Anaplastic AstrocytomaNCT05934630
Testing Cerebrospinal Fluid for Cell-free Tumor DNA in Children, Adolescents, and Young Adults With Brain Tumors
An observational study of Anaplastic Astrocytoma and Diffuse Brainstem Glioma, run by Pediatric Brain Tumor Consortium at 10 sites in 2 countries.
Trial summary
Recent advances in technology have allowed for the detection of cell-free DNA (cfDNA). cfDNA is tumor DNA that can be found in the fluid that surrounds the brain and spinal cord (called cerebrospinal fluid or CSF) and in the blood of patients with brain tumors. Read more about this study
- Who is it for?
- Ages Up to 40 years
- Conditions the study is for
- Anaplastic Astrocytoma, Diffuse Brainstem Glioma, Glioblastoma Multiforme
- How many people
- 57 participants
- Where
- 10 sitesin 2 countries
- Study dates
- 12 Jul 2023to 17 Apr 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
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 40 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Patients will enroll from and be seen at Pediatric Brain Tumor Consortium sites and will meet the eligibility criteria in order to participate.
Who may be able to join
- All patients must have a known or suspected diagnosis (based on pathology or imaging) of a primary brain tumor. All children and adolescent/young adult (AYA) patients =\< 21 years of age are eligible.
- AYA patients \< 40 are eligible with a primary brain tumor entity more common in children than adults. This includes:
- medulloblastoma
- non-medulloblastoma embryonal brain tumors
- atypical teratoid rhabdoid tumors (ATRT)
- ependymoma
Show all 26Show fewer
- CNS germ cell tumors
- Diffuse midline glioma, H3K27M-altered
- Diffuse hemispheric glioma, H3 G34-mutant
- pineoblastoma
- diffuse leptomeningeal glioneuronal tumor
- diffuse brainstem glioma
- pilocytic astrocytoma
- choroid plexus carcinoma
- ELIGIBLE PATIENTS WILL BE STRATIFIED BY DIAGNOSIS AS FOLLOWS:
- Stratum 1: Medulloblastoma
- Stratum 2: High-grade glioma (IDH-wildtype) and DIPG
- o DIPG patients must meet clinical and imaging requirements for DIPG diagnosis. Patients with newly diagnosed diffuse intrinsic pontine gliomas (DIPGs), defined as tumors with a pontine epicenter and diffuse involvement of 2/5 or more of the pons, are eligible without histologic confirmation. Patients with brainstem tumors that do not meet these criteria or not considered to be typical intrinsic pontine gliomas will only be eligible for Stratum B if the tumors have been biopsied and are proven to be an anaplastic astrocytoma, glioblastoma multiforme, gliosarcoma, anaplastic mixed glioma or fibrillary astrocytoma.
- Stratum 3: Low-grade glioma (IDH-wildtype) with multifocal/disseminated and/or leptomeningeal disease
- Stratum 4: Diffuse Leptomeningeal Glioneuronal Tumor
- Stratum 5: Pineoblastoma
- Stratum 6: All other eligible tumor types
- DISEASE STATUS: Participants will be eligible at any stage of disease.
- AGE: All children and adolescent/young adult (AYA) patients =\< 21 years of age are eligible. AYA patients \< 40 are eligible with a primary brain tumor entity more common in children than adults
- CEREBROSPINAL FLUID (CSF) COLLECTION: Patients must have a clinical indication for at least one CSF (lumbar, cisternal or ventricular) collection, or clinical circumstance where CSF sampling is feasible with no or minimal risk (e.g., endoscopic third ventriculostomy, external ventricular drain).
- INFORMED CONSENT: The patient or parent/guardian can understand the consent and is willing to sign a written informed consent document according to institutional guidelines.
Who cannot join
- DIAGNOSIS: Given the extremely low rate of CSF cfDNA positivity in patients with localized, non-disseminated low-grade glioma, those patients will be excluded from participation.
Trial locations and contacts
10 sites in 2 countries.
10 sites in 2 countries
United States9 sites
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, 45229
Nationwide Childrens Hospital
Columbus, Ohio, 43205
Texas Children's Hospital
Houston, Texas, 77030
Children's Hospital of Los Angeles
Los Angeles, California, 90026
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Lucille Packard Children's Hospital Stanford University
Palo Alto, California, 94304
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: CSF Cell-free Tumor DNA (CSF cfDNA) Liquid Biopsies for Pediatric, Adolescent, and Young Adult Patients With Primary Brain Tumors
Recent advances in technology have allowed for the detection of cell-free DNA (cfDNA). cfDNA is tumor DNA that can be found in the fluid that surrounds the brain and spinal cord (called cerebrospinal fluid or CSF) and in the blood of patients with brain tumors. The detection of cfDNA in blood and CSF is known as a "liquid biopsy" and is non-invasive, meaning it does not require a surgery or biopsy of tumor tissue. Multiple studies in other cancer types have shown that cfDNA can be used for diagnosis, to monitor disease response to treatment, and to understand the genetic changes that occur in brain tumors over time. Study doctors hope that by studying these tests in pediatric brain tumor patients, they will be able to use liquid biopsy in place of tests that have more risks for patients, like surgery.
There is no treatment provided on this study. Patients who have CSF samples taken as part of regular care will be asked to provide extra samples for this study. The study doctor will collect a minimum of one extra tube of CSF (about 1 teaspoon or 5 mL) for this study. If the patients doctor thinks it is safe, up to 2 tubes of CSF (about 4 teaspoons or up to 20 mL) may be collected. CSF will be collected through the indwelling catheter device or through a needle inserted into the lower part of the patient's spine (known as a spinal tap or lumbar puncture).
Read more
A required blood sample (about ½ a teaspoon or 2 3 mL) will be collected once at the start of the study. This sample will be used to help determine changes found in the CSF. Blood will be collected from the patient's central line or arm as a part of regular care.
An optional tumor tissue if obtained within 8 weeks of CSF collection will be collected if available. Similarities between changes in the DNA of the tissue that has caused the tumor to form and grow with the cfDNA from CSF will be compared. This will help understand if CSF can be used instead of tumor tissue for diagnosis. Up to 300 people will take part in this study.
This study will use genetic tests that may identify changes in the genes in the CSF. The report of the somatic mutations (the mutations that are found in the tumor only) will become part of the medical record. The results of the cfDNA sequencing will be shared with the patient. The study doctor will discuss what the results mean for the patient and patient's diagnosis and treatment. Looking for inheritable mutations in normal cells (blood) is not the purpose of this study. Genetic tests of normal blood can reveal information about the patient and also about the their relatives. The doctor will discuss what the tests results may mean for the patient and the their family. Patient may be monitored on this study for up to 5 years.
Read the detailed description
This is a multicenter study for children and young adults with primary brain tumor. All children and adolescent/young adult (AYA) patients 21 years of age are eligible. AYA patients \&amp;lt; 40 are eligible with a primary brain tumor entity more common in children than adults. Up to 300 people will take part in this study.
Pediatric central nervous system (CNS) tumors represent a wide range of disorders and continue to be the leading cause of cancer-related death in children and adolescents. The past few years have led to a dramatic shift in the diagnosis and pathological classification of pediatric CNS tumors. Molecular biomarkers which are now being incorporated into treatment decisions and clinical trial design. With this, the need for longitudinal molecular monitoring of disease becomes increasingly evident. The anatomical challenges in accessing pediatric CNS malignancies coupled with the move toward molecular sub-classification and molecular targeted therapies leads to an urgent need to develop a non-invasive way to repeatedly sample these tumors. This would enable: 1) diagnose tumors that are surgically inaccessible; 2) monitor molecular changes in tumor longitudinally, including at progression and/or recurrence; and 3) develop non-invasive ways to monitor treatment responses. Recent advances in technology have allowed for the detection of cell-free tumor DNA (cfDNA), i.e., small fragments of tumor DNA, shed into the cerebrospinal fluid (CSF) and bloodstream in brain tumor patients through a process known as "liquid biopsy".
Based on the successful preliminary studies in adult glioma patients, this Pediatric Brain Tumor Consortium (PBTC)-sponsored effort will attempt to integrate CSF cfDNA liquid biopsies into the clinical care of pediatric brain tumor patients across the PBTC to enhance tumor diagnosis/molecular subclassification and guide therapeutic decision making. This will be accomplished through centralized collection and processing of CSF cfDNA samples from patients with various types of pediatric CNS tumors across PBTC member sites. The primary objective will be to investigate the concordance between CSF cfDNA alterations and tumor DNA alterations in matched pairs to understand: 1) the concordance across matched samples; and 2) the genomic evolution that occurs over time across various tumor types. Additionally, the secondary objectives and exploratory objectives in this study are intended to aggregate data across various types of pediatric primary brain tumors to understand the rate of success of CSF cfDNA molecular profiling in different disease types and at various stages of disease, and to elucidate the role of CSF cfDNA profiling in guiding clinical decision making.
The primary objective of this study is to estimate the concordance of mutations detected in the tumor tissue vs. cerebrospinal fluid cell free DNA (CSF cfDNA) across samples collected within 8 weeks of each other; determine whether clonal mutations are likely to be shared across sample types.
Patients who have CSF samples taken as part of regular care will be asked to provide extra samples for this study. The study doctor will collect a minimum of one extra tube of CSF (about 1 teaspoon or 5 mL) for this study. If the patient's doctor thinks it is safe, up to 2 tubes of CSF (about 4 teaspoons or up to 20 mL) may be collected. A required blood sample (about ½ a teaspoon or 2 - 3 mL) will be collected once at the start of the study. An optional tumor tissue if obtained within 8 weeks of CSF collection will be collected if available. There is no treatment provided on this study.
- Conditions
- Anaplastic Astrocytoma, Diffuse Brainstem Glioma, Glioblastoma Multiforme, High-grade Astrocytoma NOS, Fibrillary Astrocytoma, Low-Grade Astrocytoma, Nos
- Keywords
- Liquid biopsy, CSF, Cerebrospinal fluid, cfDNA, ctDNA, Pediatric central nervous system tumors, Brain tumor
- Medical subject headings
- Astrocytoma, Glioblastoma, Choroid Plexus Carcinoma, Central Nervous System Neoplasms, Rhabdoid Tumor, Medulloblastoma, Ependymoma, Oligodendroglioma
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 |
|---|---|---|
| Stratum 1: Medulloblastoma | Participants in Arm 1 must have diagnosis of Medulloblastoma. All children and adolescent/young adult (AYA) patients <= 21 years of age are eligible. AYA patients < 40 are eligible with a primary brain tumor entity more common in children than adults. | |
| Stratum 2: High-grade Glioma (IDH-wildtype) or Diffuse Intrinsic Pontine Glioma | Participants in Arm 2 must have diagnosis of High-grade Glioma (IDH-wildtype) or Diffuse Intrinsic Pontine Glioma (DIPG). All children and adolescent/young adult (AYA) patients <= 21 years of age are eligible. AYA patients < 40 are eligible with a primary brain tumor entity more common in children than adults. DIPG patients must meet clinical and imaging requirements for DIPG diagnosis. Patients with newly diagnosed DIPGs, defined as tumors with a pontine epicenter and diffuse involvement of 2/5 or more of the pons, are eligible without histologic confirmation. Patients with brainstem tumors that do not meet these criteria or not considered to be typical intrinsic pontine gliomas will only be eligible for Stratum 2 if the tumors have been biopsied and are proven to be an anaplastic astrocytoma, glioblastoma multiforme, gliosarcoma, anaplastic mixed glioma or fibrillary astrocytoma. | |
| Stratum 3: Low-grade Glioma IDH-wildtype with multifocal/disseminated and/or leptomeningeal disease | Participants in Arm 3 must have diagnosis of Low-grade Glioma (IDH-wildtype) with multifocal/disseminated and/or leptomeningeal disease. All children and adolescent/young adult (AYA) patients =< 21 years of age are eligible. AYA patients < 40 are eligible with a primary brain tumor entity more common in children than adults. | |
| Stratum 4: Diffuse Leptomeningeal Glioneuronal Tumor | Participants in Arm 4 must have diagnosis of Diffuse Leptomeningeal Glioneuronal Tumor. All children and adolescent/young adult (AYA) patients =< 21 years of age are eligible. AYA patients < 40 are eligible with a primary brain tumor entity more common in children than adults. | |
| Stratum 5: Pineoblastoma | Participants in Arm 5 must have diagnosis of Pineoblastoma. All children and adolescent/young adult (AYA) patients =< 21 years of age are eligible. AYA patients < 40 are eligible with a primary brain tumor entity more common in children than adults. | |
| Stratum 6: All other eligible primary brain tumor types | Participants in Arm 6 must have diagnosis of all other eligible primary brain tumor types. All children and adolescent/young adult (AYA) patients =< 21 years of age are eligible. AYA patients < 40 are eligible with a primary brain tumor entity more common in children than adults. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Concordance of mutations across the tumor tissue vs. CSF
Within specific histologies with paired samples, estimate the concordance of mutation detected in the tumor tissue vs. cerebrospinal fluid cell free DNA (CSF cfDNA) across samples collected within 8 weeks of each other.
Measured: 3 months
Secondary outcomes (2)
Estimate the frequency of detection of CSF cfDNA across different types of pediatric CNS tumors in association with various disease states.
Intra-patient matched samples of CSF and tissue will be summarized for each oncogenic alteration and scored as positive vs. negative.
Measured: 3 months
Track and estimate the correlation between the levels of CSF cfDNA and disease response as determined by clinical and imaging criteria across different disease types.
Associations between the quantity of cfDNA and standard clinical and imaging disease response will be assessed by either Wilcoxon rank-sum or Spearman's correlations.
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jul 2023
- Study start12 Jul 2023
- Primary completion17 Apr 2026
- Study completion17 Apr 2026
Why it stopped: The NCI will not extend the PBTC grant beyond March 31, 2026.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anaplastic AstrocytomaStudies 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
- 57 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pediatric Brain Tumor Consortium
- Sponsor type
- Research network
- Collaborators
- Memorial Sloan Kettering Cancer Center, National Cancer Institute (NCI), American Lebanese Syrian Associated Charities
- Responsible party
- Sponsor
- Investigators
- Alexandra Miller, MD, PhD, Study chair, NYU Langone Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05934630
- Sponsor's protocol ID
- PBTC-N14
- Other IDs
- UM1CA081457, NCI-2023-02321, PBTC-N14, PBTC-N14
- Status last verified
- Apr 2026
- First submitted
- 28 Jun 2023
- Last updated
- 23 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: The Pediatric Brain Tumor Consortium (PBTC) ensures that high dimensional molecular data generated from PBTC samples will be submitted to the NIH Database of Genotypes and Phenotypes (dbGaP) or other NIH-designated data repository. PBTC is required to make data available to other investigators for use in research projects. An investigator requesting data from published PBTC studies will submit a proposal describing the studies from which data are requested, the requested data, and a list of investigators in the project and their affiliated institutions, along with the investigator's CV to the PBTC Steering Committee for review. Once approved, the requesting investigator will be asked to complete a Data Transfer Agreement before the release of deidentified data. Requests for data are typically only considered once the primary study manuscript has been published though exceptions may be made in the event of long delays in the primary study data publication.
Study at a glance
From the registry record, last updated by the sponsor on 23 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- CSF Cell-free Tumor DNA (CSF cfDNA) Liquid Biopsies for Pediatric, Adolescent, and Young Adult Patients With Primary Brain Tumors
- ClinicalTrials.gov ID
- NCT05934630
- Acronym / sponsor ID
- PBTC-N14
- Conditions
- Anaplastic Astrocytoma, Diffuse Brainstem Glioma, Glioblastoma Multiforme, High-grade Astrocytoma NOS, Fibrillary Astrocytoma, Low-Grade Astrocytoma, Nos
- Keywords
- Liquid biopsy, CSF, Cerebrospinal fluid, cfDNA, ctDNA, Pediatric central nervous system tumors, Brain tumor
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: The NCI will not extend the PBTC grant beyond March 31, 2026.
- Enrolment
- 57 participants (actual)
- Lead sponsor
- Pediatric Brain Tumor ConsortiumResearch network
- Collaborators
- Memorial Sloan Kettering Cancer Center, National Cancer Institute (NCI), American Lebanese Syrian Associated Charities
- Study start
- 12 Jul 2023 (actual)
- Primary completion
- 17 Apr 2026 (actual)
- Study completion
- 17 Apr 2026 (actual)
- First posted
- 7 Jul 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.
Recent advances in technology have allowed for the detection of cell-free DNA (cfDNA). cfDNA is tumor DNA that can be found in the fluid that surrounds the brain and spinal cord (called cerebrospinal fluid or CSF) and in the blood of patients with brain tumors. The detection of cfDNA in blood and CSF is known as a "liquid biopsy" and is non-invasive, meaning it does not require a surgery or biopsy of tumor tissue. Multiple studies in other cancer types have shown that cfDNA can be used for diagnosis, to monitor disease response to treatment, and to understand the genetic changes that occur in brain tumors over time. Study doctors hope that by studying these tests in pediatric brain tumor patients, they will be able to use liquid biopsy in place of tests that have more risks for patients, like surgery.
There is no treatment provided on this study. Patients who have CSF samples taken as part of regular care will be asked to provide extra samples for this study. The study doctor will collect a minimum of one extra tube of CSF (about 1 teaspoon or 5 mL) for this study. If the patients doctor thinks it is safe, up to 2 tubes of CSF (about 4 teaspoons or up to 20 mL) may be collected. CSF will be collected through the indwelling catheter device or through a needle inserted into the lower part of the patient's spine (known as a spinal tap or lumbar puncture).
A required blood sample (about ½ a teaspoon or 2 3 mL) will be collected once at the start of the study. This sample will be used to help determine changes found in the CSF. Blood will be collected from the patient's central line or arm as a part of regular care.
An optional tumor tissue if obtained within 8 weeks of CSF collection will be collected if available. Similarities between changes in the DNA of the tissue that has caused the tumor to form and grow with the cfDNA from CSF will be compared. This will help understand if CSF can be used instead of tumor tissue for diagnosis. Up to 300 people will take part in this study.
This study will use genetic tests that may identify changes in the genes in the CSF. The report of the somatic mutations (the mutations that are found in the tumor only) will become part of the medical record. The results of the cfDNA sequencing will be shared with the patient. The study doctor will discuss what the results mean for the patient and patient's diagnosis and treatment. Looking for inheritable mutations in normal cells (blood) is not the purpose of this study. Genetic tests of normal blood can reveal information about the patient and also about the their relatives. The doctor will discuss what the tests results may mean for the patient and the their family. Patient may be monitored on this study for up to 5 years.
Detailed description
This is a multicenter study for children and young adults with primary brain tumor. All children and adolescent/young adult (AYA) patients 21 years of age are eligible. AYA patients \&amp;lt; 40 are eligible with a primary brain tumor entity more common in children than adults. Up to 300 people will take part in this study.
Pediatric central nervous system (CNS) tumors represent a wide range of disorders and continue to be the leading cause of cancer-related death in children and adolescents. The past few years have led to a dramatic shift in the diagnosis and pathological classification of pediatric CNS tumors. Molecular biomarkers which are now being incorporated into treatment decisions and clinical trial design. With this, the need for longitudinal molecular monitoring of disease becomes increasingly evident. The anatomical challenges in accessing pediatric CNS malignancies coupled with the move toward molecular sub-classification and molecular targeted therapies leads to an urgent need to develop a non-invasive way to repeatedly sample these tumors. This would enable: 1) diagnose tumors that are surgically inaccessible; 2) monitor molecular changes in tumor longitudinally, including at progression and/or recurrence; and 3) develop non-invasive ways to monitor treatment responses. Recent advances in technology have allowed for the detection of cell-free tumor DNA (cfDNA), i.e., small fragments of tumor DNA, shed into the cerebrospinal fluid (CSF) and bloodstream in brain tumor patients through a process known as "liquid biopsy".
Based on the successful preliminary studies in adult glioma patients, this Pediatric Brain Tumor Consortium (PBTC)-sponsored effort will attempt to integrate CSF cfDNA liquid biopsies into the clinical care of pediatric brain tumor patients across the PBTC to enhance tumor diagnosis/molecular subclassification and guide therapeutic decision making. This will be accomplished through centralized collection and processing of CSF cfDNA samples from patients with various types of pediatric CNS tumors across PBTC member sites. The primary objective will be to investigate the concordance between CSF cfDNA alterations and tumor DNA alterations in matched pairs to understand: 1) the concordance across matched samples; and 2) the genomic evolution that occurs over time across various tumor types. Additionally, the secondary objectives and exploratory objectives in this study are intended to aggregate data across various types of pediatric primary brain tumors to understand the rate of success of CSF cfDNA molecular profiling in different disease types and at various stages of disease, and to elucidate the role of CSF cfDNA profiling in guiding clinical decision making.
The primary objective of this study is to estimate the concordance of mutations detected in the tumor tissue vs. cerebrospinal fluid cell free DNA (CSF cfDNA) across samples collected within 8 weeks of each other; determine whether clonal mutations are likely to be shared across sample types.
Patients who have CSF samples taken as part of regular care will be asked to provide extra samples for this study. The study doctor will collect a minimum of one extra tube of CSF (about 1 teaspoon or 5 mL) for this study. If the patient's doctor thinks it is safe, up to 2 tubes of CSF (about 4 teaspoons or up to 20 mL) may be collected. A required blood sample (about ½ a teaspoon or 2 - 3 mL) will be collected once at the start of the study. An optional tumor tissue if obtained within 8 weeks of CSF collection will be collected if available. There is no treatment provided on this study.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Stratum 1: Medulloblastoma | – | –Participants in Arm 1 must have diagnosis of Medulloblastoma. All children and adolescent/young adult (AYA) patients <= 21 years of age are eligible. AYA patients < 40 are eligible with a primary brain tumor entity more common in children than adults. |
| Stratum 2: High-grade Glioma (IDH-wildtype) or Diffuse Intrinsic Pontine Glioma | – | –Participants in Arm 2 must have diagnosis of High-grade Glioma (IDH-wildtype) or Diffuse Intrinsic Pontine Glioma (DIPG). All children and adolescent/young adult (AYA) patients <= 21 years of age are eligible. AYA patients < 40 are eligible with a primary brain tumor entity more common in children than adults. DIPG patients must meet clinical and imaging requirements for DIPG diagnosis. Patients with newly diagnosed DIPGs, defined as tumors with a pontine epicenter and diffuse involvement of 2/5 or more of the pons, are eligible without histologic confirmation. Patients with brainstem tumors that do not meet these criteria or not considered to be typical intrinsic pontine gliomas will only be eligible for Stratum 2 if the tumors have been biopsied and are proven to be an anaplastic astrocytoma, glioblastoma multiforme, gliosarcoma, anaplastic mixed glioma or fibrillary astrocytoma. |
| Stratum 3: Low-grade Glioma IDH-wildtype with multifocal/disseminated and/or leptomeningeal disease | – | –Participants in Arm 3 must have diagnosis of Low-grade Glioma (IDH-wildtype) with multifocal/disseminated and/or leptomeningeal disease. All children and adolescent/young adult (AYA) patients =< 21 years of age are eligible. AYA patients < 40 are eligible with a primary brain tumor entity more common in children than adults. |
| Stratum 4: Diffuse Leptomeningeal Glioneuronal Tumor | – | –Participants in Arm 4 must have diagnosis of Diffuse Leptomeningeal Glioneuronal Tumor. All children and adolescent/young adult (AYA) patients =< 21 years of age are eligible. AYA patients < 40 are eligible with a primary brain tumor entity more common in children than adults. |
| Stratum 5: Pineoblastoma | – | –Participants in Arm 5 must have diagnosis of Pineoblastoma. All children and adolescent/young adult (AYA) patients =< 21 years of age are eligible. AYA patients < 40 are eligible with a primary brain tumor entity more common in children than adults. |
| Stratum 6: All other eligible primary brain tumor types | – | –Participants in Arm 6 must have diagnosis of all other eligible primary brain tumor types. All children and adolescent/young adult (AYA) patients =< 21 years of age are eligible. AYA patients < 40 are eligible with a primary brain tumor entity more common in children than adults. |
Who may qualify
- Ages
- Up to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients will enroll from and be seen at Pediatric Brain Tumor Consortium sites and will meet the eligibility criteria in order to participate.
Key inclusion criteria (26 listed)
- All patients must have a known or suspected diagnosis (based on pathology or imaging) of a primary brain tumor. All children and adolescent/young adult (AYA) patients =\< 21 years of age are eligible.
- AYA patients \< 40 are eligible with a primary brain tumor entity more common in children than adults. This includes:
- medulloblastoma
- non-medulloblastoma embryonal brain tumors
- atypical teratoid rhabdoid tumors (ATRT)
Key exclusion criteria (1 listed)
- DIAGNOSIS: Given the extremely low rate of CSF cfDNA positivity in patients with localized, non-disseminated low-grade glioma, those patients will be excluded from participation.
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
Concordance of mutations across the tumor tissue vs. CSF
Time frame: 3 months
Within specific histologies with paired samples, estimate the concordance of mutation detected in the tumor tissue vs. cerebrospinal fluid cell free DNA (CSF cfDNA) across samples collected within 8 weeks of each other.
2 secondary outcomes
Estimate the frequency of detection of CSF cfDNA across different types of pediatric CNS tumors in association with various disease states.
Time frame: 3 months
Track and estimate the correlation between the levels of CSF cfDNA and disease response as determined by clinical and imaging criteria across different disease types.
Time frame: 3 months
Results and safety
The study was stopped early (reason given: The NCI will not extend the PBTC grant beyond March 31, 2026.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Apr 2026.
- Individual participant dataSharing planned: Yes. The Pediatric Brain Tumor Consortium (PBTC) ensures that high dimensional molecular data generated from PBTC samples will be submitted to the NIH Database of Genotypes and Phenotypes (dbGaP) or other NIH-designated data repository. PBTC is required to make data available to other investigators for use in research projects. An investigator requesting data from published PBTC studies will submit a proposal describing the studies from which data are requested, the requested data, and a list of investigators in the project and their affiliated institutions, along with the investigator's CV to the PBTC Steering Committee for review. Once approved, the requesting investigator will be asked to complete a Data Transfer Agreement before the release of deidentified data. Requests for data are typically only considered once the primary study manuscript has been published though exceptions may be made in the event of long delays in the primary study data publication.
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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. This study is not recruiting at the moment.
Alexandra Miller, MD, PhD study chair
NYU Langone Health
10 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Anaplastic Astrocytoma and Diffuse Brainstem Glioma, run by Pediatric Brain Tumor Consortium at 10 sites in 2 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 26 inclusion and 1 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: stopped early, as of the sponsor's last update on 23 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Canada. 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?
The study was stopped early (reason given: The NCI will not extend the PBTC grant beyond March 31, 2026.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Concordance of mutations across the tumor tissue vs. CSF (time frame: 3 months). 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 23 Apr 2026.