Updated results of REGOMA: A randomized, multicenter, controlled open-label phase II clinical trial evaluating regorafenib in relapsed glioblastoma (GBM) patients (PTS).

2047Background: There is no established treatment regimen for recurrent GBM. GBMs have activation of multiple signaling pathways in the tumor microenvironment, including the receptor tyrosine kinases, VEGFR, FGFR, and PDGFR. REG, an oral multikinase inhibitor, inhibits these angiogenic kinases and the mutant oncogenic kinases KIT, RET, and B‐RAF. Methods: We present, after the first analysis, the updated results of REGOMA trial. The primary aim of this trial was to assess REG activity in prolonging overall survival (OS) in PTS with relapsed GBM after surgery and Stupp regimen (α = 0.2, 1-sided; β = 0.2). Secondary objectives were PFS, disease control rate (DCR), safety, quality of life (QoL); exploratory objectives included analysis of metabolic tissue biomarkers as possible predictors of response. PTS with histologically confirmed GBM, ECOG PS 0‐1, documented disease progression were randomized 1:1 to receive REG 160 mg/day (3 weeks on, 1 week off) or lomustine (LOM) 110 mg/m2 (every 6 weeks) until disea...