New hope for kids with brain tumors: targeted drug mirdametinib enters trial
NCT ID NCT04923126
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This study tests a drug called mirdametinib in children, adolescents, and young adults with low-grade glioma, a type of brain tumor. The drug is designed to cross into the brain and block a protein that helps tumors grow. The trial has two phases: Phase 1 finds the safest dose, and Phase 2 checks how well the drug shrinks or stabilizes tumors. About 132 participants will take the drug by mouth twice daily in 28-day cycles.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Mirdametinib (PD-0325901), a brain-penetrant MEK inhibitor taken orally
- What this could lead to
- If successful, this could provide a new targeted treatment option for children and young adults with low-grade glioma, potentially shrinking tumors or slowing their growth.
- What could go wrong
- This is an early-phase trial (Phase 1/2) with a small number of participants, so the drug may not work as hoped or could cause unexpected side effects. Results may not apply to all patients.
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
-
St. Jude Children's Research Hospital
RECRUITINGMemphis, Tennessee, 38105, United States
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