Gastrointestinal stromal tumors (GISTs) are mostly driven by c-kit or PDGFRA mutations, and commonly occur in the stomach and small intestine. Targeted therapy is the mainstay for advanced metastatic GISTs, but there is a lack of effective regimens after drug resistance. Immune checkpoint inhibitors (ICIs) have limited efficacy as monotherapy, while tyrosine kinase inhibitor (TKI) drugs such as regorafenib can improve the immune microenvironment and exert a synergistic effect when combined with ICIs, with more significant efficacy especially in cases with kit exon 17 mutations. This study aims to explore the effectiveness of regorafenib combined with envafolimab in metastatic gastrointestinal stromal tumors with kit exon 17 mutations that have failed standard treatment.
Aged ≥ 18 years, regardless of gender;
Confirmed as gastrointestinal stromal tumor by histopathological examination;
Having at least one measurable target lesion meeting the criteria of mRECIST v1.1 (non-lymph node lesions with a long axis ≥ 1.0 cm or a long axis ≥ the thickness of 2 slides); imaging assessment should be performed within 14 days before the first medication;
Disease progression or intolerance after treatment with imatinib, sunitinib, regorafenib, or ripretinib;
A history of intolerance to regorafenib treatment, or previous receipt of immune checkpoint inhibitor therapy;
Being in pregnancy or lactation;
Those with an expected survival period of less than 3 months;
Those who have undergone major surgery or suffered significant trauma within 4 weeks before the first blood collection during the screening period, or are expected to require major surgery during the study period;
Jian Li
Lead sponsor
Peking University Cancer Hospital & Institute
Sponsor institution