Could a cancer drug shrink Children's brain tumors?

NCT ID NCT03363217

Ongoing Disease control Sponsor: St. Justine's Hospital Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 trial tests the drug trametinib in children and young adults (ages 1 month to 25 years) with low-grade glioma or plexiform neurofibroma that has not responded to prior treatment. Participants take a daily oral dose for up to 18 cycles. The study aims to see if the drug can shrink tumors and slow disease progression.

What this could mean

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

Active substance: Trametinib (Mekinist)
What this could lead to: If successful, this could provide a new treatment option for children with certain brain tumors or nerve tumors that have not responded to standard therapy.
What could go wrong: This is a mid-stage trial with a moderate number of participants. The drug may not shrink tumors effectively, and side effects like rash, diarrhea, or fatigue are possible.

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.

brain cancer brain glioma glioma low grade glioma neurofibromatosis type 1 plexiform neurofibroma

As listed by the trial registrant

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

CENTRAL NERVOUS SYSTEM GLIOMA LOW GRADE GLIOMA PLEXIFORM NEUROFIBROMA

Contacts and locations

Locations

Alberta Children's Hospital

Calgary, Alberta, T3B 6A8, Canada
CHU Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada
CHU de Québec

Québec, Quebec, G1V 4G2, Canada
Children and Women's Health Centre of British Colombia

Vancouver, British Columbia, Canada
IWK Health Centre

Halifax, Nova Scotia, Canada
Montreal Children's Hospital

Montreal, Quebec, H4A 3J1, Canada
The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

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