ONC206 treatment 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
An oral drug called ONC206 that reaches the brain is being given to children with diffuse midline gliomas and other recurrent malignant brain tumors to check safety and possible 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 ID | NIH-11196063 on NIH RePORTER |
What this research studies
This is a Phase 0/1 dose-finding effort where children with diffuse midline glioma (DMG) or other recurrent malignant pediatric brain tumors receive oral ONC206. Patients will be enrolled into defined cohorts to determine safe dosing, how the drug is processed in the body (pharmacokinetics), and early signs of tumor response. The study includes testing ONC206 alone and combined with radiation for some groups, with regular visits for scans, blood tests, and safety monitoring. Tumor and blood samples will be used to measure biological markers, including the drug target ClpP, to help understand who might benefit.
Who could benefit from this research
Good fit: Children and adolescents with diffuse midline glioma or other recurrent malignant pediatric brain tumors who meet the study's health and eligibility requirements are ideal candidates.
Not a fit: Patients with non-brain cancers, those who are medically too fragile for experimental therapy, or whose tumors lack the drug target may not receive benefit from this treatment.
Why it matters
Potential benefit: If successful, ONC206 could become a new oral therapy that reaches the brain and may slow tumor growth or improve outcomes for children with DMG and other recurrent malignant brain tumors.
How similar studies have performed: Related imipridone drugs have shown early activity in lab studies and some adult trials, and ONC206 has promising lab and animal data, but its safety and benefit in children are not yet proven.
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.