Promising drug for recurrent brain tumor in kids falls short in early trial
NCT ID NCT03904862
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested an oral drug called CX-4945 (silmitasertib) in 21 children with recurrent SHH medulloblastoma, a rare brain tumor. The trial aimed to find the best dose and see if the drug could shrink tumors. However, the study was stopped early, so we don't have clear answers on whether it works.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
CX-4945 (silmitasertib sodium), an oral drug
What this could lead to
If successful, this could point toward a new treatment option for children with recurrent SHH medulloblastoma, a rare brain tumor.
What could go wrong
The trial was terminated early with only 21 participants, so results are limited. It is unclear if the drug is effective or safe enough for broader use.
Disclaimer
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Baylor College of Medicine
Houston, Texas, 77030, United States
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Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
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Children's Hospital of Los Angeles
Los Angeles, California, 90026, United States
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Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15201, 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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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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St. Jude Children Research Hospital
Memphis, Tennessee, 38105, United States
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Stanford University and Lucile Packard Children's Hospital
Palo Alto, California, 94304, United States
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University of Florida
Gainesville, Florida, 32608, United States