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

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.

A Study of Mirdametinib in People With Central Nervous System Tumors