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

Status: Active


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 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.

Eligibility Criteria

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.

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

Locations & Contacts


Moffitt Cancer Center
Status: Active
Contact: Richard D. Kim
Phone: 800-456-7121
Email: canceranswers@moffitt.org


Brigham and Women's Hospital
Status: Active
Contact: Thomas Adam Abrams
Phone: 877-442-3324
Email: tabrams1@partners.org
Dana-Farber Cancer Institute
Status: Active
Contact: Thomas Adam Abrams
Phone: 877-442-3324
Email: tabrams1@partners.org
Massachusetts General Hospital Cancer Center
Status: Active
Name Not Available

New York

New York
Icahn School of Medicine at Mount Sinai
Status: Active
Name Not Available

Trial Phase & Type

Trial Phase

Phase I

Trial Type


Lead Organization

Lead Organization
Blueprint Medicines Corporation

Trial IDs

Primary ID BLU-554-1101
Secondary IDs NCI-2015-01683, 2015-001662-26
Clinicaltrials.gov ID NCT02508467