AstroblastomaIRBTRNCT05697874
International Rare Brain Tumor Registry
An observational study of Astroblastoma and BCOR ITD Sarcoma, run by Children's National Research Institute at 13 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 45 years
- Conditions the study is for
- Astroblastoma, BCOR ITD Sarcoma, CNS Sarcoma
- How many people
- 5,800 participants
- Where
- 13 sitesin 4 countries
- Study dates
- 1 Jan 2023to Dec 2033
- How long will I take part?
- 10 Years
- 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 Up to 45 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: The study population will include patients who have completed the informed consent process and are <= to 45 years of age at diagnosis of the following: CNS Sarcoma, BCOR-altered tumor, Astroblastoma/MNI-1 altered tumors, histologically ambiguous/unclassifiable brain tumor, rare brain tumor. Patients from anywhere in the world are eligible for enrollment.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
13 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
13 sites in 4 countries
United States6 sites, 6 recruiting
- Recruiting
Akron Children's Hospital
Akron, Ohio, 44308
Erin Wright, MD, 3305438580, ewright@akronchildrens.org
Emily Blue, eblue@akronchildrens.org
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Miriam Bornhorst, MD, 2024762140, mbornhorst@luriechildrens.org
Kelly Bruno, kbruno@luriechildrens.org
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229-3026
Scott Raskin, DO, (513) 636-4200, scott.raskin@cchmc.org
Cecilia Grizzell, Cecilia.Grizzell@cchmc.org
- Recruiting
Children's Hospital Los Angeles
Los Angeles, California, 90027
Nathan Robison, MD, 323-361-4624, nrobison@chla.usc.edu
Eliana Romero, 323-361-8957, eromero@chla.usc.edu
- Recruiting
University of California San Francisco
San Francisco, California, 94158
Marie Jaeger-Krause, MD, 4154763831, marie.jaeger-krause@ucsf.edu
Cammy Britts, (650)-796-8115, Cammy.Britts@ucsf.edu
- Recruiting
Children's National Hospital
Washington D.C., District of Columbia, 20010
Adriana Fonseca, MD, 202-476-3899, afonsecash@childrensnational.org
Hope Lovell, 202-476-3898, hlovell@childrensnational.org
Israel4 sites, 4 recruiting
- Recruiting
Rambam Health Care Campus
Haifa, 3525409
Liat Oren, MD, 04-7774718, li_oren@rmc.gov.il
Hadar Aktin Moustcki, h_aktinmoustcki@rambam.health.gov.il
- Recruiting
Schneider Children's Medical Center- CLALIT Health Services
Petah Tikva, 4920235
Helen Toledano, MD, 054-8885563, Helent@clalit.org.il
Tehila Tannin, tehilatan@clalit.org.il
- Recruiting
Dana-Dwek Children's Hospital- Tel Aviv Medical Center
Tel Aviv, 6423906
Yair Peled, MD, +97236974444, yairpe@tlvmc.gov.il
Ziva Rosenthal Galili, zivarg@tlvmc.gov.il
- Recruiting
The Sheba Medical Center
Tel Litwinsky, 52621
Michal Oren, MD, PhD, 972-523-595718, michal@droren.co.il
Orit Savyon, orit.savyon@sheba.health.gov.il
Colombia2 sites, 2 recruiting
- Recruiting
HOMI - Fundación Hospital de la Misericordia
Bogotá, Bogota D.C., 111511
Edgar V Cabrera Bernal, MD, edgarcabrerabernal@gmail.com
Edgar V Cabrera Bernal, MD
- Recruiting
Clinica Del Country
Bogotá, 110221
Isabel C Sarmiento Urbina, MD, Isabel.sarmiento@clinicadelcountry.com
Silvia N Hernandez Rodríguez, silvia.hernandez@clinicadelcountry.com
Brazil1 site, 1 recruiting
- Recruiting
TUCCA - Santa Marcelina
São Paulo, São Paulo, 08270-070
Sidnei Epelman, MD, epelman@inctrbrasil.org
Carlos E Opazo, carlos.opazocpc@santamarcelina.org
About this study
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.
Read the 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.
- Keywords
- Rare Brain Tumor, Pediatric Brain Tumor Registry, Unclassifiable tumor, Pediatric Neuro-Oncology, Rare Disease
- Medical subject headings
- Neoplasms, Neuroepithelial, Neoplasms, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event-free Survival
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.
Measured: 10 years
Secondary outcomes (2)
Molecular characterization
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.
Measured: 10 years
Radiological characterization
To analyze conventional and advanced imaging findings (including diffusion weighted imaging) of each cohort and correlate them with clinical, histopathology and molecular data (radiogenomics).
Measured: 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jan 2023
- Study start1 Jan 2023
- Primary completionDec 2033 (estimated)
- Study completionDec 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AstroblastomaStudies 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 5,800 participants (estimated)
- Target duration
- 10 Years
Sponsor and investigators
- Lead sponsor
- Children's National Research Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Adriana Fonseca, Children's National Research Institute
- Investigators
- Adriana Fonseca, MD, Principal investigator, afonsecash@childrensnational.org
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05697874
- Sponsor's protocol ID
- STUDY00000324
- Acronym
- IRBTR
- Status last verified
- Mar 2026
- First submitted
- 23 Jan 2023
- Last updated
- 7 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: IPD will not be shared with other researchers.
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- International Rare Brain Tumor Registry
- ClinicalTrials.gov ID
- NCT05697874
- Acronym / sponsor ID
- IRBTR / STUDY00000324
- Keywords
- Rare Brain Tumor, Pediatric Brain Tumor Registry, Unclassifiable tumor, Pediatric Neuro-Oncology, Rare Disease
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 5,800 participants (estimated)
- Lead sponsor
- Children's National Research InstituteUniversity, hospital or other
- Study start
- 1 Jan 2023 (actual)
- Primary completion
- Dec 2033 (estimated)
- Study completion
- Dec 2033 (estimated)
- First posted
- 26 Jan 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.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 10 Years
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- Up to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- The study population will include patients who have completed the informed consent process and are <= to 45 years of age at diagnosis of the following: CNS Sarcoma, BCOR-altered tumor, Astroblastoma/MNI-1 altered tumors, histologically ambiguous/unclassifiable brain tumor, rare brain tumor. Patients from anywhere in the world are eligible for enrollment.
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (3 listed)
- 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.
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
Event-free Survival
Time frame: 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.
2 secondary outcomes
Molecular characterization
Time frame: 10 years
Radiological characterization
Time frame: 10 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 7 Aug 2026.
- Individual participant dataSharing planned: No. IPD will not be shared with other researchers.
Related trials for Astroblastoma
Recruiting
Ultra-Rapid Profiling and Drug Screening for Treatment Selection in Patients With Relapsed or Recurrent Rare Brain Tumors
Rare CNS Tumor, FOXR2-activated
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.
Adriana Fonseca, MD principal investigator
afonsecash@childrensnational.org
13 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Astroblastoma and BCOR ITD Sarcoma, run by Children's National Research Institute at 13 sites in 4 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 6 inclusion and 3 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 7 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States, Israel, Colombia, Brazil. 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 Adriana Fonseca, MD (202-476-8399, afonsecash@childrensnational.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Event-free Survival (time frame: 10 years). 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 7 Aug 2026.