ONC206 for children with diffuse midline gliomas and other recurrent malignant brain tumors

Phase 0/1 trial of ONC206 - a novel imipridone for children with diffuse midline gliomas and recurrent malignant brain tumors

NIH-funded research University of California, San Francisco · NIH-11410956

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 typeR01 grant
Study typeNIH-funded research
Funding institutionUniversity of California, San Francisco NIH-funded
Lab location1 site (San Francisco, United States)
Project IDNIH-11410956 on NIH RePORTER

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.

Who could benefit from this research

Good fit: 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.

Not a fit: 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.

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 performed: 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

San Francisco, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.