Australian & New Zealand Childrens Haematology/Oncology Group

Clinical Trials

CONNECT2108: Phase 2 Study of the MEK inhibitor MEKTOVI® (binimetinib) for the treatment of Progressive/Recurrent Pediatric Adamantinomatous Craniopharyngioma

Adamantinomatous Craniopharyngioma

Status: Open | NSW , QLD , WA

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)

ANZCHOG acknowledges the valuable support of CONNECT1905 by the Robert Connor Dawes Foundation through the Robert Connor Dawes Clinical Trial Program and Carrie’s Beanies 4 Brain Cancer Foundation through the Carries Beanies 4 Brain Cancer Clinical Trial Program.