This study will be conducted in two phases. The first phase (phase 0) will be looking at patients with new or recurrent/ progressed craniopharyngioma tumors. These patients will be given one dose of tocilizumab before they have SOC surgery of their tumor. The objective of this phase is to see if drug reaches the tumor. If phase 0 is favorable and shows that drug is penetrating the tumor, the second phase of the study (feasibility phase) will open. Both phases will remain open concurrently and patients will be able to enroll on the Phase 0 then "roll over" and enroll on the feasibility phase. During the feasibility phase patients will be administered tocilizumab every two weeks for up to 13 cycles (approximately 1 year). Patients will be followed for up to 5 years in the feasibility phase.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
9
Phase 0: One dose of tocilizumab prior to surgery Feasibility phase: Tocilizumab administered every 2 weeks for up to 13 cycles (approximately 1 year).
Children's Hospital Colorado
Aurora, Colorado, United States
Phase 0: Presence of Tocilizumab and Metabolites
Utilize biopsy and/or drainage to identify the presence of tocilizumab and its metabolites in adamantinomatous craniopharyngioma (ACP) tumor tissue and/or cyst fluid and/or CSF following one dose of systemically administered tocilizumab.
Time frame: Within 4 to 8 hours of administration of tocilizumab
Feasibility Phase: Toxicity Profile
To define toxicities of tocilizumab therapy using CTCAE version 5.
Time frame: Start of study to end of study, up to 5 years
Phase 0: IL6 and Inflammatory Cytokines
To define levels of IL6 and other inflammatory cytokines in biopsied tissue and/or cyst fluid as measured by enzyme-linked immunosorbent assay (ELISA) following 1 dose of systemically administered tocilizumab
Time frame: Within 4 to 8 hours of administration of tocilizumab
Feasibility Phase: Progression Free Survival (PFS)
Utilize radiography to estimate PFS of subjects with newly diagnosed, unresectable or recurrent/progressive ACP (with or without prior radiation therapy) treated with systemic tocilizumab.
Time frame: From date of enrollment until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 60 months
Feasibility Phase: Pathway Activation
To demonstrate evidence of WNT (Wingless-related integration site) in tumor tissue using immunohistochemistry and transcription array
Time frame: Start of study to end of study, up to 5 years
Feasibility Phase: Pathway Activation
To demonstrate evidence of MAPK (mitogen activated protein kinases) in tumor tissue using immunohistochemistry and transcription arr
Time frame: Start of study to end of study, up to 5 years
Feasibility Phase: Immunity
To demonstrate immune cell infiltration in tumor tissue using immunohistochemistry and flow cytometry
Time frame: Start of study to end of study, up to 5 years
Feasibility Phase: Cytokines
To characterize cytokine signaling in tumor tissue and/or cyst fluid using enzyme-linked immunosorbent assay (ELISA)To characterize cytokine signaling in tumor tissue and/or cyst fluid using enzyme-linked immunosorbent assay (ELISA)
Time frame: Start of study to end of study, up to 5 years
Feasibility Phase: Overall Response Rate (ORR)
Utilize radiography to estimate the overall response rate of subjects with newly diagnosed, unresectable or recurrent/progressive ACP (with or without prior radiation therapy) treated with systemic tocilizumab.
Time frame: Start of study to end of study, or up to 5 years
Feasibility Phase: 1-Year Disease Stabilization
Utilize radiography to estimate the 1-year disease stabilization rate of subjects with newly diagnosed, unresectable or recurrent/progressive ACP (with or without prior radiation therapy) treated with systemic tocilizumab.
Time frame: Start of study to 1 year post treatment
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