Hope for kids with rare brain cancer: new drug combo trial launches

NCT ID NCT07447076

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a combination of two drugs, gemcitabine and paxalisib, in children, teens, and young adults whose atypical teratoid rhabdoid tumor (ATRT) has come back or gotten worse. The goal is to find a safe dose and see if the drugs can control the tumor. About 29 participants will receive the drugs and undergo regular scans and blood tests.

What this could mean

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

Active substance

gemcitabine and paxalisib

What this could lead to

If this works, it could offer a new treatment option to control recurrent ATRT brain tumors in children and young adults.

What could go wrong

This is a small, early-phase trial with only 29 participants, so results may not apply to everyone. The drug combo may cause side effects or fail to shrink tumors.

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.

atypical teratoid rhabdoid tumor rhabdoid tumor

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • University of California, San Francisco

    NOT_YET_RECRUITING

    San Francisco, California, 94143, United States

  • University of California, San Francisco

    RECRUITING

    San Francisco, California, 94143, United States

    Contact Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact