Phase I/II Study of TSU-68 for Advanced Hepatocellular Carcinoma

Phase I/II Study of TSU-68 for Advanced Hepatocellular Carcinoma

The purpose of this study is to evaluate the safety and efficacy of Orantinib, an oral tyrosine kinase inhibitor of vascular endothelial growth factor receptor-2, platelet-derived growth factor receptor, and fibroblast growth factor receptor, in patients with advanced hepatocellular carcinoma (HCC).

As HCC is a highly vascular tumor, a number of antiangiogenic agents have been tested for the treatment of HCC. Orantinib is an orally administered, small-molecule, multiple receptor tyrosine kinase inhibitor that targets vascular endothelial growth factor receptor-2 (VEGFR-2), platelet-derived growth factor receptor (PDGFR), and fibroblast growth factor receptor (FGFR). Phase I studies that have been conducted in Japan for patients with solid tumors recommended a dosage of 400 mg bid. As a potent antiangiogenic agent, Orantinib is also expected to be effective against HCC. However, because most HCC patients have accompanying liver cirrhosis or hepatitis, its safety must be reevaluated in the presence of liver function impairment.

Inclusion Criteria:

• Age 20-74
• PS 0-2
• Patients who did not respond to surgery, RFA, TAE, chemotherapy, or radiotherapy
• Chid-Pugh A or B
• At least one measurable lesion by RECIST criteria

Exclusion Criteria:

• Large amount of pleural effusion or ascites
• Esophageal varices
• Simultaneously active double cancer