A Phase 1 Study of BLU-554 in Patients With Hepatocellular Carcinoma



Status:Recruiting
Conditions:Liver Cancer, Liver Cancer, Cancer, Cancer
Therapuetic Areas:Oncology
Healthy:No
Age Range:18 - Any
Updated:10/5/2018
Start Date:July 2015
End Date:August 2020
Contact:Blueprint Medicines
Email:studydirector@blueprintmedicines.com
Phone:617-714-6707

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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 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.


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
We found this trial at
8
sites
1 Gustave L Levy Pl # 271
New York, New York 10029
 (212) 241-6500
Mount Sinai Med Ctr Founded in 1852, The Mount Sinai Hospital is a 1,171-bed, tertiary-care...
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185 Cambridge Street
Boston, Massachusetts 02114
617-724-5200
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Boston, MA
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100 Boulevard du Géneral Leclerc
Clichy, 92110
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Clichy,
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1475 NW 12th Ave
Miami, Florida 33136
(305) 243-1000
University of Miami, Sylvester Comprehensive Cancer Center Sylvester Comprehensive Cancer Center integrates all cancer-related activities...
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Miami, FL
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New Orleans, Louisiana 70121
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New Orleans, LA
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Rialto, California 92377
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Rialto, CA
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Salt Lake City, Utah 84112
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Salt Lake City, UT
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Tampa, Florida 33612
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Tampa, FL
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