Experimental combo targets rare childhood brain cancer

NCT ID NCT02774421

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

Summary

This pilot study tests whether a drug called trastuzumab, given with an immune booster (GM-CSF), can safely reach and treat recurrent ependymoma in children. Ten children with this rare brain tumor will receive the drugs to see if the treatment is safe and can be detected in the tumor. The goal is to gather early data for future, larger studies.

What this could mean

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

Active substance
trastuzumab (a cancer drug) and GM-CSF (an immune booster)
What this could lead to
If it works, this could point toward a new treatment option for children with recurrent ependymoma, a rare brain tumor.
What could go wrong
This is a very early, small pilot study with only 10 participants. It is designed mainly to check safety and whether the drug reaches the tumor, not to prove effectiveness. The treatment may not work or could cause side effects.

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

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States