Masitinib is safe and effective for the treatment of canine mast cell tumors

Background: Activation of the KIT receptor tyrosine kinase is associated with the development of canine mast cell tumors (MCT). Hypothesis/Objective: To evaluate the efficacy of masitinib, a potent and selective inhibitor of KIT, in the treatment of canine MCT. Animals: Two hundred and two client‐owned dogs with nonmetastatic recurrent or nonresectable grade II or III MCT. Methods: Double‐blind, randomized, placebo‐controlled phase III clinical trial. Dogs were administered masitinib (12.5 mg/kg/d PO) or a placebo. Time‐to‐tumor progression (TTP), overall survival, objective response at 6 months, and toxicity were assessed. Resulsts: Masitinib increased overall TTP compared with placebo from 75 to 118 days (P= .038). This effect was more pronounced when masitinib was used as first‐line therapy, with an increase in the median TTP from 75 to 253 days (P= .001) and regardless of whether the tumors expressed mutant (83 versus not reached P= .009]) or wild‐type KIT (66 versus 253 P= .008]). Masitinib was generally well tolerated, with mild (grade I) or moderate (grade II) diarrhea or vomiting as the most common adverse events. Conclusions and Clinical Importance: Masitinib is safe and effective at delaying tumor progression in dogs presenting with recurrent or nonresectable grade II or III nonmetastatic MCT.