Study of Mirdametinib for Central Nervous System Tumors
This study is testing mirdametinib, an oral medication, to see if it is a safe and effective treatment for central nervous system (CNS) tumors, specifically glioma. Researchers are looking for about 26 adult participants (18 years or older) who have a good general health status. The main goal is to measure how well the tumor responds to mirdametinib after one year. The current recruitment status for this study is unclear.
- Study design
- This is an interventional study, meaning participants will receive a specific treatment. It plans to enroll 26 participants.
- What's involved
- You would need to understand and sign a consent form and be willing to follow scheduled visits and treatment plans.
- Compensation
- Not stated in the trial record.
- Follow-up
- The main measure of success is taken at 1 year, which suggests a follow-up period of at least that long.
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NCT07539441
A Study of Mirdametinib in People With Central Nervous System Tumors
Recruiting
PHASE1Ages 18+InterventionalTreatmentMemorial Sloan Kettering Cancer CenterInvestigator-initiated
~26 participants
Updated 2026-05-13 on ClinicalTrials.gov
What's tested:Mirdametinib
At a glance
Recruiting sites
7 of 7 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Best overall neurologic response rate
Measured over 1 year
Conditions
Where it's being run
7 sites across 2 statesNew York4
New Jersey3
Study leadership
- Anna Piotrowski, MD · PRINCIPAL_INVESTIGATOR · Memorial Sloan Kettering Cancer Center
Who to contact
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Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
Absolute neutrophil count (ANC) \>1,500/mm3
Platelet count \>100,000 mm³
Hemoglobin \>9.0 mg/dL 2. Have adequate hepatic function, as determined by:
Total bilirubin ≤1.5 x ULN if baseline was normal or ≤1.5 x baseline if baseline was abnormal. Patients with previously documented Gilbert's Syndrome may have total bilirubin ≤3 x ULN.
AST and ALT ≤3.0 x ULN if baseline was normal or ≤3.0 x baseline if baseline was abnormal 3. Have adequate renal function, as determined by:
Left ventricular ejection fraction \>50% as assessed by multi-gated acquisition or ultrasound or echocardiography and
Corrected QT interval (QTc) \<480 ms according to the Fridericia method (QTcF) 5. Women of childbearing potential must have a negative serum pregnancy test before the start of therapy. 6. Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial 7. Patients previously treated with radiotherapy must have recovered from acute toxicities associated with such treatment. Toxicities of investigational therapies should have recovered to grade 1 or less before start of the trial medication.
Measurable disease according to modified PERCIST (mPERCIST) involving neurologic structures, i.e. meninges, brain or spinal cord parenchyma, or ocular structures (see sections below for these criteria) OR
Measurable disease according to RECIST 1.1 involving neurologic structures OR
Any of the below neurologic deficits referable to neurohistiocytosis amenable to longitudinal quantified assessment. The following are the allowable deficits, deemed referable to disease, with their corresponding assessments and minimum required abnormalities:
Dysarthria as defined Speech Intelligibility of B or worse as measured by the Frenchay Dysarthria Scale
Ataxia as defined by a score of 3 or higher on the Scale for assessment and rating of ataxia (SARA)
Diplopia as defined by Prism Diopter of 5 or higher
Loss of visual acuity defined as best corrected visual acuity (BCVA) 20/40 (0.3 LogMAR) or worse in either eye
Diminished visual field, defined as 20% or higher deficit in Humphrey Visual Field 24-2 pattern deviation.
Exclusion
Cohorts A and B: Patients with cytopenias, renal impairment or hepatic impairment deemed the direct result of disease and therefore amenable to improvement with mirdametinib treatment may be enrolled at the discretion of the treating investigator
Cohort A: There is no prior therapy requirement given the poor CNS efficacy of standard therapies, morbidity of neurohistiocytosis, and the available data about safety and efficacy in nine patients treated with mirdametinib.
The effects of mirdametinib on the developing human fetus are unknown. For this reason, female participants of child-bearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) during study therapy and for 6 weeks following the completion of study therapy. Male participants with female partners of reproductive potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) during study therapy and for 3 months following the completion of study therapy. Should a participant become pregnant or suspect pregnancy while participating in this study, they should inform their treating physician immediately.
What this trial measures
- Best overall neurologic response rate1 year
To determine the best overall neurologic response rate