiting
- Recruiting
Children's Hospital of Eastern Ontario (CHEO)
Ottawa, K1H 8L1
Consolato M Sergi, MD, PhD, MPH, csergi@cheo.on.ca
Hungary1 site, 1 recruiting
- Recruiting
Semmelweis University
Budapest, 1085
Miklós Garami, MD, MSc, PhD, miklos.garami@gmail.com
Edit Bruckner, MD, edit.brueckner@gmail.com
India1 site, 1 recruiting
- Recruiting
National Cancer Institute, All India Institutes of Medical Sciences
New Delhi, 110029
Supriya Mallick, MD, drsupriyamallick@gmail.com
Akash Kumar, MD
Iran1 site, 1 recruiting
- Recruiting
Oncology Department of Golestan hospital
Tehran, 16686 19551
Azim Mehrvar, MD, drazimmehrvar@yahoo.com
Azim Mehrvar, MD
Italy1 site, 1 recruiting
- Recruiting
IRCCS Istituto Ospedale Pediatrico Bambino Gesù
Rome, 00163
Angela Mastronuzzi, MD, PhD, angela.mastronuzzi@opbg.net
Giada Del Baldo, MD, giada.delbaldo@opbg.net
Taiwan1 site, 1 recruiting
- Recruiting
China Medical University Children's Hospital
Taichung, 40447
Su Min-Yu, MD, 035515@tool.caaumed.org.tw
Ching-Tien Peng, MD, penect@gmail.com
About this study
Central nervous system (CNS) tumors are the most common solid malignancies among children. Although some types of CNS tumors like medulloblastomas and low-grade gliomas are widespread and well-studied, there is a huge number of rare diseases that need further research. This international registry aims to establish a large multicenter database of pediatric and young adult patients with rare embryonal tumors of the central nervous system and describe the clinical presentations, diagnostics, treatment regimens, and outcomes. Embryonal tumors with multilayered rosettes (ETMR), FOXR2-activated CNS neuroblastoma, cribriform neuroepithelial tumor, and CNS tumor with BCOR internal tandem duplication are extremely rare embryonal tumors some of which were first described in the last edition of the World Health Organization (WHO) Classification of Tumors of the Central Nervous System. Objectives of the registry are 1) to evaluate prognostic factors, 2) to identify diagnostic and treatment gaps, 3) to investigate the characteristics and outcome of the disease with different treatment regimens, and 4) to generate data-based prospective diagnostic and treatment recommendations.
Read the detailed description
CNS tumors are the most common solid malignancies and the leading cause of children's cancer-related mortality. Embryonal tumors account for approximately 20-25% of all primary CNS tumors in children. Although medulloblastomas are the most commonly diagnosed malignant brain tumors, other embryonal tumors are relatively rare. Several studies of rare embryonal tumors have been published, but the number of included patients is generally small. Diagnosis of different subtypes of rare embryonal tumors can be extremely challenging. Because of limited data, there are no standard treatment recommendations for patients with rare embryonal tumors.
Embryonal tumors with multilayered rosettes (ETMR), FOXR2-activated CNS neuroblastoma, cribriform neuroepithelial tumor, CNS tumor with BCOR internal tandem duplication, and embryonal tumors not otherwise specified/not elsewhere classified (NOS/NEC) are extremely rare. Moreover, FOXR2-activated CNS neuroblastoma, cribriform neuroepithelial tumor, and CNS tumor with BCOR internal tandem duplication were first described in the fifth edition of the WHO Classification of Tumors of the Central Nervous System, published in 2021. Because of the rarity of these tumors, randomized controlled clinical trials are extremely complicated to conduct. Considering the lack of studies from low- and middle-income countries (LMICs) it is not excluded that cases of rare embryonal tumors are more common than have been described in the literature. Hence, evidence can be generated through registry studies.
This is a multicenter international retrospective and prospective registry to collect and analyze data from pediatric and young adult patients diagnosed with rare CNS embryonal tumors. Patients will be recruited directly by participating centers and national study groups. Participating centers will collect and verify the informed consent of all prospective patients enrolled at their centers.
Patients diagnosed with rare embryonal tumors of the CNS (ETMR, FOXR2-activated CNS neuroblastoma, cribriform neuroepithelial tumor, CNS tumor with BCOR internal tandem duplication, embryonal tumors NOS/NEC) since 01.01.2010 will be included. ETMR has been included in the WHO classification of CNS tumors since 2016 and encompasses three morphologically distinct embryonal tumors (Embryonal tumor with abundant neuropil and true rosettes (ETANTR), ependymoblastoma (EBL) and medulloepithelioma (MEPL)) that were previously classified as CNS primitive neuroectodermal tumors (CNS-PNETs). These histological subtypes should also be included in the study. Considering the lack of molecular genetic tests done among retrospective cases, the investigators will also include all patients diagnosed with neuroblastoma and ganglioneuroblastoma.
The following data will be collected through questionnaires:
- Patient characteristics
- Characteristics of rare CNS embryonal tumors
- Details of the diagnosis and treatment
- Complications and late effects of treatment
- Outcomes
- Follow-up information
Quality control and data management will be conducted by the Immune Oncology Research Institute.
- Conditions
- Embryonal Tumor
- Medical subject headings
- Neoplasms, Germ Cell and Embryonal
What is being measured
The primary outcome is the main question the study is designed to answer.
3-year overall survival
Measured: 3 years
5-year overall survival
Measured: 5 years
Secondary outcomes (3)
Complete remission rate
Measured: 5 years
3-year event-free survival
Measured: 3 years
5-year event-free survival
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Feb 2023
- Study start1 Feb 2023
- Primary completionFeb 2033 (estimated)
- Study completionFeb 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Embryonal TumorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Other
- Enrolment
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Immune Oncology Research Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Julieta Hoveyan, MD, Principal investigator, Immune Oncology Research Institute, Yerevan, ArmeniaRuzanna Papyan, MD, Principal investigator, Immune Oncology Research Institute, Yerevan, ArmeniaGevorg Tamamyan, MD, PhD, DSc, Study director, Immune Oncology Research Institute, Yerevan, Armenia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05711992
- Sponsor's protocol ID
- IMMONC0005
- Status last verified
- May 2025
- First submitted
- 25 Jan 2023
- Last updated
- 8 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 8 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Rare Embryonal Tumors of the Central Nervous System: International Registry
- ClinicalTrials.gov ID
- NCT05711992
- Acronym / sponsor ID
- IMMONC0005
- Conditions
- Embryonal Tumor
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Immune Oncology Research InstituteUniversity, hospital or other
- Study start
- 1 Feb 2023 (actual)
- Primary completion
- Feb 2033 (estimated)
- Study completion
- Feb 2033 (estimated)
- First posted
- 3 Feb 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Central nervous system (CNS) tumors are the most common solid malignancies among children. Although some types of CNS tumors like medulloblastomas and low-grade gliomas are widespread and well-studied, there is a huge number of rare diseases that need further research. This international registry aims to establish a large multicenter database of pediatric and young adult patients with rare embryonal tumors of the central nervous system and describe the clinical presentations, diagnostics, treatment regimens, and outcomes. Embryonal tumors with multilayered rosettes (ETMR), FOXR2-activated CNS neuroblastoma, cribriform neuroepithelial tumor, and CNS tumor with BCOR internal tandem duplication are extremely rare embryonal tumors some of which were first described in the last edition of the World Health Organization (WHO) Classification of Tumors of the Central Nervous System. Objectives of the registry are 1) to evaluate prognostic factors, 2) to identify diagnostic and treatment gaps, 3) to investigate the characteristics and outcome of the disease with different treatment regimens, and 4) to generate data-based prospective diagnostic and treatment recommendations.
Detailed description
CNS tumors are the most common solid malignancies and the leading cause of children's cancer-related mortality. Embryonal tumors account for approximately 20-25% of all primary CNS tumors in children. Although medulloblastomas are the most commonly diagnosed malignant brain tumors, other embryonal tumors are relatively rare. Several studies of rare embryonal tumors have been published, but the number of included patients is generally small. Diagnosis of different subtypes of rare embryonal tumors can be extremely challenging. Because of limited data, there are no standard treatment recommendations for patients with rare embryonal tumors.
Embryonal tumors with multilayered rosettes (ETMR), FOXR2-activated CNS neuroblastoma, cribriform neuroepithelial tumor, CNS tumor with BCOR internal tandem duplication, and embryonal tumors not otherwise specified/not elsewhere classified (NOS/NEC) are extremely rare. Moreover, FOXR2-activated CNS neuroblastoma, cribriform neuroepithelial tumor, and CNS tumor with BCOR internal tandem duplication were first described in the fifth edition of the WHO Classification of Tumors of the Central Nervous System, published in 2021. Because of the rarity of these tumors, randomized controlled clinical trials are extremely complicated to conduct. Considering the lack of studies from low- and middle-income countries (LMICs) it is not excluded that cases of rare embryonal tumors are more common than have been described in the literature. Hence, evidence can be generated through registry studies.
This is a multicenter international retrospective and prospective registry to collect and analyze data from pediatric and young adult patients diagnosed with rare CNS embryonal tumors. Patients will be recruited directly by participating centers and national study groups. Participating centers will collect and verify the informed consent of all prospective patients enrolled at their centers.
Patients diagnosed with rare embryonal tumors of the CNS (ETMR, FOXR2-activated CNS neuroblastoma, cribriform neuroepithelial tumor, CNS tumor with BCOR internal tandem duplication, embryonal tumors NOS/NEC) since 01.01.2010 will be included. ETMR has been included in the WHO classification of CNS tumors since 2016 and encompasses three morphologically distinct embryonal tumors (Embryonal tumor with abundant neuropil and true rosettes (ETANTR), ependymoblastoma (EBL) and medulloepithelioma (MEPL)) that were previously classified as CNS primitive neuroectodermal tumors (CNS-PNETs). These histological subtypes should also be included in the study. Considering the lack of molecular genetic tests done among retrospective cases, the investigators will also include all patients diagnosed with neuroblastoma and ganglioneuroblastoma.
The following data will be collected through questionnaires:
- Patient characteristics
- Characteristics of rare CNS embryonal tumors
- Details of the diagnosis and treatment
- Complications and late effects of treatment
- Outcomes
- Follow-up information
Quality control and data management will be conducted by the Immune Oncology Research Institute.
Study design
- Observational model
- Case only
- Time perspective
- Other
Who may qualify
- Ages
- 1 day to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- All patients diagnosed with rare embryonal tumors
Key inclusion criteria (9 listed)
- Patients diagnosed with rare embryonal tumors of CNS since 01.01.2010:
- Patients ≤ 25 years of age
- Signed informed consent form for prospective patients ≥ 18 years of age
- Signed parental permission and child assent forms for prospective patients < 18 years of age
Key exclusion criteria (1 listed)
- CNS metastases of extracranial embryonal tumors
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
3-year overall survival
Time frame: 3 years
5-year overall survival
Time frame: 5 years
3 secondary outcomes
Complete remission rate
Time frame: 5 years
3-year event-free survival
Time frame: 3 years
5-year event-free survival
Time frame: 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 May 2025.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Julieta Hoveyan, MD principal investigator
Immune Oncology Research Institute, Yerevan, Armenia
Ruzanna Papyan, MD principal investigator
Immune Oncology Research Institute, Yerevan, Armenia
Gevorg Tamamyan, MD, PhD, DSc study director
Immune Oncology Research Institute, Yerevan, Armenia
11 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Embryonal Tumor, run by Immune Oncology Research Institute at 11 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 8 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in Mexico, Peru, Armenia, Canada, Hungary and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Julieta Hoveyan, MD (+374 10 28 38 00, julia.hoveyan95@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
3-year overall survival (time frame: 3 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 May 2025.