Hepatocellular Carcinoma (HCC)Phase 1NCT02508467
A Phase 1 Study of Fisogatinib (BLU-554) in Patients With Hepatocellular Carcinoma
A Phase 1 study testing Fisogatinib (BLU-554) in people with Hepatocellular Carcinoma (HCC), run by Blueprint Medicines Corporation at 41 sites in 12 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hepatocellular Carcinoma (HCC)
- What is being tested?
- Fisogatinib (BLU-554)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 146 participants
- Where
- 41 sitesin 12 countries
- Study dates
- 31 Jul 2015to 28 Feb 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
41 sites in 12 countries.
41 sites in 12 countries
China14 sites
Jilin Cancer Hospital
Changchun, Jilin, 130012
Jilin University the First Affiliated Hospital
Changchun, Jilin, 130021
Hunan Cancer Hospital, Radioactive Interventional Department
Changsha, Hunan, 410013
West China Hospital, Sichuan University
Chengdu, Sichuan, 610041
Nanfang Hospital
Guangzhou, Guangdong, 510445
Zhejiang Cancer Hospital
Hangzhou, Gongshu District, 310022
The First Affiliated Hospital, College of Medicine, Zhejiang University
Hangzhou, Zhejiang, 310003
Harbin Medical University Cancer Hospital
Harbin, Heilongjiang, 150081
The Chinese People's Liberation Army 81 Hospital
Nanjing, Jiangsu, 210002
Nantong Tumor Hospital
Nantong, Jiangsu, 226361
Fudan University Shanghai Cancer Center
Shanghai, Xuhui District, 200032
Tianjin Medical University Cancer Institute & Hospital, Hepatobiliary Oncology Department
Tianjin, West Lake District, 300060
Fudan University Zhongshan Hospital
Xuhui, Shanghai City, 200032
Henan Cancer Hospital
Zhengzhou, Henan, 450008
United States7 sites
Massachusetts General Hospital
Boston, Massachusetts, 02144
University of Miami - Sylvester Comprehensive Cancer Center
Miami, Florida, 33136
Ochsner Cancer Institute
New Orleans, Louisiana, 70121
Mount Sinai Medical Center
New York, New York, 10029
Inland Empire Liver Foundation
Rialto, California, 92377
Huntsman Cancer Institute
Salt Lake City, Utah, 84112
H. Lee Moffitt Cancer Center
Tampa, Florida, 33612
South Korea4 sites
National Cancer Center
Gyeonggi-do, 10408
Seoul National University Hospital
Seoul, 03080
Asan Medical Center
Seoul, 05505
Samsung Medical Center
Seoul, 06351
United Kingdom4 sites
University of Liverpool - Clatterbridge Cancer Centre
Bebington, CH63 4JY
Royal Free Hospital
London, NW3 2QG
Guy's Hospital
London, SE1 9RY
University College London
London, W1T 7HA
France2 sites
Hospital Beaujon
Clichy, 92110
Institut Gustave Roussy
Villejuif, 94805
Germany2 sites
University of Frankfurt
Frankfurt, 60590
Johannes Gutenberg University Mainz - University Medical Center
Mainz, Rhineland-Palatine, 55131
Hong Kong2 sites
Prince of Wales Hospital
Hong Kong
Queen Mary Hospital
Hong Kong
Taiwan2 sites
National Cheng Kung University Hospital
Tainan, 704
National Taiwan University Hospital
Taipei, 100
About this study
Official title: A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of BLU-554 in Patients With Hepatocellular Carcinoma
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
- Hepatocellular Carcinoma (HCC)
- Being tested
- Fisogatinib (BLU-554)
- Keywords
- Liver cancer, FGF19 gene amplification, FGF19 overexpression, FGF19 upregulation, Cyclin D1 (CCND1) gene amplification, Cyclin D1 (CCND1) copy number gain, BLU-554, FGFR4, Hepatocellular carcinoma, Liver Disease, Liver Neoplasms
- Medical subject headings
- Carcinoma, Hepatocellular, Liver Neoplasms, Liver Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fisogatinib (BLU-554)Drug
Also called BLU-554
| Study group | Role | What they receive |
|---|---|---|
| Fisogatinib (BLU-554) | Experimental | Fisogatinib (BLU-554) capsules for oral administration. Receives: Drug: Fisogatinib (BLU-554) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum tolerated dose (MTD) on qd and bid schedules
Measured: 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
Measured: 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
Measured: 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
Blood samples may be taken at pre-dose, and 0.5, 1, 2, 4, 6, 8 and 24 hrs post dose on Cycle 1 Day 1 and Cycle 1 Day 15, Pre-dose of Cycle 2 to 4, Day 1 and end of treatment (EOT)
Measured: 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
Blood samples may be taken at pre-dose, and 0.5, 1, 2, 4, 6, 8 and 24 hrs post dose on Cycle 1 Day 1 and Cycle 1 Day 15, Pre-dose of Cycle 2 to 4, Day 1 and EOT
Measured: 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
Measured: Cycle 2 (Day 56)
Levels of FGF19 in blood and tumor samples
Measured: Cycle 1 (Day 28)
Preliminary evidence of fisogatinib (BLU-554) antineoplastic activity
Measured: 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jul 2015 (estimated)
- Study start31 Jul 2015
- Primary completion30 Jun 2021
- Study completion28 Feb 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hepatocellular Carcinoma (HCC)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 146 participants (actual)
Sponsor and investigators
- Lead sponsor
- Blueprint Medicines Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02508467
- Sponsor's protocol ID
- BLU-554-1101
- Other IDs
- 2015-001662-26, 2015
- Status last verified
- Mar 2024
- First submitted
- 9 Jul 2015
- Last updated
- 12 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of BLU-554 in Patients With Hepatocellular Carcinoma
- ClinicalTrials.gov ID
- NCT02508467
- Acronym / sponsor ID
- BLU-554-1101
- Conditions
- Hepatocellular Carcinoma (HCC)
- Keywords
- Liver cancer, FGF19 gene amplification, FGF19 overexpression, FGF19 upregulation, Cyclin D1 (CCND1) gene amplification, Cyclin D1 (CCND1) copy number gain, BLU-554, FGFR4
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 146 participants (actual)
- Lead sponsor
- Blueprint Medicines CorporationIndustry (pharma, biotech, devices)
- Study start
- 31 Jul 2015 (actual)
- Primary completion
- 30 Jun 2021 (actual)
- Study completion
- 28 Feb 2024 (actual)
- First posted
- 27 Jul 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fisogatinib (BLU-554) | Experimental | Drug: Fisogatinib (BLU-554)Fisogatinib (BLU-554) capsules for oral administration. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (8 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Fisogatinib (BLU-554)Also: BLU-554 | Drug | Fisogatinib (BLU-554) |
Primary outcome
Maximum tolerated dose (MTD) on qd and bid schedules
Time frame: 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
Time frame: 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
Time frame: Every cycle (28 days) for approximately 24 months or earlier if patient terminates from the study
5 secondary outcomes
Maximum plasma concentration of fisogatinib (BLU-554) on qd and bid schedules
Time frame: 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
Time frame: 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
Time frame: Cycle 2 (Day 56)
Levels of FGF19 in blood and tumor samples
Time frame: Cycle 1 (Day 28)
Preliminary evidence of fisogatinib (BLU-554) antineoplastic activity
Time frame: 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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Apr 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
41 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Fisogatinib (BLU-554) in people with Hepatocellular Carcinoma (HCC), run by Blueprint Medicines Corporation at 41 sites in 12 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 41 sites in China, United States, South Korea, United Kingdom, France and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum tolerated dose (MTD) on qd and bid schedules (time frame: During cycle 1 (28 days) of treatment and will be determined by approximately 24 months after start of the study or earlier). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Apr 2024.