Trial Title:
Rare Embryonal Tumors of the Central Nervous System: International Registry
NCT ID:
NCT05711992
Condition:
Embryonal Tumor
Conditions: Official terms:
Neoplasms, Germ Cell and Embryonal
Central Nervous System Neoplasms
Study type:
Observational
Overall status:
Recruiting
Study design:
Time perspective:
Other
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. 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:
1. Patient characteristics
2. Characteristics of rare CNS embryonal tumors
3. Details of the diagnosis and treatment
4. Complications and late effects of treatment
5. Outcomes
6. Follow-up information
Quality control and data management will be conducted by the Immune Oncology Research
Institute.
Criteria for eligibility:
Study pop:
All patients diagnosed with rare embryonal tumors
Sampling method:
Non-Probability Sample
Criteria:
Inclusion Criteria:
- 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
- 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
Exclusion Criteria:
• CNS metastases of extracranial embryonal tumors
Gender:
All
Minimum age:
1 Day
Maximum age:
25 Years
Healthy volunteers:
No
Locations:
Facility:
Name:
Hematology Center named after prof. R. Yeolyan
Address:
City:
Yerevan
Zip:
0014
Country:
Armenia
Status:
Recruiting
Contact:
Last name:
Julieta Hoveyan, MD
Phone:
+374 10 28 38 00
Email:
julia.hoveyan95@gmail.com
Contact backup:
Last name:
Ruzanna Papyan, MD
Phone:
+374 10 28 38 00
Email:
ruzannapapyan92@gmail.com
Facility:
Name:
Children's Hospital of Eastern Ontario (CHEO)
Address:
City:
Ottawa
Zip:
K1H 8L1
Country:
Canada
Status:
Recruiting
Contact:
Last name:
Consolato M Sergi, MD, PhD, MPH
Email:
csergi@cheo.on.ca
Facility:
Name:
Semmelweis University
Address:
City:
Budapest
Zip:
1085
Country:
Hungary
Status:
Recruiting
Contact:
Last name:
Miklós Garami, MD, MSc, PhD
Email:
miklos.garami@gmail.com
Contact backup:
Last name:
Edit Bruckner, MD
Email:
edit.brueckner@gmail.com
Facility:
Name:
National Cancer Institute, All India Institutes of Medical Sciences
Address:
City:
New Delhi
Zip:
110029
Country:
India
Status:
Recruiting
Contact:
Last name:
Supriya Mallick, MD
Email:
drsupriyamallick@gmail.com
Contact backup:
Last name:
Akash Kumar, MD
Facility:
Name:
Oncology Department of Golestan hospital
Address:
City:
Tehran
Zip:
16686 19551
Country:
Iran, Islamic Republic of
Status:
Recruiting
Contact:
Last name:
Azim Mehrvar, MD
Email:
drazimmehrvar@yahoo.com
Investigator:
Last name:
Azim Mehrvar, MD
Email:
Principal Investigator
Investigator:
Last name:
Maryam Tashvighi, MD
Email:
Sub-Investigator
Investigator:
Last name:
Narjes Mehrvar, PhD
Email:
Sub-Investigator
Facility:
Name:
IRCCS Istituto Ospedale Pediatrico Bambino Gesù
Address:
City:
Rome
Zip:
00163
Country:
Italy
Status:
Recruiting
Contact:
Last name:
Angela Mastronuzzi, MD, PhD
Email:
angela.mastronuzzi@opbg.net
Contact backup:
Last name:
Giada Del Baldo, MD
Email:
giada.delbaldo@opbg.net
Facility:
Name:
Hospital Civil de Guadalajara "Dr. Juan I. Menchaca"
Address:
City:
Guadalajara
Zip:
44340
Country:
Mexico
Status:
Recruiting
Contact:
Last name:
Regina Mallinalli Navarro Martin Del Campo, MD
Email:
reginamallinalli@hotmail.com
Facility:
Name:
Centro Médico ABC
Address:
City:
Mexico City
Zip:
01120
Country:
Mexico
Status:
Recruiting
Contact:
Last name:
Alejandra Jimena García Velázquez, MD
Email:
ale.garciavelazquez@gmail.com
Facility:
Name:
Hospital Nacional Alberto Sabogal Sologuren - EsSalud
Address:
City:
Bellavista
Zip:
07011
Country:
Peru
Status:
Recruiting
Contact:
Last name:
Omar Reyes Cruzado, MD
Email:
omarreyescruzado@hotmail.com
Contact backup:
Last name:
Sofia Aguilar Moreno, MD
Facility:
Name:
Hospital Nacional Edgardo Rebagliati Martins
Address:
City:
Lima
Zip:
15072
Country:
Peru
Status:
Recruiting
Contact:
Last name:
Ana Maria Glória Paredes Guerra, MD
Email:
gloriaparedes2000@yahoo.com
Facility:
Name:
China Medical University Children's Hospital
Address:
City:
Taichung
Zip:
40447
Country:
Taiwan
Status:
Recruiting
Contact:
Last name:
Su Min-Yu, MD
Email:
035515@tool.caaumed.org.tw
Contact backup:
Last name:
Ching-Tien Peng, MD
Email:
penect@gmail.com
Start date:
February 1, 2023
Completion date:
February 2033
Lead sponsor:
Agency:
Immune Oncology Research Institute
Agency class:
Other
Source:
Immune Oncology Research Institute
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT05711992
