PNOC-022 (DMG-ACT): A Combination Therapy Trial using an Adaptive Platform Design for Children and Young Adults with Diffuse Midline Gliomas including Diffuse Intrinsic Pontine Gliomas at Initial Diagnosis, Post-Radiation Therapy and at Time of Progression.

Diffuse Midline Gliomas (DMGs) are a group of brain tumours that are extremely aggressive and grow rapidly. Despite extensive research, there are few effective treatment options for patients, hence survival rates are low. This trial will provide a long term framework to test multiple drug options in various stages of this disease.

The first phase of this trial will test if adding the drug ONC201 in combination with two other new drugs (panobinostat and paxalisib) to standard treatments improves outcomes for patients with DMGs. The design of this study is flexible so as to allow other drugs to be rapidly included into the trial in response to promising research emerging in Australia and internationally.

Disease Stage: Newly diagnosed, post radiation therapy or progressive disease

Patient Age Range: 2 to 39 years of age

Trial sponsors

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