New hope for kids with deadly brain cancer? drug ONC206 enters human trials

NCT ID NCT04732065

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

Summary

This early-phase trial tests a new drug called ONC206 in children and young adults with diffuse midline glioma or other recurrent brain tumors. The drug aims to kill cancer cells by causing stress in them, without harming healthy cells. Researchers are looking for the safest dose and checking for side effects, with up to 208 participants.

What this could mean

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

Active substance

ONC206

What this could lead to

If successful, this could point toward a new treatment option for children with aggressive brain tumors like diffuse midline glioma.

What could go wrong

This is an early phase 1 trial, so the drug may not work or could have side effects. It is small and focused on safety, not yet proving effectiveness.

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.

central nervous system cancer central nervous system neoplasm diffuse intrinsic pontine glioma diffuse midline glioma, H3 K27-altered ependymoma glioblastoma grade III glioma primary brain neoplasm spinal cord glioma

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Children's National Hospital

    RECRUITING

    Washington D.C., District of Columbia, 20010, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Emory University

    RECRUITING

    Atlanta, Georgia, 30322, United States

    Contact Email: •••••@•••••

  • Princess Máxima Center for Pediatric Oncology

    RECRUITING

    Utrecht, 3584, Netherlands

    Contact Phone: •••-•••-••••

    Contact Email: •••••@•••••

    Contact

  • The University Children's Hospital in Zurich

    RECRUITING

    Zurich, Canton of Zurich, 8032, Switzerland

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • University of California, San Francisco

    RECRUITING

    San Francisco, California, 94143, United States

    Contact Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • University of Michigan

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

    Contact

    Contact Email: •••••@•••••