ONC206 — an oral drug for children with diffuse midline glioma and recurrent malignant brain tumors
Phase 0/1 trial of ONC206 - a novel imipridone for children with diffuse midline gliomas and recurrent malignant brain tumors
This work will see if the oral drug ONC206 is safe and shows early signs of benefit for children with diffuse midline glioma and other recurrent malignant brain 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 ID | NIH-11410955 on NIH RePORTER |
What this research studies
If you join, you would receive oral ONC206 in carefully monitored dose groups to find a safe dose and learn how the drug behaves in the body. The trial includes four cohorts testing ONC206 alone and in combination with radiation for newly diagnosed or re-irradiated diffuse midline glioma and other recurrent malignant pediatric brain tumors. Doctors will collect blood and tumor-related markers to measure drug effects on the mitochondrial target ClpP, monitor side effects, and look for early signs of tumor response. The main goals are to define a recommended phase 2 dose, characterize safety and pharmacokinetics, and gather preliminary evidence of benefit.
Who could benefit from this research
Good fit: Children with diffuse midline glioma (including H3K27M-mutant tumors) or other recurrent malignant pediatric brain tumors who meet the study's medical and safety criteria and can attend visits at the study site.
Not a fit: Patients without these tumor types, those who do not meet safety or organ-function requirements, or those unable to travel to the study location are unlikely to benefit from participation.
Why it matters
Potential benefit: If successful, ONC206 could provide a new treatment option that crosses the blood–brain barrier and may shrink or slow tumors in children with these aggressive brain cancers.
How similar studies have performed: Related imipridone drugs such as ONC201 have shown early signals of activity in some brain tumors, but ONC206 is newer and its pediatric safety and effectiveness are not yet established.
Where this research is happening
San Francisco, United States
- University of California, San Francisco — San Francisco, United States (Active)
Researchers
- Principal investigator: Mueller, Sabine — University of California, San Francisco
- Study coordinator: Mueller, Sabine
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 below.