MIKIE: A randomized, double-blind, regimen-controlled, phase II, multicenter study to assess the efficacy and safety of two different vismodegib regimens in patients with multiple basal cell carcinomas.

TPS9121 Background: Vismodegib, a Hedgehog signaling pathway inhibitor, is FDA and EMA approved for patients (pts) with advanced basal cell carcinoma (BCC) who are not candidates for surgery or radiotherapy. While vismodegib reduces BCC tumor burden and blocks growth of new BCCs in pts with basal cell carcinoma nevus syndrome (BCCNS), further studies are required to optimize the therapeutic index of long-term (chronic) therapy. The MIKIE study (NCT01815840) assesses the efficacy and safety of 2 intermittent vismodegib dosing regimens in pts with multiple BCCs. Methods: MIKIE is a randomized, double-blind, regimen-controlled phase II study, in adults with multiple BCCs (including BCCNS) ≥18 years with ≥1 histopathologically confirmed BCC and ≥6 clinically evident BCCs (at least three ≥5 mm in diameter). 200 pts are planned to be randomized (1:1) to regimen A or B and stratified by BCCNS status, geographic region, and immunosuppression status. In regimen A, pts receive 3 courses of vismodegib (150 mg/d) for...