NCT05286788

MEKTOVI® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma

Phase 2 Study of the MEK Inhibitor MEKTOVI® (Binimetinib) for the Treatment of Pediatric Adamantinomatous Craniopharyngioma

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 38 (estimated)
• Sponsor: Nationwide Children's Hospital · Academic / Other
• Sex: All
• Age: 1 Year – 39 Years
• Healthy volunteers: Not accepted

Summary

MEKTOVI (binimetinib) is an oral, highly selective reversible inhibitor of mitogen-activated extracellular signal regulated kinase 1 (MEK1) and MEK2. The biological activity of binimetinib that has been evaluated bith in vitro and in vivo in a wide variety of tumor types In this Phase II, the drug will be used to treat pediatric patients diagnosed with recurrent Adamantinomatous Craniopharyngioma including patients who have undergone surgery and/or radiation therapy.

Detailed description

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).

Conditions

• Adamantinous Craniopharyngioma
• Recurrent Adamantinomatous Craniopharyngioma

Interventions

Type	Name	Description
DRUG	Binimetinib Oral Tablet [Mektovi]	Binimetinib oral continuous dosing 32 mg/m2 PO BID for 4 weeks

Timeline

• Start date: 2023-04-10
• Primary completion: 2027-04-10
• Completion: 2027-04-10
• First posted: 2022-03-18
• Last updated: 2026-04-08

Locations

10 sites across 3 countries: United States, Australia, Canada

Regulatory

• FDA-regulated drug study