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.

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.

astrocytoma (excluding glioblastoma) central neurocytoma gangliocytoma ganglioglioma low grade glioma neuroepithelial neoplasm oligodendroglioma

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

  • St. Jude Children's Research Hospital

    RECRUITING

    Memphis, Tennessee, 38105, United States

    Contact

    Contact

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