Current Research Studies

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


What is the goal of the study?

Rationale: Adamantinomatous Craniopharyngioma (ACP) is a highly debilitating pediatric brain tumor that lacks medical anti-tumor 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 available agents, including IL-6 pathway blockers may have efficacy in the control of ACP. Hypothesis: Single-agent tocilizumab will be safe and effective at inducing tumor response in children with residual adamantinotamous craniopharyngioma. Study Design: A multicenter single-agent tocilizumab Phase II trial with two strata ? Stratum 1: Patients with progressive or recurrent adamantinomatous craniopharyngiomas following radiation therapy ? Stratum 2: Patients with measurable adamantinomatous craniopharyngioma who have undergone surgery but have not previously received radiation therapy. Progressive disease is allowed but not required Patient Population: Patients aged >1 year and <25 years with unresectable acp who may have been previously treated with radiation (stratum 1) or without radiation (stratum 2).>

Who can participate in the study?

Please contact the study team listed below to learn more.

Study Team: