A Study of Mirdametinib in People With Central Nervous System Tumors
Phase 1/2 Trial of Mirdametinib in Patients With MAPK Pathway Mutant Central Nervous System Tumors
1 other identifier
interventional
26
1 country
7
Brief Summary
The purpose of this study to find out whether mirdametinib is a safe and effective treatment for Central Nervous System/CNS tumors (glioma and neurohistiocytosis).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
Started Apr 2026
Typical duration for phase_1
7 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
April 10, 2026
CompletedFirst Submitted
Initial submission to the registry
April 13, 2026
CompletedFirst Posted
Study publicly available on registry
April 20, 2026
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 10, 2029
ExpectedStudy Completion
Last participant's last visit for all outcomes
October 10, 2029
April 27, 2026
April 1, 2026
3.5 years
April 13, 2026
April 22, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Best overall neurologic response rate
To determine the best overall neurologic response rate
1 year
Study Arms (3)
Cohort A: Mirdametinib
EXPERIMENTAL11 participants with refractory neurohistiocytosis will be treated with mirdametinib in continuous treatment cycles
Cohort B: Perioperative mirdametinib
EXPERIMENTAL15 participants with recurrent NF1-mutant glioma will be randomized in a 2:1 ratio to receive either perioperative mirdametinib (for 5 days) or no drug before standard of care surgery. All 15 participants will be treated with mirdametinib twice daily after surgery, continuously until clinical or radiographic progression of disease
Cohort B: No perioperative mirdametinib
NO INTERVENTION15 participants with recurrent NF1-mutant glioma will be randomized in a 2:1 ratio to receive either perioperative mirdametinib (for 5 days) or no drug before standard of care surgery. All 15 participants will be treated with mirdametinib twice daily after surgery, continuously until clinical or radiographic progression of disease
Interventions
Mirdametinib is a highly selective and potent, non-ATP-competitive oral inhibitor of MEK1 and MEK2 kinases
Eligibility Criteria
You may qualify if:
- Demographic Characteristics
- a. Be \> 18 years of age
- General Criteria
- Have Karnofsky Performance Status (KPS) of ≥ 70% or ECOG Performance Status of ≤ 2
- Is able to understand and provide written informed consent for the trial prior to any study-specific procedures and is willing to comply with scheduled visits, treatment plans, procedures and laboratory tests. A legally authorized representative may consent on behalf of a subject who is otherwise unable to provide informed consent, if acceptable to and approved by the institutional review board.
- Medical and Therapeutic Criteria
- Have adequate bone marrow function, as determined by:
- Absolute neutrophil count (ANC) \>1,500/mm3
- Platelet count \>100,000 mm³
- Hemoglobin \>9.0 mg/dL
- 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
- Have adequate renal function, as determined by:
- o Creatinine clearance (CrCL) of ≥50 mL/min by the Cockcroft-Gault formula
- +26 more criteria
You may not qualify if:
- Subjects who meet any of the following criteria will not be enrolled in the study:
- General criteria
- Is pregnant or nursing
- Is participating in another interventional study at the same time; participation in non-therapeutic registries is allowed
- Medical and Therapeutic criteria
- Receipt of tumor directed therapy (chemotherapy, targeted therapy, biologic, investigational) within 28 days or 5 half-lives (whichever is shorter) before the first dose of mirdametinib.
- Concomitant use of medications that strongly induce CYP3A
- History of allergic reaction to the study agent(s), compounds of similar chemical or biologic composition to the study agent (s) (or any of its excipients).
- Evidence of serious active infections. Patients are allowed to enroll if they have been fever free for at least 48 hours
- Uncontrolled or severe intercurrent medical condition
- Have significant active cardiac disease within 6 months before the start of treatment, including New York Heart Association Class III or IV congestive heart failure, atrial fibrillation, myocardial infarction, unstable angina and/or stroke.
- Have significant active ophthalmologic disease within 6 months before the start of treatment, including central retinal vein occlusion
- Has a history of or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the study, interfere with the subject's participation for the full duration of the study, or is not in the best interest of the subject to participate, in the opinion of the treating Investigator
- Cohort A only: Patients with documented driver mutations outside of the MAPK pathway are not eligible for this study. These are uncommon and include mutations in ALK, RET, CSF1R, NTRK, and other kinases20 .
- On Study Guidelines:
- +4 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (7)
Memorial Sloan Kettering Basking Ridge (Limited Protocol Activities)
Basking Ridge, New Jersey, 07920, United States
Memorial Sloan Kettering Monmouth (Limited protocol activities)
Middletown, New Jersey, 07748, United States
Memorial Sloan Kettering Bergen (Limited Protocol Activities)
Montvale, New Jersey, 07645, United States
Memorial Sloan Kettering Suffolk - Commack (Limited Protocol Activities)
Commack, New York, 11725, United States
Memorial Sloan Kettering Westchester (Limited Protocol Activities)
Harrison, New York, 10604, United States
Memorial Sloan Kettering Cancer Center (All Protocol Activities)
New York, New York, 10065, United States
Memorial Sloan Kettering Nassau (Limited Protocol Activities)
Rockville Centre, New York, 11570, United States
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Anna Piotrowski, MD
Memorial Sloan Kettering Cancer Center
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 13, 2026
First Posted
April 20, 2026
Study Start
April 10, 2026
Primary Completion (Estimated)
October 10, 2029
Study Completion (Estimated)
October 10, 2029
Last Updated
April 27, 2026
Record last verified: 2026-04
Data Sharing
- IPD Sharing
- Will share
Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made following one year after publication and for up to 36 months later. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.