Mirdametinib for Brain Tumor
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial investigates whether mirdametinib, a potential drug, is a safe and effective treatment for certain brain tumors, specifically those affecting the central nervous system, such as glioma and neurohistiocytosis. Researchers will divide participants into groups to test mirdametinib in different scenarios: one group with neurohistiocytosis will receive continuous treatment, while another group with glioma will receive the drug around the time of their surgery. The trial seeks individuals with glioma featuring specific genetic markers or confirmed neurohistiocytosis, particularly if previous treatments have been ineffective. Participants should have measurable tumors and symptoms of glioma or neurohistiocytosis.
As a Phase 1, Phase 2 trial, this research aims to understand how the treatment works in people and to measure its effectiveness in an initial, smaller group.
Do I have to stop taking my current medications?
The trial requires that you stop taking any tumor-directed therapy (like chemotherapy or targeted therapy) at least 28 days before starting the study drug. Also, you cannot use medications that strongly induce CYP3A. It's best to discuss your specific medications with the study team.
Is there any evidence suggesting that mirdametinib is likely to be safe for humans?
Research has shown that mirdametinib is usually well-tolerated. In one study, it helped 87% of children with low-grade glioma, indicating effectiveness for many. The FDA has approved mirdametinib for treating neurofibromatosis type 1 (NF1), confirming its safety for that condition. However, another study found that some patients discontinued the drug due to side effects, highlighting the need to monitor for negative effects. Participants in this trial will contribute to further evaluating its safety for brain tumors.12345
Why do researchers think this study treatment might be promising for brain tumors?
Most treatments for brain tumors, like surgery and chemotherapy, aim to remove or kill cancer cells but often struggle to fully prevent progression. Mirdametinib is unique because it targets a specific pathway involved in tumor growth, known as the MEK pathway, which is particularly active in certain types of brain tumors like NF1-mutant gliomas. Unlike standard treatments, mirdametinib can be administered both before and after surgery, potentially reducing tumor size and preventing recurrence more effectively. Researchers are excited because this targeted approach could lead to better outcomes with fewer side effects, offering new hope for patients with difficult-to-treat brain tumors.
What evidence suggests that mirdametinib might be an effective treatment for CNS tumors?
Studies have shown that mirdametinib yields promising results for brain tumors. Specifically, one study found that it helped 87% of children and young adults with low-grade glioma, a type of brain tumor. Research also indicates that mirdametinib significantly improves symptoms for patients with NF1-PN, another condition related to brain tumors. The treatment is generally well-tolerated, with many patients experiencing lasting benefits. In this trial, participants in Cohort A will receive mirdametinib for refractory neurohistiocytosis, while participants in Cohort B will be randomized to receive either perioperative mirdametinib or no drug before surgery, followed by continuous mirdametinib treatment. These findings suggest mirdametinib could effectively treat certain types of brain tumors.12367
Who Is on the Research Team?
Anna Piotrowski, MD
Principal Investigator
Memorial Sloan Kettering Cancer Center
Are You a Good Fit for This Trial?
This trial is for adults with certain brain or central nervous system tumors, including glioma (with specific genetic changes) and neurohistiocytosis. Participants must have good organ function, be able to care for themselves, not be pregnant or nursing, and meet other health criteria.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants in Cohort A receive continuous treatment cycles of mirdametinib. Participants in Cohort B receive perioperative mirdametinib for 5 days or no drug before surgery, followed by continuous mirdametinib treatment post-surgery.
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Mirdametinib
Trial Overview
The study is testing mirdametinib as a treatment for people with CNS tumors like glioma and neurohistiocytosis. It aims to see if the drug is safe and effective in these patients.
How Is the Trial Designed?
3
Treatment groups
Experimental Treatment
Active Control
15 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
11 participants with refractory neurohistiocytosis will be treated with mirdametinib in continuous treatment cycles
15 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
Find a Clinic Near You
Who Is Running the Clinical Trial?
Memorial Sloan Kettering Cancer Center
Lead Sponsor
SpringWorks Therapeutics, Inc.
Industry Sponsor
Citations
1.
onclive.com
onclive.com/view/mirdametinib-generates-responses-and-is-safe-in-pediatric-low-grade-gliomaMirdametinib Generates Responses and Is Safe in ...
Mirdametinib demonstrated an 87% overall response rate in pediatric low-grade glioma with MAPK pathway activation, with a recommended dose of ...
Study Details | NCT07521657 | Efficacy of Mirdametinib ...
This is a phase 2, open label, parallel multi-arm study of mirdametinib in combination with radiation in participants with recurrent ...
CTP-08. Efficacy and tolerability of mirdametinib (PD-0325901 ...
As of May 2025, data cutoff, 15(43%) remain on-therapy, 14(40%) completed therapy, and 6(17%) stopped prematurely (4 progressions, 2 toxicity).
Phase I/II evaluation of single agent mirdametinib (PD ... - PMC
Median time on therapy is 6.6 (2.2-7) months. No disease progressions have occurred. CONCLUSION: Thus far, mirdametinib is well-tolerated and clinically ...
5.
trial.medpath.com
trial.medpath.com/news/mirdametinib-achieves-87-response-rate-in-pediatric-low-grade-glioma-phase-1-2-trialMirdametinib Achieves 87% Response Rate in Pediatric Low ...
Mirdametinib demonstrated an 87% overall response rate in children and young adults with recurrent or progressive low-grade glioma with MAPK ...
Mirdametinib - NF1 Treatment
GOMEKLI (mirdametinib) is approved in the U.S. for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) ...
ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults ...
In a phase II trial in 19 adults and adolescents with inoperable NF1-PN, eight (42%) patients achieved a partial response to mirdametinib by ...
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