St. Jude Children's Research Hospital National Cancer Institute (NCI)
Estimated Enrollment
Phase I Trial (skeletally-immature patients): 6-10 patients per year Surgical cohort: 2-3 patients per year Phase II Trial (skeletally-mature patients): 10-12 patients per year
Objective
Phase I Trial ( Dose Escalation Phase)
Primary endpoint
To estimate the maximum tolerated dose (MTD) and/or the recommended phase II dose (RP2D).
To describe the toxicity profile and define the dose-limiting toxicities (DLTs) and to characterize the pharmacokinetics profile.
Secondary endpoint
To document preliminary antitumor activity
To estimate the maximum tolerated dose (MTD) and/or the recommended phase II dose (RP2D)
Surgical cohort
Primary endpoint
To characterize the concentrations of CX-4945 in tumor after administration of CX-4945 and surgical resection
Secondary endpoint
To explore the ability of CX-4945 at the MTD/ RP2D to inhibit CK2-mediated signaling in tumor
Phase II Trial (to establish the safety of 1000mg/m2 BID given continuously)
Primary endpoint
To establish the safety and characterize the toxicity of 1000mg/m2 BID continuous dosing of CX-4945
To estimate the objective response rate
Secondary endpoint
To characterize the pharmacokinetics
To perform a genomic analysis within the confines of a Phase II study
