ONC206 for pediatric brain tumors: a first-in-children trial
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-11413761 · Data updated 2026-11-12
A first-in-children trial will test the safety and early effectiveness of ONC206, a pill that can reach brain tumors, in kids with diffuse midline gliomas and other recurrent brain cancers.
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 | 2026 |
| Award (this year) | $499,535 |
| NIH project number | 5R01CA266596-05 |
| Project ID | NIH-11413761 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children or young adults with malignant brain tumors, including diffuse midline gliomas, for whom standard treatments are limited or ineffective.
✕Less likely to help
Some patients may not benefit from the drug and could experience treatment-related side effects.
What this research studies
This trial will enroll children and young adults to receive ONC206, look at how the drug behaves in the body, and monitor for side effects. Researchers will determine the highest safe dose (RP2D) and explore potential early signs of benefit, both alone and with radiation in some groups. The study builds on lab data showing ONC206 may trigger cancer cell death via mitochondrial pathways and ClpP activation. Participants will undergo standard medical care alongside careful safety and biomarker assessments to guide future research. The goal is to lay groundwork for a potentially more effective treatment option for pediatric brain tumors.
Why it matters
Potential benefit: If successful, participants may experience a safer, more effective therapy option and pave the way for future pediatric trials.
How similar studies have gone: Preclinical data suggest ONC206 can induce tumor cell death and improve survival in models, supporting early testing 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
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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.