Phase II study of cixutumumab (IMC-A12, NSC742460; C) in hepatocellular carcinoma (HCC).

4043 Background: C is an anti-IGF-1R monoclonal antibody. IGF-IR is implicated in hepatic carcinogenesis. A phase I study of C yielded a partial response in HCC. Methods: Patients (pts) with advanced HCC, Child-Pugh A-B8, and KPS ≥ 60%, received 6 mg/kg of C weekly. The trial had a planned accrual of 50 pts in a two-stage design. Primary endpoints were PFS at 4 months (futility 42%) and response rate (RR), by RECIST (futility 5%). IGF-1R staining was evaluated by immunohistochemistry (IHC), and serum free IGF-I, total IGF-II and IGFBP-1 and -3 were measured by ELISA (Beckman Coulter/DSL) at baseline, and studied for correlation with PFS, OS, and stable disease (SD). Child-Pugh, CLIP, and GETCH scores were similarly measured and studied. Results: The study accrued to the first stage only because of futility. N= 24 with no prior systemic therapy including no sorafenib: median age 67.5 years (range 49-83), KPS 80%(70-90%), 20 males (83%), 9 stage III (37%)/15 stage IV (63%), 11 HBV (46%) /10 HCV (42%), 10 (4...