A Multicenter International Phase 2 Trial Of Pazopanib In Metastatic And Progressive Medullary Thyroid Carcinoma: Mc057h

6026 Background: Pazopanib is a small molecule inhibitor of kinases principally including vascular endothelial growth factor receptors (VEGFR)-1,-2, and -3, platelet-derived growth factor receptors (PDGFR)-α and –β; and c-Kit. We previously reported a tumor response rate of 49% in patients with advanced differentiated thyroid cancer and 0% in patients with advanced anaplastic thyroid cancer. The present report details results of pazopanib therapy in advanced medullary thyroid cancer (MTC). Methods: Having noted preclinical activity of pazopanib in MTC, patients with advanced MTC who had disease progression within the preceding 6 months were accrued to this multi-institutional international Phase II clinical trial to assess tumor response rate (by RECIST criteria) and safety of pazopanib given orally once daily at 800 mg until disease progression or intolerability. Results: From September 22, 2008 to December 11, 2011, 35 individuals (80% males, median age 60 years) were enrolled. All patients have been fo...