Ignite Creation Date:
2025-12-25 @ 12:39 AM
Ignite Modification Date:
2026-01-06 @ 3:55 PM
Study NCT ID:
NCT02508467
Status:
COMPLETED
Last Update Posted:
2024-04-12
First Post:
2015-07-09
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Phase 1 Study of Fisogatinib (BLU-554) in Patients With Hepatocellular Carcinoma
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006528', 'term': 'Carcinoma, Hepatocellular'}, {'id': 'D008113', 'term': 'Liver Neoplasms'}, {'id': 'D008107', 'term': 'Liver Diseases'}], 'ancestors': [{'id': 'D000230', 'term': 'Adenocarcinoma'}, {'id': 'D002277', 'term': 'Carcinoma'}, {'id': 'D009375', 'term': 'Neoplasms, Glandular and Epithelial'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D004067', 'term': 'Digestive System Neoplasms'}, {'id': 'D009371', 'term': 'Neoplasms by Site'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000709257', 'term': 'fisogatinib'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 146}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2015-07-31', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-03', 'completionDateStruct': {'date': '2024-02-28', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-04-10', 'studyFirstSubmitDate': '2015-07-09', 'studyFirstSubmitQcDate': '2015-07-23', 'lastUpdatePostDateStruct': {'date': '2024-04-12', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2015-07-27', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2021-06-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'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', 'timeFrame': 'Every cycle (28 days) for approximately 24 months or earlier if patient terminates from the study'}], 'secondaryOutcomes': [{'measure': 'Maximum plasma concentration of fisogatinib (BLU-554) on qd and bid schedules', 'timeFrame': 'Every cycle (28 days) up to cycle 4 and at end of treatment (approximately 24 months or earlier if patient terminates from the study)', 'description': '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)'}, {'measure': 'Time to maximum plasma concentration of fisogatinib (BLU-554) on qd and bid schedules', 'timeFrame': 'Every cycle (28 days) up to cycle 4 and at end of treatment (approximately 24 months or earlier if patient terminates from the study)', 'description': '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'}, {'measure': 'Fibroblast growth factor 19 (FGF19) status in tumor tissue', 'timeFrame': 'Cycle 2 (Day 56)'}, {'measure': 'Levels of FGF19 in blood and tumor samples', 'timeFrame': 'Cycle 1 (Day 28)'}, {'measure': 'Preliminary evidence of fisogatinib (BLU-554) antineoplastic activity', 'timeFrame': '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'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'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'], 'conditions': ['Hepatocellular Carcinoma (HCC)']}, 'descriptionModule': {'briefSummary': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n* 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).\n* 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.\n* For Part 3, the patient has not received prior treatment with a TKI.\n* Child-Pugh class A with no clinically apparent ascites\n* Eastern Cooperative Oncology Group (ECOG) performance status of 0-1\n* 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)\n* 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.\n\nKey Exclusion Criteria:\n\n* Central nervous system metastases\n* Platelet count \\<75,000/mL\n* Absolute neutrophil count \\<1000/mL\n* Hemoglobin \\<8 g/dL\n* Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \\>5x the upper limit of normal (ULN)\n* Total bilirubin \\>2.5 mg/dL\n* International normalized ratio (INR) \\>2.3 or prothrombin time (PT) \\>6 seconds above control\n* Estimated (Cockroft-Gault formula) or measured creatinine clearance \\<40 mL/min'}, 'identificationModule': {'nctId': 'NCT02508467', 'briefTitle': 'A Phase 1 Study of Fisogatinib (BLU-554) in Patients With Hepatocellular Carcinoma', 'organization': {'class': 'INDUSTRY', 'fullName': 'Blueprint Medicines Corporation'}, 'officialTitle': 'A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of BLU-554 in Patients With Hepatocellular Carcinoma', 'orgStudyIdInfo': {'id': 'BLU-554-1101'}, 'secondaryIdInfos': [{'id': '2015-001662-26', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Fisogatinib (BLU-554)', 'description': 'Fisogatinib (BLU-554) capsules for oral administration.', 'interventionNames': ['Drug: Fisogatinib (BLU-554)']}], 'interventions': [{'name': 'Fisogatinib (BLU-554)', 'type': 'DRUG', 'otherNames': ['BLU-554'], 'armGroupLabels': ['Fisogatinib (BLU-554)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '92377', 'city': 'Rialto', 'state': 'California', 'country': 'United States', 'facility': 'Inland Empire Liver Foundation', 'geoPoint': {'lat': 34.1064, 'lon': -117.37032}}, {'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Miami - Sylvester Comprehensive Cancer Center', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '33612', 'city': 'Tampa', 'state': 'Florida', 'country': 'United States', 'facility': 'H. Lee Moffitt Cancer Center', 'geoPoint': {'lat': 27.94752, 'lon': -82.45843}}, {'zip': '70121', 'city': 'New Orleans', 'state': 'Louisiana', 'country': 'United States', 'facility': 'Ochsner Cancer Institute', 'geoPoint': {'lat': 29.95465, 'lon': -90.07507}}, {'zip': '02144', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Massachusetts General Hospital', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '10029', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Mount Sinai Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '84112', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'Huntsman Cancer Institute', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '310022', 'city': 'Hangzhou', 'state': 'Gongshu District', 'country': 'China', 'facility': 'Zhejiang Cancer Hospital', 'geoPoint': {'lat': 30.29365, 'lon': 120.16142}}, {'zip': '510445', 'city': 'Guangzhou', 'state': 'Guangdong', 'country': 'China', 'facility': 'Nanfang Hospital', 'geoPoint': {'lat': 23.11667, 'lon': 113.25}}, {'zip': '150081', 'city': 'Harbin', 'state': 'Heilongjiang', 'country': 'China', 'facility': 'Harbin Medical University Cancer Hospital', 'geoPoint': {'lat': 45.75, 'lon': 126.65}}, {'zip': '450008', 'city': 'Zhengzhou', 'state': 'Henan', 'country': 'China', 'facility': 'Henan Cancer Hospital', 'geoPoint': {'lat': 34.75778, 'lon': 113.64861}}, {'zip': '410013', 'city': 'Changsha', 'state': 'Hunan', 'country': 'China', 'facility': 'Hunan Cancer Hospital, Radioactive Interventional Department', 'geoPoint': {'lat': 28.19874, 'lon': 112.97087}}, {'zip': '210002', 'city': 'Nanjing', 'state': 'Jiangsu', 'country': 'China', 'facility': "The Chinese People's Liberation Army 81 Hospital", 'geoPoint': {'lat': 32.06167, 'lon': 118.77778}}, {'zip': '226361', 'city': 'Nantong', 'state': 'Jiangsu', 'country': 'China', 'facility': 'Nantong Tumor Hospital', 'geoPoint': {'lat': 32.03028, 'lon': 120.87472}}, {'zip': '130012', 'city': 'Changchun', 'state': 'Jilin', 'country': 'China', 'facility': 'Jilin Cancer Hospital', 'geoPoint': {'lat': 43.88, 'lon': 125.32278}}, {'zip': '130021', 'city': 'Changchun', 'state': 'Jilin', 'country': 'China', 'facility': 'Jilin University the First Affiliated Hospital', 'geoPoint': {'lat': 43.88, 'lon': 125.32278}}, {'zip': '200032', 'city': 'Xuhui', 'state': 'Shanghai City', 'country': 'China', 'facility': 'Fudan University Zhongshan Hospital', 'geoPoint': {'lat': 31.19594, 'lon': 121.44709}}, {'zip': '610041', 'city': 'Chengdu', 'state': 'Sichuan', 'country': 'China', 'facility': 'West China Hospital, Sichuan University', 'geoPoint': {'lat': 30.66667, 'lon': 104.06667}}, {'zip': '300060', 'city': 'Tianjin', 'state': 'West Lake District', 'country': 'China', 'facility': 'Tianjin Medical University Cancer Institute & Hospital, Hepatobiliary Oncology Department', 'geoPoint': {'lat': 39.14222, 'lon': 117.17667}}, {'zip': '200032', 'city': 'Shanghai', 'state': 'Xuhui District', 'country': 'China', 'facility': 'Fudan University Shanghai Cancer Center', 'geoPoint': {'lat': 31.22222, 'lon': 121.45806}}, {'zip': '310003', 'city': 'Hangzhou', 'state': 'Zhejiang', 'country': 'China', 'facility': 'The First Affiliated Hospital, College of Medicine, Zhejiang University', 'geoPoint': {'lat': 30.29365, 'lon': 120.16142}}, {'zip': '92110', 'city': 'Clichy', 'country': 'France', 'facility': 'Hospital Beaujon', 'geoPoint': {'lat': 48.90018, 'lon': 2.30952}}, {'zip': '94805', 'city': 'Villejuif', 'country': 'France', 'facility': 'Institut Gustave Roussy', 'geoPoint': {'lat': 48.7939, 'lon': 2.35992}}, {'zip': '55131', 'city': 'Mainz', 'state': 'Rhineland-Palatine', 'country': 'Germany', 'facility': 'Johannes Gutenberg University Mainz - University Medical Center', 'geoPoint': {'lat': 49.98185, 'lon': 8.28008}}, {'zip': '60590', 'city': 'Frankfurt', 'country': 'Germany', 'facility': 'University of Frankfurt', 'geoPoint': {'lat': 49.68333, 'lon': 10.53333}}, {'city': 'Hong Kong', 'country': 'Hong Kong', 'facility': 'Prince of Wales Hospital', 'geoPoint': {'lat': 22.27832, 'lon': 114.17469}}, {'city': 'Hong Kong', 'country': 'Hong Kong', 'facility': 'Queen Mary Hospital', 'geoPoint': {'lat': 22.27832, 'lon': 114.17469}}, {'zip': '21033', 'city': 'Milan', 'country': 'Italy', 'facility': 'IRCCS Foundation - National Institute of Tumors', 'geoPoint': {'lat': 45.46427, 'lon': 9.18951}}, {'zip': '169610', 'city': 'Singapore', 'country': 'Singapore', 'facility': 'National Cancer Centre', 'geoPoint': {'lat': 1.28967, 'lon': 103.85007}}, {'zip': '10408', 'city': 'Gyeonggi-do', 'country': 'South Korea', 'facility': 'National Cancer Center', 'geoPoint': {'lat': 37.58944, 'lon': 126.76917}}, {'zip': '03080', 'city': 'Seoul', 'country': 'South Korea', 'facility': 'Seoul National University Hospital', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'zip': '05505', 'city': 'Seoul', 'country': 'South Korea', 'facility': 'Asan Medical Center', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'zip': '06351', 'city': 'Seoul', 'country': 'South Korea', 'facility': 'Samsung Medical Center', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'zip': '08035', 'city': 'Barcelona', 'country': 'Spain', 'facility': "Vall d'Hebron Institute of Oncology", 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': '3010', 'city': 'Bern', 'country': 'Switzerland', 'facility': 'Inselspital Bern', 'geoPoint': {'lat': 46.94809, 'lon': 7.44744}}, {'zip': '704', 'city': 'Tainan', 'country': 'Taiwan', 'facility': 'National Cheng Kung University Hospital', 'geoPoint': {'lat': 22.99083, 'lon': 120.21333}}, {'zip': '100', 'city': 'Taipei', 'country': 'Taiwan', 'facility': 'National Taiwan University Hospital', 'geoPoint': {'lat': 25.05306, 'lon': 121.52639}}, {'zip': 'CH63 4JY', 'city': 'Bebington', 'country': 'United Kingdom', 'facility': 'University of Liverpool - Clatterbridge Cancer Centre', 'geoPoint': {'lat': 53.35, 'lon': -3.01667}}, {'zip': 'NW3 2QG', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Royal Free Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'SE1 9RY', 'city': 'London', 'country': 'United Kingdom', 'facility': "Guy's Hospital", 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'W1T 7HA', 'city': 'London', 'country': 'United Kingdom', 'facility': 'University College London', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Blueprint Medicines Corporation', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}