Title
A Phase 1 Study of Fisogatinib (BLU-554) in Patients With Hepatocellular Carcinoma
A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of BLU-554 in Patients With Hepatocellular Carcinoma
Phase
Phase 1Lead Sponsor
Blueprint Medicines CorporationStudy Type
InterventionalStatus
Active, not recruitingIndication/Condition
Hepatocellular Carcinoma (HCC)Intervention/Treatment
blu-554 ...Study Participants
150This 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.
Fisogatinib (BLU-554) capsules for oral administration.
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
