A Phase 1 Study of Fisogatinib (BLU-554) in Patients With Hepatocellular Carcinoma
Purpose
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.
Condition
- Hepatocellular Carcinoma (HCC)
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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
Study Design
- Phase
- Phase 1
- Study Type
- Interventional
- Allocation
- N/A
- Intervention Model
- Single Group Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Fisogatinib (BLU-554) |
Fisogatinib (BLU-554) capsules for oral administration. |
|
More Details
- Status
- Completed
- Sponsor
- Blueprint Medicines Corporation