Dose-Modulated Sonidegib Therapy for Periocular Basal Cell Carcinoma

NCT07068568 | Completed FDA Drug

• 1 other identifier: AOU Federico II n. 2025/040325
• Study Type: observational
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical trial is to evaluate the safety, tolerability, and clinical outcomes of a dose-modulation strategy of Sonidegib in adult patients with locally advanced periocular basal cell carcinoma (laBCC) who are not candidates for surgery or radiotherapy. The main questions it aims to answer are:

• Can dose-adjusted Sonidegib treatment preserve ocular function and avoid disfiguring surgery in laBCC patients?
• What is the rate of tumor response, recurrence, and adverse events during dose-modulated treatment? Researchers will compare different dosing regimens based on treatment response, tolerability, and toxicity profiles to assess whether intermittent dosing or dose reductions can maintain clinical benefits while minimizing adverse effects. This monocentric retrospective observational real-world evidence (RWE) study included 12 patients (8 male, 4 female; aged between 67 and 92 years; mean 83.25 years) with histologically confirmed periocular laBCC (staged T3-T4b) treated with sonidegib between 2021-2023 at the Oculoplasty Unit of the University of Naples Federico II. Eligible patients were those for whom conventional surgery or radiotherapy would result in loss of vision or unacceptable cosmetic outcomes. Participants:
• Took Sonidegib orally, starting at 200 mg daily, with possible dose interruptions or reductions based on individual tolerance and tumor response.
• Underwent clinical and ophthalmologic evaluations every 4-6 weeks.
• Had imaging and dermatologic assessments to monitor tumor regression.
• Completed follow-up visits after treatment completion to assess long-term control and safety. The results of this study aim to support an eye-sparing, patient-centered approach for complex periocular tumors.

Conditions

Basal Cell Cancer; Eyelid Tumor; Basal Cell Carcinoma (BCC); Basal Cell Carcinoma of the Head and Neck

Keywords

eyelid basal cell carcinoma; sonidegib

Outcome Measures

Primary Outcomes (1)

• Tumor Control Rate

  The primary outcome is the proportion of patients who achieved disease control, defined as complete response, partial response, or stable disease without requiring disfiguring surgery or orbital exenteration. Tumor response was assessed clinically and photographically during dermatologic evaluations, in combination with ophthalmologic monitoring for visual function preservation. Classification was based on established dermatologic and ophthalmologic criteria adapted to periocular anatomy. Treatment tolerability and the need for dose modulation were also recorded as part of the outcome analysis.

  From baseline to end of follow-up (ranging from 12 to 33 months)

Secondary Outcomes (1)

• Preservation of Visual Function and Ocular Structure

  From treatment initiation through the final follow-up visit (12 to 33 months)

Study Arms (1)

Sonidegib-Treated laBCC Patients

This cohort includes adult patients diagnosed with locally advanced periocular basal cell carcinoma (laBCC) who were treated with oral Sonidegib (200 mg daily) at the University of Naples Federico II. All patients were considered unsuitable for surgical excision or radiotherapy due to the location or extent of the tumor, where standard approaches posed risks of vision loss or disfigurement. A personalized dose modulation strategy was applied based on treatment tolerance and clinical response, including temporary discontinuation or adjustment of dosing frequency. Patients were followed with regular dermatologic and ophthalmologic assessments to evaluate tumor response, functional outcomes, adverse events, and recurrence rates. The cohort reflects real-world clinical practice in managing complex periocular laBCC.

Drug: Sonidegib

Interventions

Sonidegib DRUG

Sonidegib was administered orally at a dose of 200 mg once daily as per approved guidelines. The intervention was applied in a real-world clinical setting to patients with histologically confirmed, locally advanced periocular basal cell carcinoma (laBCC) deemed unsuitable for surgical resection or radiotherapy due to the tumor's extent or proximity to critical ocular structures. A flexible, individualized dose modulation strategy was implemented to manage adverse effects and optimize patient adherence. Modifications included temporary treatment interruptions or reduced dosing frequency, tailored to each patient's clinical status and tolerance. The goal of the intervention was to achieve local tumor control while preserving ocular structures, visual function, and cosmetic appearance. All patients received regular multidisciplinary follow-up, including ophthalmologic and dermatologic assessments.

Sonidegib-Treated laBCC Patients

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study population consisted of 12 adult patients with histologically confirmed, locally advanced periocular basal cell carcinoma (laBCC), all enrolled and treated at the Oculoplasty Unit of the University of Naples Federico II. All participants were treatment-naïve and presented with tumors unsuitable for surgical excision or radiotherapy due to the high risk of vision loss or anatomical disruption. The cohort included 8 males and 4 females, aged between 67 and 92 years, with a mean age of 83.25 years. The study was conducted in a real-world clinical setting and aimed to preserve ocular function and periocular anatomy through targeted oral therapy with Sonidegib.

You may qualify if:

• Adults (≥18 years) with histologically confirmed, locally advanced periocular basal cell carcinoma (laBCC)
• Tumor location or extent deemed unsuitable for surgical resection or radiotherapy due to high risk of orbital invasion, vision loss, or cosmetic disfigurement
• ECOG performance status 0-2
• Life expectancy ≥12 months
• Willing and able to comply with study procedures and follow-up assessments
• Provided written informed consent

You may not qualify if:

• Evidence of metastatic basal cell carcinoma
• Prior treatment with Hedgehog pathway inhibitors
• History of other malignancies within 5 years (except adequately treated non-melanoma skin cancer or carcinoma in situ)
• Severe hepatic or renal impairment
• Active systemic infection or uncontrolled medical conditions
• Pregnant or breastfeeding women
• Inability to undergo dermatologic or ophthalmologic evaluation due to physical or cognitive impairment

Sponsors & Collaborators

• Federico II University: lead

Study Sites (1)

Federico II University

Naples, Campania, 80131, Italy

Location

MeSH Terms

Conditions

Neoplasms, Basal Cell; Eyelid Neoplasms; Carcinoma, Basal Cell

Interventions

sonidegib

Condition Hierarchy (Ancestors)

Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Facial Neoplasms; Head and Neck Neoplasms; Neoplasms by Site; Eye Neoplasms; Eye Diseases; Eyelid Diseases; Carcinoma

Study Officials

• Diego Strianese, MD, PhD

  Federico II University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof

Study Record Dates

• First Submitted: July 7, 2025
• First Posted: July 16, 2025
• Study Start: January 11, 2021
• Primary Completion: December 20, 2023
• Study Completion: March 20, 2024
• Last Updated: July 16, 2025

Record last verified: 2025-06

Locations

IT (1)