New hope for kids with deadly brain cancer?
NCT ID NCT03605550
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial is testing the safety of an experimental drug called PTC596 (Unesbulin) when given alongside radiation therapy to children and young adults newly diagnosed with diffuse intrinsic pontine glioma (DIPG) or high-grade glioma. The study aims to find the safest dose and see how the drug behaves in the body and tumor. It involves 64 participants aged 1 to 21.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
PTC596 (Unesbulin) tablets
What this could lead to
If it works, this could point toward a new treatment option for children with these aggressive brain tumors.
What could go wrong
This is an early phase 1 trial focused on safety and dosing, not yet testing effectiveness. The drug may cause side effects or fail to improve outcomes.
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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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Dana-Farber Cancer Institute
Boston, Massachusetts, 02215, United States
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Duke University Medical Center
Durham, North Carolina, 27710, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States