Adamantinomatous Craniopharyngioma (ACP) is a highly debilitating paediatric brain tumour that lacks medical anti-tumour therapies. Current therapy, which depends largely on surgery and radiation, is associated with poor quality of life and becomes more challenging and risky in the setting of recurrent disease. 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.
CONNECT1905 is a Phase II, open-label study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of Tocilizumab in pediatric and young adult patients with progressive or recurrent adamantinomatous craniopharyngioma (ACP). Tocilizumab is administered intravenously to target the interleukin-6 (IL-6) pathway, which is implicated in the inflammatory tumour microenvironment of ACP. The primary objective is to assess the objective response rate (ORR). Secondary endpoints include progression-free survival (PFS), overall survival (OS), duration of response, safety, and pharmacokinetics. This study aims to determine whether IL-6 pathway inhibition can provide a novel, targeted treatment approach for patients with ACP, a rare brain tumour with limited therapeutic options following recurrence or progression.