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 Read more

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.

diffuse intrinsic pontine glioma glioma malignant glioma

As listed by the trial registrant

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

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