IRBTR: International Rare Brain Tumor Registry

Sponsor

Children's National Research Institute (Other)

Overall Status

Recruiting

CT.gov ID

NCT05697874

Collaborator

(none)

5,800

Enrollment

Location

131

Anticipated Duration (Months)

44.3

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Astroblastoma BCOR ITD Sarcoma CNS Sarcoma Unclassified Tumor, Malignant

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, and other 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.

Study Design

Study Type:

Observational [Patient Registry]

Anticipated Enrollment :

5800 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

International Rare Brain Tumor Registry

Actual Study Start Date :

Jan 1, 2023

Anticipated Primary Completion Date :

Dec 1, 2033

Anticipated Study Completion Date :

Dec 1, 2033

Arms and Interventions

Arm	Intervention/Treatment
CNS Sarcoma Patients diagnosed with Central nervous system (CNS) sarcomas
BCOR-altered Patients diagnosed with tumors characterized by alterations in the BCOR gene.
Astroblastoma/MN-1- altered Patients diagnosed with Astroblastomas/MN-1 alterations
Unclassifiable tumors Patients diagnosed with histologically ambiguous tumors or tumors that fail to classify with the current diagnostic methods.
Other Rare Brain tumors Patients diagnosed with other rare brain tumors that do not meet the criteria for cohorts 1-4.

Outcome Measures

Primary Outcome Measures

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 Outcome Measures

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).

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 45 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.