New hope for kids with deadly brain tumors: combination drug trial launches
NCT ID NCT05009992
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital Los Angeles
RECRUITINGLos Angeles, California, 90027, United States
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Children's Hospital Minnesota
RECRUITINGMinneapolis, Minnesota, 55404, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
Contact Email: •••••@•••••
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Children's National Hospital
RECRUITINGWashington D.C., District of Columbia, 20010, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Dana-Farber Cancer Institute Harvard University
RECRUITINGBoston, Massachusetts, 02215-6024, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Duke University
RECRUITINGDurham, North Carolina, 27708, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hackensack Meridian Health
RECRUITINGHackensack, New Jersey, 07601, United States
Contact Email: •••••@•••••
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Indiana University Riley Children's Hospital
RECRUITINGIndianapolis, Indiana, 46202, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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John Hunter Children's Hospital
RECRUITINGNew Lambton Heights, New South Wales, 2305, Australia
Contact Email: •••••@•••••
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Johns Hopkins University
RECRUITINGBaltimore, Maryland, 21287, United States
Contact Email: •••••@•••••
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Monash Children's Hospital
RECRUITINGClayton, Victoria, 3168, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43205, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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New York University
RECRUITINGNew York, New York, 10016, United States
Contact Email: •••••@•••••
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Perth Children's Hospital
RECRUITINGNedlands, Washington, 6009, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Princess Maxima Center
ACTIVE_NOT_RECRUITINGUtrecht, Netherlands
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Queensland Children's Hospital
ACTIVE_NOT_RECRUITINGSouth Brisbane, Queensland, Australia
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Seattle Children's Hospital
RECRUITINGSeattle, Washington, 98101, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Shaare Zedek Medical Center
RECRUITINGJerusalem, 9103102, Israel
Contact Email: •••••@•••••
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Sheba Medical Center
RECRUITINGTel Litwinsky, Ramat Gan, Israel
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Starship Children's Hospital
RECRUITINGAuckland, New Zealand
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Sydney Children's Hospital
ACTIVE_NOT_RECRUITINGSydney, 2031, Australia
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The Children's Hospital at Westmead
ACTIVE_NOT_RECRUITINGWestmead, New South Wales, 2152, Australia
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The Royal Children's Hospital Melbourne
ACTIVE_NOT_RECRUITINGMelbourne, Victoria, 3052, Australia
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The University Children's Hospital in Zurich
RECRUITINGZurich, Switzerland
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Alabama at Birmingham
RECRUITINGBirmingham, Alabama, 35233, United States
Contact Email: •••••@•••••
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University of California, San Diego / Rady Children's Hospital, San Diego
RECRUITINGSan Diego, California, 92123, United States
Contact Email: •••••@•••••
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University of California, San Francisco
RECRUITINGSan Francisco, California, 94143, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Michigan
RECRUITINGAnn Arbor, Michigan, 48109, United States
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University of Utah
RECRUITINGSalt Lake City, Utah, 84101, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Washington University in St. Louis
RECRUITINGSt Louis, Missouri, 63110, United States
Contact Email: •••••@•••••
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Women and Children's Hospital
RECRUITINGAdelaide, Australia
Contact