CONNECT2108
Phase 2 Study of the MEK inhibitor MEKTOVI® (binimetinib) for the treatment of Pediatric Adamantinomatous Craniopharyngioma
Brain and other Central Nervous System (CNS) Tumours
Adamantinomatous Craniopharyngioma (ACP) is a highly debilitating paediatric brain tumour that has very limited treatment options. Current therapy, which depends largely on surgery and radiation, is associated with poor quality of life. If the disease recurs, further treatment is more challenging and risky. 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 be effective in the control of ACP. Binimetinib is one such agent.
In this study, up to 38 patients will receive oral medication binimetinib for up to two years. The study will investigate if binimetinib will be safe and effective at inducing tumour response in children with residual ACP.
Disease Stage: Progressive/Recurrent
Patient Age Range: Patients aged >1 year and <25 years
Trial sponsors:
- National – ANZCHOG
- International – COllaborative Network for Neuro-oncology Clinical Trials (CONNECT)
Sites
Perth Children’s Hospital
Queensland Children’s Hospital
Sydney Children’s Hospital
