Targeted drug aims to shrink recurrent brain tumors by blocking key growth enzyme
NCT ID NCT03598244
First seen Jun 27, 2026 · Last updated Jul 31, 2026 · Updated 2 times
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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
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Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
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Lucile Packard Children's Hospital Stanford University
Palo Alto, California, 94304, United States
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Lurie Children's Hospital-Chicago
Chicago, Illinois, 60611, United States
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Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Saint Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Targeted radiation zaps childhood brain tumors in early trial
- Parental distress may undermine treatment in children with recurrent brain tumors
- Experimental vaccine aims to rally immune system against recurrent brain cancer
- Immunotherapy drug nivolumab tested in young brain cancer patients
- Targeted drug shows promise for kids with tough cancers
- New hope for kids with Hard-to-Treat cancers: targeted drug shows promise