ONC206 for children with diffuse midline gliomas and other recurrent malignant brain tumors
Official title: Phase 0/1 trial of ONC206 - a novel imipridone for children with diffuse midline gliomas and recurrent malignant brain tumors
Funded at University of California, San Francisco · NIH-11410956 · Data updated 2026-11-12
This trial tests a new oral drug called ONC206 that can reach the brain in children with diffuse midline gliomas and other recurrent malignant brain tumors to check safety and early signs it can fight tumors.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project start | September 7, 2022 |
| Project end | August 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $45,321 |
| NIH project number | 3R01CA266596-04S1 |
| Project ID | NIH-11410956 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children with diffuse midline gliomas or other recurrent malignant pediatric brain tumors who meet the trial's medical and safety criteria are the intended candidates for this trial.
✕Less likely to help
Adults outside the pediatric focus, people with cancers other than the specified brain tumors, or those who are medically ineligible or too frail for the treatment are unlikely to receive benefit from joining this trial.
What this research studies
If you join, doctors will give you an oral drug called ONC206 and follow you closely for side effects and how the tumor responds. The study starts with very low doses and rises carefully to find the right dose for children, and will measure drug levels in the blood. Some groups will receive ONC206 alone and others will get it together with radiation to see if the combination is safe and helpful. Doctors will also collect tumor and blood samples to learn how the drug works against tumor cells.
Why it matters
Potential benefit: If successful, ONC206 could become a new oral treatment that reaches brain tumors and improves tumor control or survival for children with these aggressive tumors.
How similar studies have gone: Related drugs in the imipridone family (for example ONC201) have shown early promise in adults and strong preclinical results support ONC206, but ONC206 is just beginning testing in children.
Where this research is happening
-
University of California, San Francisco
San Francisco, United States Active
Researchers
- Principal investigator: Mueller, Sabine — University of California, San Francisco
- Study coordinator: Mueller, Sabine
Related research
Other NIH-funded projects on the same topics.
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ONC206 for children with brain tumors: a first-in-children trial
A first-in-children trial will test the safety and early signs of effectiveness of ONC206, taken by mouth, in kids with malignant brain tumors like diffuse midline gliomas.
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ONC206 for children with malignant brain tumors
A first-in-children trial tests the safety and early effectiveness of a pill that can cross the brain barrier to treat pediatric brain tumors.
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ONC206 for children with dangerous brain tumors
A first-in-children study tests the safety and early effectiveness of an oral drug, ONC206, for kids with high-risk brain tumors that have spread or recurred.
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Oral bacteria and brain metastasis: how the mouth may influence brain tumors
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Bioengineered brain barrier chip to improve drug screening for brain tumors
A small, patient-friendly brain barrier chip is being developed to test drugs for glioblastoma more accurately before they reach clinical trials.
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Understanding how epigenetic changes drive pediatric brain tumor recurrence and how reversing them could help
A pediatric brain cancer research project is exploring how an epigenetic change (H3K27M) and tumor environment contribute to tumor coming back, using a reversible mouse model to study potential ways to prevent recurrence.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.