Phase II Trial of First-line Axitinib plus Nivolumab in Patients with Advanced Mucosal Melanoma

Mucosal melanoma is a rare subtype of melanoma with inferior response rates and outcomes to immunotherapy. Vascular endothelial growth factor (VEGF) signaling may contribute to immune resistance and represents a potential therapeutic target. We evaluated nivolumab plus axitinib in untreated, advanced mucosal melanoma in a Western population and explored escalation to triplet strategies for PD-1-refractory disease.

In this single-center, investigator-initiated phase II study, patients with previously untreated unresectable or metastatic mucosal melanoma received nivolumab every 4 weeks and axitinib twice daily. The primary endpoint was objective response rate (ORR) per RECIST v1.1. A sequential phase Ib exploratory cohort evaluated treatment intensification with the addition of ipilimumab or stereotactic body radiotherapy (SBRT) at progression, with safety as the primary endpoint.