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A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of BLU-554 in Patients with Hepatocellular Carcinoma
This study is designed to obtain information on the safety and tolerability of an experimental drug called BLU-554 when it is given to patients with Hepatocellular Carcinoma (HCC) (cancer of the liver) and Cholangiocarcinoma (cancer of the bile duct).
Primary: To determine the MTD and recommended Phase 2 dose (RP2D) of BLU-554. To determine the safety and tolerability of BLU-554. Secondary: To characterize the pharmacokinetic (PK) profile of BLU-554. To assess FGF19 status in tumor tissue via immunohistochemistry (IHC) and fluorescent in situ hybridization (FISH). To characterize the pharmacodynamics (PD) of BLU-554, including, but not limited to, changes in blood FGF19, glucose, cholesterol, triglycerides, bile acid precursors, alpha fetoprotein, and cancer antigen 19-9 (CA 19-9) levels; and changes in tumor Ki-67 and cleaved caspase-3 levels. To describe preliminary evidence of BLU-554 antineoplastic activity according to Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1.