Testing a new treatment for children with aggressive brain tumors

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

['FUNDING_R01'] · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · NIH-10917106

This study is testing a new oral medication called ONC206 to see how safe and effective it is for children with malignant brain tumors, especially those with diffuse midline gliomas, by helping the body get rid of damaged brain cells.

Quick facts

Phase['FUNDING_R01']
Study typeNih_funding
SexAll
SponsorUNIVERSITY OF CALIFORNIA, SAN FRANCISCO (nih funded)
Locations1 site (SAN FRANCISCO, UNITED STATES)
Trial IDNIH-10917106 on ClinicalTrials.gov

What this research studies

This research investigates the safety and early effectiveness of a new oral medication called ONC206 for children suffering from malignant brain tumors, particularly diffuse midline gliomas. The study aims to understand how ONC206 works by activating a specific protein that helps eliminate damaged cells in the brain. Participants will receive this treatment and be monitored for any side effects and how well the medication works. The trial will include various groups of pediatric patients, including those newly diagnosed and those whose tumors have returned.

Who could benefit from this research

Good fit: Ideal candidates for this research are pediatric patients diagnosed with diffuse midline gliomas or recurrent malignant brain tumors.

Not a fit: Patients with non-malignant brain tumors or those who do not meet the specific criteria for this trial may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could provide a new treatment option that improves survival rates for children with aggressive brain tumors.

How similar studies have performed: Other research has shown promising results with similar approaches targeting mitochondrial pathways in cancer treatment, suggesting potential for success in this novel application.

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.

View on NIH RePORTER →

Last reviewed 2026-05-15 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.