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The 12-month analysis from Basal Cell Carcinoma Outcomes with LDE225 Treatment (BOLT): A phase II, randomized, double-blind study of sonidegib in patients with advanced basal cell carcinoma JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY Dummer, R., Guminski, A., Gutzmer, R., Dirix, L., Lewis, K. D., Combemale, P., Herd, R. M., Kaatz, M., Loquai, C., Stratigos, A. J., Schulze, H., Plummer, R., Gogov, S., Pallaud, C., Yi, T., Mone, M., Chang, A. S., Cornelis, F., Kudchadkar, R., Trefzer, U., Lear, J. T., Sellami, D., Migden, M. R. 2016; 75 (1): 113-+


The hedgehog pathway inhibitor sonidegib demonstrated meaningful tumor shrinkage in more than 90% of patients with locally advanced basal cell carcinoma (BCC) or metastatic BCC in the BCC Outcomes with LDE225 Treatment study.This report provides long-term follow-up data collected up to 12 months after the last patient was randomized.In this multicenter, randomized, double-blind phase II study, patients were randomized 1:2 to sonidegib 200 or 800 mg. The primary end point was objective response rate assessed by central review.Objective response rates in the 200- and 800-mg arms were 57.6% and 43.8% in locally advanced BCC and 7.7% and 17.4% in metastatic BCC, respectively. Among the 94 patients with locally advanced BCC who responded, only 18 progressed or died and more than 50% had responses lasting longer than 6 months. In addition, 4 of 5 responders with metastatic BCC maintained an objective response. Grade 3/4 adverse events and those leading to discontinuation were less frequent with sonidegib 200 versus 800 mg (38.0% vs 59.3%; 27.8% vs 37.3%, respectively).No placebo or comparator arms were used because sonidegib demonstrated efficacy in advanced BCC in a phase I study, and the hedgehog pathway inhibitor vismodegib was not yet approved.With longer follow-up, sonidegib demonstrated sustained tumor responses in patients with advanced BCC.

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