Infigratinib in patients with advanced cholangiocarcinoma with <i>FGFR2</i> gene fusions/translocations: the PROOF 301 trial
Shalini Makawita, Ghassan K. Abou‐Alfa, Sameek Roychowdhury, Saeed Sadeghi, Ivan Borbath, Lipika Goyal, Allen Lee Cohn, Angela Lamarca, Do‐Youn Oh, Teresa Macarulla, Rachna T. Shroff, Michael F. Howland, Ai Li, Terry Cho, Amit Pande, Milind Javle
Abstract
Cholangiocarcinoma is an aggressive malignancy with poor overall survival. Approximately 15% of intrahepatic cholangiocarcinomas contain FGFR alterations. Infigratinib is an oral FGFR 1–3 kinase inhibitor. Favorable results from a Phase II trial of infigratinib in advanced/metastatic FGFR-altered cholangiocarcinomas has led to its further investigation in the front-line setting. In this article we describe the design, objectives and rationale for PROOF 301, a Phase III multicenter, open label, randomized trial of infigratinib in comparison to standard of care gemcitabine and cisplatin in advanced/metastatic cholangiocarcinoma with FGFR2 translocations. The results of this study have the potential to define a new role for a chemotherapy-free, targeted therapy option in the front-line setting for these patients. Clinical Trial Registration: NCT03773302 (ClincalTrials.gov)