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Gastrointestinal stromal tumors (GISTs) are highly responsive to inhibitors of the KIT proto-oncogene receptor tyrosine kinase and platelet-derived growth factor receptor α (PDGFRA). However, when resistance develops, there are no standard therapy options beyond imatinib, sunitinib, and regorafenib.
Investigators now report results of an industry-sponsored, multicenter, double-blind, placebo-controlled, randomized, phase III trial involving 129 patients with refractory GIST who were assigned 2:1 to receive ripretinib (an oral switch-control tyrosine kinase inhibitor that is active against KIT and PDGFRA mutations) or placebo. Of these patients, 55% to 59% were male, 41% to 47% had gastric primaries, 62% to 64% had received three prior therap…