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
2 other identifiers
interventional
146
12 countries
41
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_1
Started Jul 2015
Longer than P75 for phase_1
41 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 9, 2015
CompletedFirst Posted
Study publicly available on registry
July 27, 2015
CompletedStudy Start
First participant enrolled
July 31, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 30, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
February 28, 2024
CompletedApril 12, 2024
March 1, 2024
5.9 years
July 9, 2015
April 10, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (3)
Maximum tolerated dose (MTD) on qd and bid schedules
During cycle 1 (28 days) of treatment and will be determined by approximately 24 months after start of the study or earlier
Recommended Phase 2 dose of fisogatinib (BLU-554) on qd and bid schedules
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
Number of patients with adverse events, serious adverse events and changes in physical findings, vital signs, clinical laboratory results and ECG findings
Every cycle (28 days) for approximately 24 months or earlier if patient terminates from the study
Secondary Outcomes (5)
Maximum plasma concentration of fisogatinib (BLU-554) on qd and bid schedules
Every cycle (28 days) up to cycle 4 and at end of treatment (approximately 24 months or earlier if patient terminates from the study)
Time to maximum plasma concentration of fisogatinib (BLU-554) on qd and bid schedules
Every cycle (28 days) up to cycle 4 and at end of treatment (approximately 24 months or earlier if patient terminates from the study)
Fibroblast growth factor 19 (FGF19) status in tumor tissue
Cycle 2 (Day 56)
Levels of FGF19 in blood and tumor samples
Cycle 1 (Day 28)
Preliminary evidence of fisogatinib (BLU-554) antineoplastic activity
Screening, Day 1 of every odd numbered cycle starting with Cycle 3, End of treatment (at approximately 24 months or earlier if patient terminates from the study) and every three months post EOT
Study Arms (1)
Fisogatinib (BLU-554)
EXPERIMENTALFisogatinib (BLU-554) capsules for oral administration.
Interventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (41)
Inland Empire Liver Foundation
Rialto, California, 92377, United States
University of Miami - Sylvester Comprehensive Cancer Center
Miami, Florida, 33136, United States
H. Lee Moffitt Cancer Center
Tampa, Florida, 33612, United States
Ochsner Cancer Institute
New Orleans, Louisiana, 70121, United States
Massachusetts General Hospital
Boston, Massachusetts, 02144, United States
Mount Sinai Medical Center
New York, New York, 10029, United States
Huntsman Cancer Institute
Salt Lake City, Utah, 84112, United States
Zhejiang Cancer Hospital
Hangzhou, Gongshu District, 310022, China
Nanfang Hospital
Guangzhou, Guangdong, 510445, China
Harbin Medical University Cancer Hospital
Harbin, Heilongjiang, 150081, China
Henan Cancer Hospital
Zhengzhou, Henan, 450008, China
Hunan Cancer Hospital, Radioactive Interventional Department
Changsha, Hunan, 410013, China
The Chinese People's Liberation Army 81 Hospital
Nanjing, Jiangsu, 210002, China
Nantong Tumor Hospital
Nantong, Jiangsu, 226361, China
Jilin Cancer Hospital
Changchun, Jilin, 130012, China
Jilin University the First Affiliated Hospital
Changchun, Jilin, 130021, China
Fudan University Zhongshan Hospital
Xuhui, Shanghai City, 200032, China
West China Hospital, Sichuan University
Chengdu, Sichuan, 610041, China
Tianjin Medical University Cancer Institute & Hospital, Hepatobiliary Oncology Department
Tianjin, West Lake District, 300060, China
Fudan University Shanghai Cancer Center
Shanghai, Xuhui District, 200032, China
The First Affiliated Hospital, College of Medicine, Zhejiang University
Hangzhou, Zhejiang, 310003, China
Hospital Beaujon
Clichy, 92110, France
Institut Gustave Roussy
Villejuif, 94805, France
Johannes Gutenberg University Mainz - University Medical Center
Mainz, Rhineland-Palatine, 55131, Germany
University of Frankfurt
Frankfurt, 60590, Germany
Prince of Wales Hospital
Hong Kong, Hong Kong
Queen Mary Hospital
Hong Kong, Hong Kong
IRCCS Foundation - National Institute of Tumors
Milan, 21033, Italy
National Cancer Centre
Singapore, 169610, Singapore
National Cancer Center
Gyeonggi-do, 10408, South Korea
Seoul National University Hospital
Seoul, 03080, South Korea
Asan Medical Center
Seoul, 05505, South Korea
Samsung Medical Center
Seoul, 06351, South Korea
Vall d'Hebron Institute of Oncology
Barcelona, 08035, Spain
Inselspital Bern
Bern, 3010, Switzerland
National Cheng Kung University Hospital
Tainan, 704, Taiwan
National Taiwan University Hospital
Taipei, 100, Taiwan
University of Liverpool - Clatterbridge Cancer Centre
Bebington, CH63 4JY, United Kingdom
Royal Free Hospital
London, NW3 2QG, United Kingdom
Guy's Hospital
London, SE1 9RY, United Kingdom
University College London
London, W1T 7HA, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 9, 2015
First Posted
July 27, 2015
Study Start
July 31, 2015
Primary Completion
June 30, 2021
Study Completion
February 28, 2024
Last Updated
April 12, 2024
Record last verified: 2024-03