New hope for kids with deadly brain cancer?

NCT ID NCT03605550

Ongoing Disease control Sponsor: Nationwide Children's Hospital Source: ClinicalTrials.gov ↗

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.

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 glioma malignant glioma

As listed by the trial registrant

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

DIFFUSE INTRINSIC PONTINE GLIOMA HIGH GRADE GLIOMA

Contacts and locations

Locations

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

Chicago, Illinois, 60611, United States
Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States
Duke University Medical Center

Durham, North Carolina, 27710, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Seattle Children's Hospital

Seattle, Washington, 98105, United States
Texas Children's Hospital

Houston, Texas, 77030, United States
The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

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