ONC206 treatment for children with diffuse midline glioma 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-11410957 · Data updated 2026-11-12
An oral drug called ONC206 that can reach the brain is being given to children with diffuse midline gliomas and other recurrent malignant brain tumors to check safety and look for early signs of benefit.
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) | $9,000 |
| NIH project number | 3R01CA266596-04S3 |
| Project ID | NIH-11410957 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 eligibility criteria and can travel to the study site may be eligible.
✕Less likely to help
Patients for whom the tumor does not express the drug's target or who are medically unfit for the trial treatments may not receive benefit from participation.
What this research studies
If you join, doctors will give ONC206 by mouth and start with very low doses that are increased carefully to find a safe dose for children. The team will do blood tests to measure drug levels, regular scans to watch the tumor, and monitor for side effects. Some patients will get ONC206 alone and others will get it together with radiation depending on whether the tumor is newly diagnosed or recurrent. Researchers will also study tumor and blood markers linked to the drug's action on a mitochondrial protein called ClpP to see who might respond.
Why it matters
Potential benefit: If successful, ONC206 could offer a new treatment option that reaches the brain and may slow tumor growth or extend survival for children with these aggressive brain tumors.
How similar studies have gone: Related drugs in the imipridone class have shown early signals of activity in brain tumors, but ONC206 is a newer compound being tested in humans.
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
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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
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ONC206 for children with dangerous brain tumors
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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.