Clinical trial · Interventional
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
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Hepatocellular Carcinoma (HCC) | Hepatocellular Carcinoma | ONTOLOGY_EXACT | 0.85 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Fisogatinib (BLU-554) | Drug | Fisogatinib | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Fisogatinib (BLU-554)
- description
- Fisogatinib (BLU-554) capsules for oral administration.
- interventionNames
- Drug: Fisogatinib (BLU-554)
Primary outcomes (3)
- measure
- Maximum tolerated dose (MTD) on qd and bid schedules
- timeFrame
- During cycle 1 (28 days) of treatment and will be determined by approximately 24 months after start of the study or earlier
- measure
- Recommended Phase 2 dose of fisogatinib (BLU-554) on qd and bid schedules
- timeFrame
- At the end of every cycle (28 days) of treatment and will be determined by approximately 24 months after start of the study or earlier
- measure
- Number of patients with adverse events, serious adverse events and changes in physical findings, vital signs, clinical laboratory results and ECG findings
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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
References
Publications (0)
Data not yet available