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Randomized double-blind phase II study of regorafenib in patients with metastatic osteosarcoma

Journal of Clinical Oncology Apr 28, 2019

Davis LE, et al. - In this phase 2 trial (SARC024), researchers assessed the efficacy of regorafenib in specific sarcoma subtypes, including advanced osteosarcoma, to determine whether it can offer improved progression-free survival (PFS). Participants in this study included 42 patients from 12 centers who received ≥ 1 prior line of therapy but still had progressive metastatic osteosarcoma with measurable disease by RECIST. The primary end point was PFS, which was powered to detect a difference of ≥ 3 months in median PFS. Regorafenib offered significantly improved median PFS vs placebo: 3.6 months vs 1.7 months, respectively. Thus, the primary end point was met. In patients with progressive metastatic osteosarcoma, the activity of regorafenib demonstrated no new safety signals. Based on these findings, researchers recommended considering regorafenib for treating patients with relapsed metastatic osteosarcoma.

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