What is mirdametinib studying for Central Nervous System tumors?

Mirdametinib is being studied to find out whether it is a safe and effective treatment for Central Nervous System tumors like glioma and neurohistiocytosis. The research aims to understand how this drug affects patients with specific genetic mutations in their tumors. Memorial Sloan Kettering Cancer Center is leading this clinical trial.

Who may be eligible for this Central Nervous System tumor clinical trial?

Adults 18 years or older with a diagnosis of glioma or neurohistiocytosis and a certain level of overall health may be eligible. Both men and women can participate if they meet performance status and organ function criteria and are willing to follow study requirements. Please review eligibility criteria for more details.

Is there any cost to participate in this Central Nervous System tumor clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel expenses may vary and are not guaranteed to be covered. Participants should plan accordingly for any travel or related costs.

Actively Recruiting

Phase 1

Phase 2

Age: 18Years +

All Genders

ID07539441

Phase 12 Study of Mirdametinib for Adults With MAPK Pathway Mutant Central Nervous System Tumors Including Glioma and Neurohistiocytosis

Led by Memorial Sloan Kettering Cancer Center · Updated on 2026-05-13

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating whether mirdametinib is a safe and effective treatment for Central Nervous System tumors, including glioma and neurohistiocytosis. This phase 12 trial focuses on patients with MAPK pathway mutant CNS tumors to better understand the drugs impact on these conditions. The study is sponsored by Memorial Sloan Kettering Cancer Center and aims to measure the best overall neurologic response rate within one year. Participants are divided into two main groups. Eleven patients with refractory neurohistiocytosis will receive continuous cycles of mirdametinib. Meanwhile, 30 participants with recurrent NF1-mutant glioma will be randomized in a 21 ratio to either receive perioperative mirdametinib for five days before surgery or no drug before surgery. After surgery, all glioma participants will take mirdametinib twice daily continuously until disease progression or clinical changes occur. During the study, participants will undergo regular evaluations including neurological assessments and monitoring of disease progression via imaging scans. Researchers will collect data on safety, tolerability, and treatment response. The study includes scheduled visits, laboratory tests, and performance status assessments. Participation will continue until disease progression or other criteria require stopping treatment, with ongoing monitoring throughout the trial period.

CONDITIONS

Brief Title

A Study of Mirdametinib in People With Central Nervous System Tumors

Who Can Participate

Age: 18Years +

All Genders

Eligibility Criteria

You may qualify if you...

Be older than 18 years of age
Have a Karnofsky Performance Status of 70% or higher, or an ECOG Performance Status of 2 or less
Able to understand and provide written informed consent, or have a legally authorized representative
Adequate bone marrow function: ANC greater than 1,500/mm3, platelet count over 100,000/mm3, hemoglobin above 9.0 mg/dL
Adequate liver function: total bilirubin within 1.5 times the upper limit of normal or baseline, AST and ALT within 3 times the upper limit of normal or baseline
Adequate kidney function: creatinine clearance of 50 mL/min or higher
Adequate heart function: left ventricular ejection fraction over 50%, corrected QT interval under 480 ms
Women of childbearing potential must have a negative pregnancy test before treatment
Patients with prior or concurrent malignancy allowed if it does not interfere with the study
Recovery from prior radiotherapy toxicities to grade 1 or less
For Neurohistiocytosis cohort: documented or likely MAPK pathway mutation, confirmed disease, and measurable neurologic disease
For Glioma cohort: somatic or germline NF1 mutation, confirmed glioma diagnosis, measurable MRI-evaluable disease, recurrent or progressive disease after prior treatment, surgery indicated but not urgent, expected survival over 3 months

You will not qualify if you...

Pregnant or nursing
Participating in another interventional study at the same time
Received tumor-directed therapy within 28 days before starting mirdametinib
Taking medications that strongly induce CYP3A
History of allergic reaction to mirdametinib or similar compounds
Serious active infections without at least 48 hours fever-free
Uncontrolled or severe medical conditions
Significant active heart disease within 6 months prior to treatment
Significant active eye disease within 6 months prior to treatment
Any condition or therapy that might interfere with study results or participant safety
For Neurohistiocytosis cohort: documented driver mutations outside the MAPK pathway
Female participants of childbearing potential must agree to use contraception during and after treatment; male participants with partners of reproductive potential must also use contraception during and after treatment

Research Team

Anna Piotrowski, MD

Eli Diamond, MD

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