Experimental antibody takes on childhood brain cancers

NCT ID NCT03389802

First seen Nov 01, 2025 · Last updated Jun 24, 2026 · Updated 40 times

Summary

This early-stage trial is testing an experimental drug called APX005M in 32 children with brain tumors that have come back or are hard to treat, including a type of brain stem glioma. The drug works by activating the immune system to attack cancer cells. The main goals are to find the safest dose and see what side effects occur.

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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.

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Contacts and locations

Locations

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Children Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania, 15224, United States

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30322, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90026, United States

  • Childrens National Medical Center

    Washington D.C., District of Columbia, 20010-2970, United States

  • Cincinnati Children Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Lucile Packard Children Hospital Stanford University

    Palo Alto, California, 94304, United States

  • Lurie Childrens Hospital-Chicago

    Chicago, Illinois, 60614, United States

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065, United States

  • St. Jude Children Research Hospital

    Memphis, Tennessee, 38105, United States

What this could mean

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

Active substance

APX005M (an experimental antibody that activates the immune system)

What this could lead to

If successful, this could point toward a new treatment option for children with hard-to-treat brain tumors.

What could go wrong

This is a very early Phase 1 trial with only 32 participants, so it is primarily testing safety and dosing. The drug may not shrink tumors or improve survival, and side effects like cytokine release syndrome are possible.

Conditions

The condition(s) this trial relates to.

ependymoma glioblastoma medulloblastoma

As listed by the trial registrant

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