Phase 2 Study of the MEK Inhibitor MEKTOVI® (Binimetinib) for the Treatment of Pediatric Adamantinomatous Craniopharyngioma
Nationwide Children's Hospital
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.
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…
Eligibility
- Age range
- 1–39 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Age: Patients must be ≥ 12 months and ≤ 39 years of age at the time of study enrollment. 2. Diagnosis: Patients with histologically-confirmed adamantinomatous craniopharyngioma (ACP) Histologic confirmation of ACP may be made on solid tumor or, if no solid tumor can be safely obtained, cyst fluid with classic ACP characteristics of thick, cholesterol-rich, greenish-brown liquid in the context of imaging features consistent with craniopharyngioma, including lobulated, cystic/solid mass with calcifications that originates in the sellar/suprasellar region. 3. Disease Statu…
Interventions
- DrugBinimetinib Oral Tablet [Mektovi]
Binimetinib oral continuous dosing 32 mg/m2 PO BID for 4 weeks
Locations (10)
- Children's Hospital ColoradoAurora, Colorado
- Children's National Medical CenterWashington D.C., District of Columbia
- Nicklaus Children's HospitalMiami, Florida
- Cincinnati Children's Hospital Medical CenterCincinnati, Ohio
- Nationwide Children's HospitalColumbus, Ohio
- Sydney Children's HospitalRandwick, New South Wales