New hope for kids with deadly brain tumors: combination drug trial launches

NCT ID NCT05009992

Recruiting now Disease control Sponsor: University of California, San Francisco Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This phase 2 trial is testing whether combining the drug ONC201 with other treatments (like paxalisib or a cancer-killing virus called DNX-2401) can help children and young adults with diffuse midline gliomas, a rare and aggressive brain cancer. The study enrolls 360 participants at different stages of their disease, including after radiation or when the tumor progresses. The goal is to see if these combinations can slow tumor growth and improve survival.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: ONC201 (drug) combined with other drugs like paxalisib or the virus DNX-2401, plus radiation therapy
What this could lead to: If successful, this could lead to better treatment options that slow tumor growth and extend survival for children and young adults with these aggressive brain tumors.
What could go wrong: This is a phase 2 trial, so the combinations are still being tested for safety and effectiveness. The tumors are very hard to treat, and results may not lead to a lasting benefit or apply to all patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

diffuse intrinsic pontine glioma diffuse midline glioma, H3 K27M-mutant glioblastoma grade III glioma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DIFFUSE INTRINSIC PONTINE GLIOMA DIFFUSE MIDLINE GLIOMA, H3 K27M-MUTANT RECURRENT DIFFUSE INTRINSIC PONTINE GLIOMA RECURRENT DIFFUSE MIDLINE GLIOMA, H3 K27M-MUTANT RECURRENT WHO GRADE III GLIOMA WHO GRADE III GLIOMA

Contacts and locations

Locations

Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITING

Chicago, Illinois, 60611, United States
Children's Hospital Los Angeles
RECRUITING

Los Angeles, California, 90027, United States
Children's Hospital Minnesota
RECRUITING

Minneapolis, Minnesota, 55404, United States
Children's Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Children's National Hospital
RECRUITING

Washington D.C., District of Columbia, 20010, United States
Dana-Farber Cancer Institute Harvard University
RECRUITING

Boston, Massachusetts, 02215-6024, United States
Duke University
RECRUITING

Durham, North Carolina, 27708, United States
Hackensack Meridian Health
RECRUITING

Hackensack, New Jersey, 07601, United States
Indiana University Riley Children's Hospital
RECRUITING

Indianapolis, Indiana, 46202, United States
John Hunter Children's Hospital
RECRUITING

New Lambton Heights, New South Wales, 2305, Australia
Johns Hopkins University
RECRUITING

Baltimore, Maryland, 21287, United States
Monash Children's Hospital
RECRUITING

Clayton, Victoria, 3168, Australia
Nationwide Children's Hospital
RECRUITING

Columbus, Ohio, 43205, United States
New York University
RECRUITING

New York, New York, 10016, United States
Perth Children's Hospital
RECRUITING

Nedlands, Washington, 6009, Australia
Princess Maxima Center
ACTIVE_NOT_RECRUITING

Utrecht, Netherlands
Queensland Children's Hospital
ACTIVE_NOT_RECRUITING

South Brisbane, Queensland, Australia
Seattle Children's Hospital
RECRUITING

Seattle, Washington, 98101, United States
Shaare Zedek Medical Center
RECRUITING

Jerusalem, 9103102, Israel
Sheba Medical Center
RECRUITING

Tel Litwinsky, Ramat Gan, Israel
Starship Children's Hospital
RECRUITING

Auckland, New Zealand
Sydney Children's Hospital
ACTIVE_NOT_RECRUITING

Sydney, 2031, Australia
The Children's Hospital at Westmead
ACTIVE_NOT_RECRUITING

Westmead, New South Wales, 2152, Australia
The Royal Children's Hospital Melbourne
ACTIVE_NOT_RECRUITING

Melbourne, Victoria, 3052, Australia
The University Children's Hospital in Zurich
RECRUITING

Zurich, Switzerland
University of Alabama at Birmingham
RECRUITING

Birmingham, Alabama, 35233, United States
University of California, San Diego / Rady Children's Hospital, San Diego
RECRUITING

San Diego, California, 92123, United States
University of California, San Francisco
RECRUITING

San Francisco, California, 94143, United States
University of Michigan
RECRUITING

Ann Arbor, Michigan, 48109, United States
University of Utah
RECRUITING

Salt Lake City, Utah, 84101, United States
Washington University in St. Louis
RECRUITING

St Louis, Missouri, 63110, United States
Women and Children's Hospital
RECRUITING

Adelaide, Australia

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