CompletedNot Applicable

Dose-Modulated Sonidegib Therapy for Periocular Basal Cell Carcinoma

Italy12 participantsStarted 2021-01-11

Plain-language 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.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Tumor Control Rate

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

Trial details

NCT IDNCT07068568

SponsorFederico II University

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2023-12-20

View on ClinicalTrials.gov More Basal Cell Cancer trials