A gastrointestinal stromal tumor (GIST) is a tumor in the gastrointestinal tract, most commonly in the small intestine or stomach. Patients with GIST experienced reduced resistance against KIT and PDGFRA inhibitors. This study aims to examine the efficacy and safety of ripretinib, a switch-control tyrosine kinase inhibitor, in patients with advanced GIST.
This randomized, placebo-controlled, double-blind, phase-3 study included a total of 129 patients aged 18 years or older with advanced GIST with progression or tolerance to imatinib, sunitinib, and/or regorafenib. The patients were randomly assigned in a 2:1 ratio to receive oral ripretinib 150 mg once daily (ripretinib group, n=85) or placebo once daily (placebo group, n=44). The primary outcome of the study was progression-free survival, assessed by the blinded independent central review (BICR).
During a median follow-up of 6.3 months in the ripretinib group, 51 patients had progression-free survival events, as compared with 37 patients in the placebo group during a median follow-up of 1.6 months. The median progression-free survival during the double-blind period was 6.3 months in the ripretinib group and 1.o months in the placebo group.
The research concluded that ripretinib significantly improved the median progression-free survival time in patients with advanced GIST, as compared with placebo.