Study on Mirdametinib for Children, Teens, and Young Adults with Low-Grade Brain Tumors

Sponsor: St. Jude Children's Research Hospital

Sponsor score: 60

ClinicalTrials ID:NCT04923126

Provider View

This clinical trial is testing a new drug, mirdametinib, to see if it is safe and effective for treating low-grade brain tumors in children, adolescents, and young adults. The study will explore how well the drug works, its side effects, and how it affects patients' quality of life.

Patient Parameters

Program Overview

This study is investigating the use of mirdametinib, a drug that can enter the brain, to treat low-grade gliomas, a type of brain tumor, in young patients. The trial is divided into two phases. Phase 1 will focus on finding the safest dose and understanding how the drug behaves in the body. Phase 2 will evaluate how effective the drug is in treating the tumors and will include patients with newly diagnosed, recurrent, or progressive tumors.

Description

The study is divided into two phases:

Phase 1:

  • The main goal is to find the safest dose of mirdametinib that can be given to children and young adults with low-grade gliomas. Researchers will also study how the drug moves through the body.
  • Participants will take mirdametinib twice a day, and the dose will be adjusted to find the best balance between effectiveness and side effects.

Phase 2:

  • This phase will include three groups of patients:
    • Cohort 1: Patients with newly diagnosed low-grade gliomas who have not received any treatment other than surgery.
    • Cohort 2: Patients with tumors that have come back or are getting worse, who have not been treated with MEK inhibitors before.
    • Cohort 3: Patients with tumors that have come back or are getting worse, who have been treated with MEK inhibitors before.
  • The study will look at how well mirdametinib works in shrinking tumors and improving survival rates. It will also monitor side effects and how the treatment affects patients' daily lives.

Participants will take mirdametinib in 28-day cycles for up to 24 months, as long as the treatment is working and side effects are manageable. The study will use both tablet and capsule forms of the drug.

Eligibility Criteria

Inclusion Criteria

  • Children, teens, and young adults aged 2 to under 25 years with confirmed or suspected low-grade glioma.
  • Must have enough tumor tissue available for review.
  • Must be able to understand and agree to the study requirements, with consent from a guardian if underage.
  • Must have adequate organ function and meet specific health criteria.
  • For Phase 1, participants must have a body surface area within a certain range.
  • For Phase 2, there are no upper body surface area restrictions.
  • Must not have received prior MEK inhibitor treatment for certain cohorts.
  • Must have measurable or evaluable disease.
  • Must be able to take mirdametinib without interference from other medications.

Exclusion Criteria

  • Patients with high-grade tumors or certain genetic mutations.
  • Patients with eye conditions like central serous retinopathy or retinal vein occlusion.
  • Patients with significant medical conditions that could interfere with the study.
  • Patients who are pregnant or not using effective birth control.
  • Patients unable to follow the study guidelines.

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