A Phase 1 Study of Fisogatinib (BLU-554) in Patients With Hepatocellular Carcinoma (NCT02508467) | Clinical Trial Compass
CompletedPhase 1
A Phase 1 Study of Fisogatinib (BLU-554) in Patients With Hepatocellular Carcinoma
United States, China, France146 participantsStarted 2015-07-31
Plain-language summary
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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this was a Phase 1 trial focused on finding a safe dose of fisogatinib rather than proving it works, what do the early safety and tolerability findings mean for someone in my situation — and is there a later-phase trial now available that might offer more established data?
2This trial measured adverse events, serious adverse events, and changes in things like lab results and heart rhythms — what kinds of side effects showed up, and how would those risks compare to what I might face with my current standard-of-care options for HCC?
3Since the trial is now completed, has the recommended Phase 2 dose been published, and does that mean there are follow-up studies of fisogatinib I might be eligible to discuss as a next step?
4Fisogatinib targets a specific pathway — do you know whether my tumor has been tested for FGFR4 or FGF19 alterations, which this drug was designed to address, and would that affect whether it's even worth exploring trials like this one?
5Given that this was a dose-finding study in hepatocellular carcinoma patients, how does my current liver function and overall health compare to the kinds of patients who were enrolled, and would standard treatments still be a better first path for me?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
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
2
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
3
Number of patients with adverse events, serious adverse events and changes in physical findings, vital signs, clinical laboratory results and ECG findings
Timeframe: Every cycle (28 days) for approximately 24 months or earlier if patient terminates from the study