Clinical Trial 18322

Cancer Type: Gastrointestinal Tumor

Study Type: Treatment
Phase of Study: Phase I

  • Richard Kim


Study Title

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.

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. Patient has unresectable disease and has been previously treated with sorafenib, has declined treatment with sorafenib, or does not have access to sorafenib. - OR -
  • Confirmed diagnosis of a relapsed or refractory advanced solid tumor other than HCC that has evidence of aberrant FGF19/FGFR4 pathway activity [e.g. tumor or blood/plasma alterations including, but not limited to, 11q13.1 locus (FGF19/CCND1 (cyclin D1)) amplification/copy number gain].
  • Child-Pugh class A with no clinically apparent ascites.
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0-1.
  • 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).

  • Exclusion Criteria

  • Central nervous system metastases.
  • Platelet count > Absolute neutrophil count > Hemoglobin > 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 (Cockcroft-Gault formula) or measured creatinine clearance <40 mL/min.