A prospective, single-arm, multicenter, phase IV study to evaluate the efficacy and safety of sonidegib in Chinese patients with locally advanced basal cell carcinoma.

Abstract

e21555 Background: Alterations in hedgehog signaling are implicated in the pathogenesis of basal-cell carcinoma. Sonidegib, a hedgehog pathway inhibitor (HPI), was approved for the treatment of adult patients with locally advanced basal cell carcinoma (BCC) that has recurred following surgery or radiation therapy, or those who are not candidates for surgery or radiation therapy, by the FDA and EMA. However, the efficacy and safety profile of sonidegib in Chinese patients was unknown. Herein, we present the results from a phase IV study of sonidegib conducted among Chinese patients. Methods: The study (NCT06880848) recruited patients (pts) aged ≥18 years who had locally advanced BCC that was not amenable to radiation therapy, curative surgery, or other local therapies. All patients received 200 mg oral sonidegib once daily for up to 1 year. The primary endpoint was the independent review committee (IRC)-assessed objective response rate (ORR) based on the modified response evaluation criteria in solid tumors (mRECIST). Results: Between June 21, 2023, and July 23, 2024, 160 pts were enrolled and treated. Median age was 68 years (range, 25 – 96 years). As of data cutoff (August 26, 2025), median follow-up was 12·7 months. The IRC-assessed ORR was 58.1% (95% CI 50.1-65.9%), which was consistent across all predefined subgroups. Median duration of response (DOR) and progression-free survival (PFS) by IRC were not reached; the 12-month DOR and PFS rates were 70.8% and 67.4%, respectively. Treatment-related adverse events (TRAEs) were reported in all patients, with most graded 1-2. The most common TRAEs were elevated blood creatine kinase (60.6%), alopecia (44.4%), muscle spasms (33.8%), weight decrease (29.4%), anorexia (24.4%), dysgeusia (23.1%), aspartate aminotransferase increased (21.3%), and alanine aminotransferase increased (20.0%). The incidence of grade ≥3 TRAEs was 28.1%, with elevated blood creatine kinase as the most common (15%). Serious adverse events (SAEs) occurred in 39 patients (24.4%), of which 17 (10.6%) were treatment-related SAEs. TRAEs led to treatment discontinuation in 9 (5.6%) pts. No TRAEs led to death. Conclusions: In Chinese patients with locally advanced BCC, sonidegib demonstrated robust efficacy, consistent with previous reports in global study populations. The safety profile of sonidegib was manageable, and no new safety signals were observed. Clinical trial information: NCT06880848 .