The benefit-to-risk profile of 200mg sonidegib, a Hedgehog signaling inhibitor, might offer a new treatment option for patients with advanced basal cell carcinoma, a recent study published online first in the journal The Lancet Oncology has shown.
For the ongoing multicenter, double-blind, phase II BOLT trial, researchers enrolled 230 patients with locally advanced basal cell carcinoma who were not amenable to curative surgery or radiation or metastatic basal cell carcinoma. Participants were randomly assigned to either receive sonidegib 200mg or 800mg.
Results showed that during a median follow-up of 13.9 months, 36% (95% CI: 24-50) of those receiving sonidegib 200mg and 34% (95% CI: 25-43) of those receiving sonidegib 800mg achieved an objective response.
In regard to safety, the most common grade 3 to 4 adverse events were elevated creatine kinase and lipase concentration, and serious adverse events occurred in 14% of those the 200mg group and 30% of the 800mg group.
In addition, fewer adverse events resulting in dose interruption, dose reduction, or treatment discontinuation occurred in the 200mg group.
Patients with advanced basal cell carcinoma have limited treatment options. Hedgehog pathway signalling is aberrantly activated in around 95% of tumours. We assessed the antitumour activity of sonidegib, a Hedgehog signalling inhibitor, in patients with advanced basal cell carcinoma.