Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT05711992 |
| Other study ID # |
IMMONC0005 |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
February 1, 2023 |
| Est. completion date |
February 2033 |
Study information
| Verified date |
December 2023 |
| Source |
Immune Oncology Research Institute |
| Contact |
Julieta Hoveyan, MD |
| Phone |
+374 10 28 38 00 |
| Email |
julia.hoveyan95[@]gmail.com |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical 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. 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.
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.
