Basal Cell CancerBCC-SonidegibNCT07068568
Dose-Modulated Sonidegib Therapy for Periocular Basal Cell Carcinoma
An observational study of Basal Cell Cancer and Eyelid Tumor, run by Federico II University at 1 site in Italy.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Basal Cell Cancer, Eyelid Tumor, Basal Cell Carcinoma (BCC)
- What is being tested?
- Sonidegib
- Type of treatment
- Pill or oral medicine
- How many people
- 12 participants
- Where
- 1 siteItaly
- Study dates
- 11 Jan 2021to 20 Mar 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Dose Modulation Strategy of Sonidegib for Eye-Sparing Treatment of Locally Advanced Periocular Basal Cell Carcinoma
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?
Read more
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.
Read the detailed description
This monocentric, open-label, real-world study investigated the clinical efficacy and tolerability of Sonidegib, a selective Hedgehog pathway inhibitor, in the treatment of locally advanced periocular basal cell carcinoma (laBCC). The study specifically focused on patients for whom standard surgical or radiotherapy options were contraindicated due to the risk of vision loss, functional impairment, or unacceptable cosmetic outcomes.
A total of 12 adult patients with histologically confirmed periocular laBCC were enrolled at the Oculoplasty Unit of the University of Naples Federico II. Inclusion criteria targeted individuals with tumors in functionally or aesthetically sensitive locations-such as the eyelid margins, medial/lateral canthi, or deep orbital tissues-where conventional excision would require extensive, disfiguring surgery. All patients received oral Sonidegib 200 mg daily as first-line systemic therapy.
A dose-modulation strategy was implemented in response to adverse effects or individual tolerance, allowing temporary drug interruption or reduced-frequency administration. This personalized therapeutic approach aimed to preserve treatment adherence and quality of life while maintaining tumor control.
Patients were monitored through multidisciplinary follow-up visits at regular intervals, including dermatologic evaluation, photographic tumor documentation, and ophthalmologic assessments such as best-corrected visual acuity (BCVA), slit-lamp biomicroscopy, and fundus examination. Tumor response was assessed using clinical measurements and standardized criteria adapted for periocular anatomy.
The primary endpoint was the proportion of patients achieving disease control (complete response, partial response, or stable disease) without requiring exenteration or vision-threatening surgery. Secondary endpoints included time to response, duration of therapy, adverse event frequency and severity (graded per CTCAE), patient-reported functional and cosmetic outcomes, and post-treatment recurrence rates.
This study offers preliminary real-world evidence that Sonidegib, when administered with a flexible dosing strategy, can achieve significant therapeutic responses while minimizing toxicity in patients with complex periocular laBCC. The results support its role as an eye-sparing, function-preserving alternative to surgery in selected cases.
- Conditions
- Basal Cell Cancer, Eyelid Tumor, Basal Cell Carcinoma (BCC), Basal Cell Carcinoma of the Head and Neck
- Being tested
- Sonidegib
- Keywords
- eyelid basal cell carcinoma, sonidegib
- Medical subject headings
- Neoplasms, Basal Cell, Eyelid Neoplasms, Carcinoma, Basal Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SonidegibDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Sonidegib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From baseline to end of follow-up (ranging from 12 to 33 months)
Secondary outcomes (1)
Preservation of Visual Function and Ocular Structure
This secondary outcome measures the proportion of patients maintaining functional vision and anatomical integrity of the eye during and after Sonidegib treatment. Evaluations included best-corrected visual acuity (BCVA), intraocular pressure, and slit-lamp and fundus examinations performed at baseline and at follow-up visits. Structural preservation was defined as avoidance of orbital invasion, enucleation, or significant deformation of periocular tissues. Changes in ocular function were documented and graded by the ophthalmology team to assess any Sonidegib-related ocular toxicity or surgery-preventing benefit.
Measured: From treatment initiation through the final follow-up visit (12 to 33 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jul 2025
- Study start11 Jan 2021
- Primary completion20 Dec 2023
- Study completion20 Mar 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Basal Cell CancerStudies of the same condition, open and under way.
- More trials run by Federico II UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Federico II University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Diego Strianese, Federico II University
- Investigators
- Diego Strianese, MD, PhD, Principal investigator, Federico II University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07068568
- Sponsor's protocol ID
- AOU Federico II n. 2025/040325
- Acronym
- BCC-Sonidegib
- Status last verified
- Jun 2025
- First submitted
- 7 Jul 2025
- Last updated
- 16 Jul 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Dose Modulation Strategy of Sonidegib for Eye-Sparing Treatment of Locally Advanced Periocular Basal Cell Carcinoma
- ClinicalTrials.gov ID
- NCT07068568
- Acronym / sponsor ID
- BCC-Sonidegib / AOU Federico II n. 2025/040325
- Conditions
- Basal Cell Cancer, Eyelid Tumor, Basal Cell Carcinoma (BCC), Basal Cell Carcinoma of the Head and Neck
- Keywords
- eyelid basal cell carcinoma, sonidegib
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Federico II UniversityUniversity, hospital or other
- Study start
- 11 Jan 2021 (actual)
- Primary completion
- 20 Dec 2023 (actual)
- Study completion
- 20 Mar 2024 (actual)
- First posted
- 16 Jul 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This monocentric, open-label, real-world study investigated the clinical efficacy and tolerability of Sonidegib, a selective Hedgehog pathway inhibitor, in the treatment of locally advanced periocular basal cell carcinoma (laBCC). The study specifically focused on patients for whom standard surgical or radiotherapy options were contraindicated due to the risk of vision loss, functional impairment, or unacceptable cosmetic outcomes.
A total of 12 adult patients with histologically confirmed periocular laBCC were enrolled at the Oculoplasty Unit of the University of Naples Federico II. Inclusion criteria targeted individuals with tumors in functionally or aesthetically sensitive locations-such as the eyelid margins, medial/lateral canthi, or deep orbital tissues-where conventional excision would require extensive, disfiguring surgery. All patients received oral Sonidegib 200 mg daily as first-line systemic therapy.
A dose-modulation strategy was implemented in response to adverse effects or individual tolerance, allowing temporary drug interruption or reduced-frequency administration. This personalized therapeutic approach aimed to preserve treatment adherence and quality of life while maintaining tumor control.
Patients were monitored through multidisciplinary follow-up visits at regular intervals, including dermatologic evaluation, photographic tumor documentation, and ophthalmologic assessments such as best-corrected visual acuity (BCVA), slit-lamp biomicroscopy, and fundus examination. Tumor response was assessed using clinical measurements and standardized criteria adapted for periocular anatomy.
The primary endpoint was the proportion of patients achieving disease control (complete response, partial response, or stable disease) without requiring exenteration or vision-threatening surgery. Secondary endpoints included time to response, duration of therapy, adverse event frequency and severity (graded per CTCAE), patient-reported functional and cosmetic outcomes, and post-treatment recurrence rates.
This study offers preliminary real-world evidence that Sonidegib, when administered with a flexible dosing strategy, can achieve significant therapeutic responses while minimizing toxicity in patients with complex periocular laBCC. The results support its role as an eye-sparing, function-preserving alternative to surgery in selected cases.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sonidegib-Treated laBCC Patients | – | Drug: SonidegibThis 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SonidegibSonidegib 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. | Drug | Sonidegib-Treated laBCC Patients |
Primary outcome
Tumor Control Rate
Time frame: From baseline to end of follow-up (ranging from 12 to 33 months)
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.
1 secondary outcome
Preservation of Visual Function and Ocular Structure
Time frame: From treatment initiation through the final follow-up visit (12 to 33 months)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Diego Strianese, MD, PhD principal investigator
Federico II University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Basal Cell Cancer and Eyelid Tumor, run by Federico II University at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Tumor Control Rate (time frame: From baseline to end of follow-up (ranging from 12 to 33 months)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2025.