Phase 2 Brain Cancer Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

General (most cohorts, ages 2–39):

• People aged 2 to 39 years with a diagnosis of a brain or spinal cord tumour called Diffuse Midline Glioma (DMG), confirmed by imaging and/or tissue testing
• People whose body weight is at least 10 kilograms
• People whose blood counts, kidney function, liver function, and heart function meet specific minimum levels as measured by blood tests and other assessments
• People who have recovered from side effects of any previous cancer treatments before joining
• People with a seizure disorder may be considered if their seizures are well controlled
• People who are able to carry out at least some daily activities, as measured by a standard functioning scale (specific score requirements apply depending on age and cohort)
• People who are willing and able to provide tumour tissue samples (at least 10–20 unstained tissue slides or one tissue block with sufficient tumour content)
• People who have had previous exposure to the study drug ONC201 may be considered, with some exceptions (confirm with trial site)
• Females who could become pregnant and male participants must agree to use adequate contraception during the trial

Cohort 1 (newly diagnosed DMG, before or at time of starting radiation):

• People newly diagnosed with DMG who are able to begin standard radiation therapy within 6 weeks of diagnosis
• In some sub-groups, tissue confirmation of specific tumour features (H3K27M mutation or certain other tumour types) is required

Cohort 2 (newly diagnosed DMG, after completing radiation):

• People with DMG who have completed standard radiation therapy, where radiation began within 6 weeks of diagnosis, and who are enrolling between 4 and 14 weeks after finishing radiation

Cohort 3 (DMG that has come back after treatment):

• People whose DMG has shown signs of returning or growing after previous treatment, who have not yet received any treatment for this recurrence, and who have not previously had repeat radiation therapy

Cohort 4 (newly diagnosed or progressed DMG, not eligible for other ONC201 trials):

• People with DMG who are not currently eligible for any other clinical trial that includes the drug ONC201, across three stages: before radiation, after radiation, or at the time of disease progression
• Specific sub-groups require tissue confirmation of H3K27-altered tumour features

Cohort 5 (DMG with specific targetable genetic changes, not eligible for other ONC201 trials):

• People with DMG whose tumour has one of the following specific genetic changes: BRAFV600E, PDGFRA (mutation or amplification), FGFR1 (mutation, fusion, or amplification), or NF1
• People with disease that has spread in certain ways (multifocal or leptomeningeal disease) may be considered for this cohort
• Not currently eligible for any other clinical trial that includes ONC201

Cohort 6 (newly diagnosed DMG in the thalamus or pons, after radiation — different requirements apply):

• People aged 2–39 with a newly diagnosed DMG located specifically in the thalamus or pons (not spinal cord), who have completed standard radiation therapy, with radiation having started within 6 weeks of diagnosis, and enrolling between 4 and 14 weeks after finishing radiation
• People who are assessed as medically suitable for anaesthesia and surgery
• People whose functioning meets a higher minimum level than other cohorts (confirm specific score with trial site)
• People who have not received a live vaccine within 30 days before the study drug is given, and who agree not to receive live vaccines during the study

Who may not be able to join:

General (most cohorts):

• People who have a tumour type classified as H3 wildtype grade II diffuse astrocytoma
• People who are currently taking another experimental drug as part of another trial
• People who are currently receiving other cancer treatments
• People with a known immune system disorder such as HIV, hepatitis B or C, or an autoimmune condition requiring certain medications
• People with an infection or other serious illness that is not currently under control
• Females who are pregnant or breastfeeding, or who have a positive pregnancy test before starting treatment
• People with active illegal drug use or a diagnosis of alcoholism
• People who have had allergic reactions to drugs with a similar makeup to those used in this trial
• People whose cancer has spread widely, including to the fluid around the brain and spinal cord (except in Cohort 5, where some spread may be allowed)
• People with another active or recently treated cancer (within 3 years of starting the study drug)
• People taking certain medications that strongly affect how the body processes drugs (specific enzyme inhibitors or inducers, including some seizure medications — confirm with trial site)
• People who previously took part in certain Chimerix-sponsored trials of ONC201 in the upfront (newly diagnosed) setting may not be eligible (confirm with trial site)

Cohort 1:

• People who have previously received radiation therapy
• People with DMG located in the thalamus or cerebellum with a specific tumour feature (H3K27M mutation)

Cohort 2:

• People with DMG not centred in the pons or spinal cord, specifically those with thalamic or cerebellar H3K27M DMG who received standard radiation without any additional treatment alongside it (other than temozolomide)
• People in sub-groups 1A and 2A who are assessed as not suitable for a tumour biopsy or resection procedure

Cohort 3:

• People who have previously had repeat radiation treatment for tumour progression
• People with thalamic or cerebellar H3K27M mutant DMG (with some exceptions — confirm with trial site)

Cohorts 4 and 5:

• People with thalamic or cerebellar H3K27M mutant DMG, with some limited exceptions for people who received ONC201 from certain sources before 2024, or US patients enrolled under specific regulatory review conditions (confirm with trial site)
• People who have previously received radiation (for sub-groups starting before radiation)
• People who have previously received repeat radiation for progression (for sub-groups at the time of progression)

Cohort 6:

• People whose DMG is located outside the thalamus or pons, including bilateral thalamic tumours
• People who are assessed as having unacceptable risk from anaesthesia or surgery
• People whose tumour is causing significant pressure or shifting of brain structures, as seen on imaging
• People with a known history of abnormal blood clotting or a significant bleeding episode within the past 12 months
• People who require blood-thinning medication that cannot be safely paused around surgical procedures
• People with an active viral infection or who are currently receiving antiviral treatment
• People with known or suspected immune system disorders, including inherited or acquired immune deficiency conditions, or autoimmune diseases
• People with a genetic condition called Li-Fraumeni Syndrome, or a known inherited fault in the retinoblastoma gene or its related biological pathway
• People who have received a live or live-attenuated vaccine within 30 days before the study drug is given
• People whose disease has spread to multiple locations, the lining of the brain and spinal cord, or the cerebrospinal fluid

Important: Always verify eligibility with the trial site directly before applying.