MEKTOVI® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma

Adamantinomatous Craniopharyngioma (ACP) is a highly debilitating pediatric brain tumor that lacks medical anti-tumor therapies. Current therapy, which depends largely on surgery and radiation, is associated with poor quality of life and becomes more challenging and risky in the setting of recurrent disease. Recent discoveries regarding the biological characteristics of ACP indicate that available agents, including Mitogen-activated protein kinase/extracellular signal-regulated kinase (MAPK/ERK) pathway inhibitors may have efficacy in the control of ACP. Binimetinib is one such agent. In this study, up to 38 patients will receive oral binimetinib at the recommended phase 2 pediatric dose (RP2D) of 32 mg/m2/dose PO every 12 hours for 4 weeks which represents one cycle. Cycles will last 28 days and treatment may continue for up to two years (26 cycles). It will be a multi-center Phase 2 trial with two strata for patients aged \>1 year and \<39 years with measurable ACP who may have been previously treated with radiation (Stratum 1, 19 patients) or without radiation (Stratum 2, 19 patients, NOT CURRENTLY ENROLLING).