CONNECT1905: Phase 2 Study of Systemic IL-6 Receptor Antagonist ACTEMRA® (tocilizumab) for the treatment of Progressive/Recurrent Pediatric Adamantinomatous Craniopharyngioma

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 IL-6 pathway blockers currently used to treat a range of other types of diseases may be effective in the control of ACP.

In this study, up to 38 patients will receive the IL-6 pathway blocker ACTEMRA (tocilizumab) for up to 2 years. The study will investigate if tocilizumab 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)