Australian & New Zealand Childrens Haematology/Oncology Group

Clinical Trials

PNOC029: Nivolumab and DAY101 for the treatment of newly diagnosed or recurrent craniopharyngioma in children and young adults


Craniopharyngioma (CP) is a highly debilitating childhood brain tumour that has few treatment options. Current treatment options are mainly surgery and/or radiation.  Both of these treatments can cause children to have a poor quality of life and also become riskier if the cancer progresses.

Recent studies show that numerous body system pathways are active in CP, such as immune, inflammatory and MAPK pathways.  This provides potential drug targets for treatment.  Early phase clinical studies in CP have supported the use of chemotherapy medicines to inactivate MAPK pathways, and immune and inflammatory pathways.

PNOC029 will test nivolumab and tovorafenib (also known as DAY101) either administered together or separately. These medicines will be prescribed to children and young adults who have already had surgery or are going to have surgery for the removal of their tumour.  The combination of these two chemotherapy medicines before and after surgery, may help to provide better, and less toxic treatment, resulting in children having a better quality of life.

Disease Stage: Newly Diagnosed, Recurrent

Patient Age Range: Patients 1 to 39 Years

Trial sponsors

  • National – ANZCHOG
  • International –  Pacific Pediatric Neuro-Oncology Consortium (PNOC)

ANZCHOG acknowledges the valuable support of PNOC029 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.