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Clinical Trial 18322

Cancer Type: Gastrointestinal Tumor

Study Type: Treatment
Phase of Study: Phase I

  • Richard Kim

Call 813-745-6100
or 1-800-679-0775

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

  • 18 years of age or older
  • For Part 1, patient is 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).
  • Confirmed diagnosis of Hepatocellular Carcinoma (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 (Bruix et al, 2005, Part 1, Part 2, and Part 3), or patient has relapsed or refractory solid tumor other than HCC that has evidence of aberrant FGF19/FGFR4 pathway activity (Part 1 enrichment cohorts ONLY). For Part 1 and Part 2, the patient has unresectable disease and has been previously treated with sorafenib, has declined sorafenib, or does not have access to sorafenib. For Part 3, the patient has not received prior treatment with a TKI. Prior immunotherapy may be allowed upon approval by Medical Monitor.
  • For Part 2 and Part 3, patient has at least one target lesion evaluable by RECIST, version 1.1.
  • For Part 2 and Part 3, all participants must have an FGF19 IHC result available. Only FGF19 IHC+ HCC patients will be eligible for Part 3.
  • Child-Pugh class A with no clinically apparent ascites
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0-1
  • Additional criteria may apply

  • Exclusion Criteria

  • Central nervous system metastases
  • Platelet count less than 75,000/mL
  • Absolute neutrophil count less than 1000/mL
  • Hemoglobin less than 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 less than 40 mL/min
  • Additional criteria may apply