Clinical trial · Observational
Dose-Modulated Sonidegib Therapy for Periocular Basal Cell Carcinoma
Dose Modulation Strategy of Sonidegib for Eye-Sparing Treatment of Locally Advanced Periocular Basal Cell Carcinoma
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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
Conditions (4)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Basal Cell Cancer | Skin Basal Cell Carcinoma | ALIAS | 0.90 |
| Basal Cell Carcinoma (BCC) | Basal Cell Carcinoma | ONTOLOGY_EXACT | 0.85 |
| Basal Cell Carcinoma of the Head and Neck | Basal Cell Neoplasm | PROBABILISTIC | 0.70 |
| Eyelid Tumor | Eyelid Neoplasm | ALIAS | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Sonidegib | Drug | Sonidegib | ALIAS |
Design
Arms and outcomes
Arms (1)
- label
- Sonidegib-Treated laBCC Patients
- description
- 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.
- interventionNames
- Drug: Sonidegib
Primary outcomes (1)
- measure
- Tumor Control Rate
- timeFrame
- From baseline to end of follow-up (ranging from 12 to 33 months)
- description
- 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.
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * 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 Exclusion Criteria: * 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
References
Publications (0)
Data not yet available