For Healthcare Professionals

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

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About the study

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.
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Who can take part

You may be eligible to participate in the study if you meet the following criteria:

INCLUSION CRITERIA

  1. 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).
  2. 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.
  3. For Part 3, the patient has not received prior treatment with a TKI.
  4. Child-Pugh class A with no clinically apparent ascites
  5. Eastern Cooperative Oncology Group (ECOG) performance status of 0-1
  6. For Part 1, willing to provide archived tumor tissue (if available) and willing to undergo pre
  7. and on-treatment tumor biopsy (if considered safe and medically feasible by the treating investigator)
  8. 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

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

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

How to Apply


Contact the study center to learn if this study is a good match for you.
Phone iconCall 617-714-6707Email iconEmail Study Center

Study’s details


Contition

Cholangiocarcinoma,Hepatocellular Carcinoma (HCC)

Age

18+

Phase

Phase 1

Participants needed

150

Est. Completion Date

Dec 2022

Treatment type

Interventional


Sponsor

Blueprint Medicines Corporation

ClinicalTrials.gov identifier

NCT02508467

Study number

BLU-554-1101

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