New pill shows promise for teens with rare brain cancer

NCT ID NCT07286292

First seen Jun 25, 2026 · Last updated Aug 11, 2026 · Updated 3 times

Summary

This study tests a daily pill called vorasidenib in 10 teenagers (ages 12 to 17) with a slow-growing brain tumor (grade 2 glioma) that has a specific genetic mutation (IDH1 or IDH2). The goal is to see if the drug is safe, how it affects growth and development, and whether it can shrink or stabilize the tumor. Participants will take the drug for up to 5 years and have regular check-ups including blood tests, MRIs, and growth assessments.

What this could mean

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

Active substance
Vorasidenib (a targeted drug taken daily as a pill)
What this could lead to
If successful, this could offer a new treatment option for teenagers with certain slow-growing brain tumors, potentially delaying or avoiding the need for more aggressive therapies.
What could go wrong
This is a small, early-phase study with only 10 participants, so results may not apply to all patients. The drug may cause side effects or fail to control tumor growth.

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

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Kids Cancer Centre, Sydney Children's Hospital Randwick

    RECRUITING

    Sydney, 2031, Australia

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