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.

Dummer, Reinhard;Guminski, Alexander;Gutzmer, Ralf;Dirix, Luc;Migden, Michael R;et.al.
(2016) Journal of the American academy of dermatology — Vol. 75, n° 1, p. 113-125 (2016)

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Authors
  • Dummer, Reinhard
    Author
  • Guminski, Alexander
    Author
  • Gutzmer, Ralf
    Author
  • Dirix, Luc
    Author
  • Cornelis, FrankUCLouvain
    Author
  • Migden, Michael R
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Abstract
Background 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. Objective This report provides long-term follow-up data collected up to 12 months after the last patient was randomized. Methods 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. Results 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). Limitations 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. Conclusion With longer follow-up, sonidegib demonstrated sustained tumor responses in patients with advanced BCC
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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.-J., Plummer, R., Gogov, S., Pallaud, C., Yi, T., Mone, M., Chang, A. L. S., Cornelis, F., Kudchadkar, R., et al. (2016). 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, 75(1), 113-125. https://doi.org/10.1016/j.jaad.2016.02.1226 (Original work published 2016)