Promising drug for recurrent brain tumor in kids falls short in early trial

NCT ID NCT03904862

Terminated Disease control Sponsor: Pediatric Brain Tumor Consortium Source: ClinicalTrials.gov ↗

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.

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.

childhood medulloblastoma medulloblastoma medulloblastoma SHH activated microphthalmia, isolated, with coloboma 5

As listed by the trial registrant

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

MEDULLOBLASTOMA MEDULLOBLASTOMA RECURRENT MEDULLOBLASTOMA, CHILDHOOD

Contacts and locations

Locations

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States
Baylor College of Medicine

Houston, Texas, 77030, United States
Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States
Children's Hospital of Los Angeles

Los Angeles, California, 90026, United States
Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15201, 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
Memorial Sloan-Kettering Cancer Center

New York, New York, 10065, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
St. Jude Children Research Hospital

Memphis, Tennessee, 38105, United States
Stanford University and Lucile Packard Children's Hospital

Palo Alto, California, 94304, United States
University of Florida

Gainesville, Florida, 32608, United States

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