An open-label pilot study of vemurafenib in previously treated metastatic melanoma patients with brain metastases.

Abstract

8548 Background: Melanoma is a common cause of cerebral metastases, and indicates a poor prognosis. The BRAF inhibitor vemurafenib (V; RG7204, PLX4032) has shown dramatic activity in Phase I and II studies in metastatic melanoma harboring V600-mutated BRAF. These studies excluded patients with active brain metastasis. The activity of V for brain metastases is unknown. Methods: Open label, single arm trial of V to evaluate the safety and efficacy of V in patients with metastatic melanoma with BRAF V600 mutations (by the cobas 4800 BRAF V600 Mutation Test) and non-resectable-brain mets, pretreated by radiotherapy and/or chemotherapy. Patients may have symptoms related to their brain mets and be on a stable dose of steroids. V is administered continuously at the dose of 960 mg BID. Twenty patients are planned to be include in this study. Staging (MRI brain and CT thorax/abdomen) is scheduled every 4 weeks in the first 2 months and then every 8 weeks. In addition, the serum S100 levels are determined. Results...