A Phase 1 Study of Fisogatinib (BLU-554) in Patients With Hepatocellular Carcinoma
A Phase 1 Study of Fisogatinib (BLU-554) in Patients With Hepatocellular Carcinoma
This is a Phase 1, open-label, first-in-human (FIH) study designed to evaluate the safety,
 tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antineoplastic
 activity of fisogatinib (formerly known as BLU- 554) administered orally in patients with
 FGF19 IHC+ hepatocellular carcinoma (HCC). The study consists of 3 parts, a dose-escalation
 part (Part 1), an expansion part (Part 2) exploring a once daily (qd) dosing schedule at the
 recommended Phase 2 dose (RP2D), and a Part 3 expansion of the qd dosing schedule at the RP2D
 in TKI naive patients.
Key Inclusion Criteria:

 -  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.

 Key Exclusion Criteria:

 -  Central nervous system metastases

 -  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