Targeted drug aims to shrink recurrent brain tumors by blocking key growth enzyme

NCT ID NCT03598244

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

Summary

This trial tests the drug savolitinib in children and adults whose brain tumors (medulloblastoma, high-grade glioma, or DIPG) have come back or not responded to treatment. The drug works by blocking an enzyme that some tumor cells need to grow. The study aims to find the safest dose and see if the drug can shrink tumors or slow their growth.

What this could mean

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

Active substance

Savolitinib

What this could lead to

If it works, this could offer a new treatment option for people with certain hard-to-treat brain tumors that have come back or not responded to standard therapy.

What could go wrong

This is an early-phase trial with a small number of participants, so the drug may not work as hoped. Side effects could be significant, and the benefit may be limited to a small subset of patients.

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 diffuse intrinsic pontine glioma glioma malignant glioma medulloblastoma primary brain neoplasm

As listed by the trial registrant

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

Contacts and locations

Locations

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania, 15224, 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

  • Hospital for Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • Lucile Packard Children's Hospital Stanford University

    Palo Alto, California, 94304, United States

  • Lurie Children's Hospital-Chicago

    Chicago, Illinois, 60611, United States

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Saint Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

  • Texas Children's Hospital

    Houston, Texas, 77030, United States