A Phase 1 Study of Fisogatinib (BLU-554) in Patients With Hepatocellular Carcinoma

Lab values

• Platelet count \<75,000/mL
• Absolute neutrophil count \<1000/mL
• Hemoglobin \<8 g/dL
• Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \>5x the upper limit of normal (ULN)
• Total bilirubin \>2.5 mg/dL
• International normalized ratio (INR) \>2.3 or prothrombin time (PT) \>6 seconds above control
• Estimated (Cockroft-Gault formula) or measured creatinine clearance \<40 mL/min

Other

• Central nervous system metastases

• Confirmed diagnosis of HCC by histological examination or by non-invasive criteria according to European Association for the Study of the Liver (EASL) or American Association for the Study of Liver Disease (AASLD) guidelines (Part 1, 2 and 3).
• For Part 1 and 2, the patient has unresectable disease and has been previously treated with sorafenib, has declined treatment with sorafenib, or does not have access to sorafenib.
• For Part 3, the patient has not received prior treatment with a TKI.
• Child-Pugh class A with no clinically apparent ascites
• Eastern Cooperative Oncology Group (ECOG) performance status of 0-1
• For Part 1, willing to provide archived tumor tissue (if available) and willing to undergo pre- and on-treatment tumor biopsy (if considered safe and medically feasible by the treating investigator)
• For Part 2 and 3, all patients must have an FGF19 IHC result available. Only FGF19 IHC+ HCC patients will be eligible for Part 3.