Astroblastoma, BCOR ITD Sarcoma, CNS Sarcoma, Unclassified Tumor, Malignant
Conditions
Keywords
Rare Brain Tumor, Pediatric Brain Tumor Registry, Unclassifiable tumor, Pediatric Neuro-Oncology, Rare Disease
Brief summary
The objective of the International Rare Brain Tumor Registry (IRBTR) is to better understand rare brain tumors through the collection of biospecimens and matched clinical data of children, adolescents, and young adult patients diagnosed with rare brain tumors.
Detailed description
The International Rare Brain Tumor Registry (IRBTR) is a prospective observational study that will collect tumor samples and matched clinical and radiological data to better understand the outcomes of patients with rare brain tumors in particular: CNS sarcoma, BCOR, MN-1 altered tumors, PLAG/L1, and other rare or unclassified rare brain tumors. Data collected include demographics, disease characteristics, treatment information, radiological imaging, and biospecimen collection if available ( tumor tissues Patients will be followed longitudinally to obtain outcome data. Data collection will continue for approximately 10 years.
Interventions
None listed
Sponsors
Children's National Research Institute
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
No minimum to 45 Years
Healthy volunteers
No
Inclusion criteria
* Patients with a known or suspected CNS Sarcoma. * Patients with a known or suspected BCOR-altered brain tumor * Patients with a known or suspected Astroblastoma/NM-1 altered brain tumor * Patients with known or suspected histologically ambiguous/unclassifiable brain tumor * Patients with a known or suspected rare brain tumor. * Signed informed consent by patient/ parent or guardian (assent where applicable) to participate in the study.
Exclusion criteria
* The patient has an extra-CNS primary tumor. * The patient is older than 46 years of age at diagnosis. * The patient or family is not willing to participate or does not sign informed consent.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Event-free Survival | 10 years | The primary outcome measure will be time from diagnosis to an event, defined as the occurrence of progression or recurrence of the disease, occurrence of a second malignant neoplasm, or death from any cause. Each cohort will be analyzed separately. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Molecular characterization | 10 years | To determine molecular characteristics of each cohort using somatic and germline SNV calling, methylation profiling, fusion calling and gene expression profiling. Molecular findings will be correlated with clinical characteristics to identify risk factors and subgroup-specific therapeutic susceptibilities. |
| Radiological characterization | 10 years | To analyze conventional and advanced imaging findings (including diffusion weighted imaging) of each cohort and correlate them with clinical, histopathology and molecular data (radiogenomics). |
Countries
Brazil, Colombia, Israel, United States
Contacts
CONTACTAdriana Fonseca, MD
CONTACTHope Lovell
PRINCIPAL_INVESTIGATORAdriana Fonseca, MD
afonsecash@childrensnational.org
Outcome results
None listed