Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome

Study Overview
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Brief Summary
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The aim of this clinical study is to find out how well Patidegib Gel 2% works in preventing new basal cell carcinomas (BCCs) developing on the face of adults with Gorlin syndrome, and how safe Patidegib Gel 2% is to use. Participants will apply either Patidegib Gel 2% or a Vehicle Gel (with no active drug substance) to their face twice a day for a year (12 months). The number of new BCCs on the face will be compared between participants who used Patidegib Gel 2% or Vehicle Gel after 12 months.

Detailed Description
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This is a global, multicenter, randomized, double-blind, stratified, vehicle-controlled study of the efficacy and safety of Patidegib Gel 2%, applied topically twice daily to the face of adult participants with Gorlin syndrome. Participants will be randomized (1:1) to receive either Patidegib Gel 2% or Vehicle Gel for 12 months. The assignment of participants to the 2 groups will be stratified by sex assigned at birth, age (≥60 or <60 years), and number of facial BCC lesions (10-15, 16-30 or >30) at baseline. All BCCs at baseline, and any new BCCs which develop during the study, will be photographed and tracked during the study. New BCCs will be confirmed by dermoscopy analysis.

Official Title
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A Multicenter, Randomized, Double Blind, Vehicle-controlled, Phase 3 Efficacy and Safety Study of Patidegib Gel 2% for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects With Gorlin Syndrome

Conditions
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Gorlin Syndrome

Intervention / Treatment
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* Drug: Patidegib Topical Gel
* Drug: Patidegib Topical Gel with no active patidegib


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: The subject must be at least 18 years old at the Screening Visit. The subject must be confirmed to have a PTCH1 mutation. The subject must have at least 10 BCCs on the face at Randomization (Baseline/Day 1). The subject must be willing to abstain from application of any non-study topical medication (prescription or over the counter) to their facial skin for the duration of the trial, except as prescribed by the investigator. Key Exclusion Criteria: The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study Investigational Product (IP). The subject is known to have hypersensitivity to any of the ingredients in the IP formulation. The subject has uncontrolled systemic disease. The subject has been treated for invasive cancer within the past 5 years excluding chronic lymphocytic leukemia Stage 0 and non-melanoma skin cancer, Stage I cervical cancer, or ductal carcinoma in situ of the breast. Previous Hedgehog inhibitor therapy was considered to have failed for reasons other than unfavorable side effect(s).

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Patidegib Gel 2%<br>Patidegib Gel 2% (w/w), applied topically to the face twice daily for 12 months | Drug: Patidegib Topical Gel<br>* Patidegib Gel 2% is a smooth, clear, colorless-to-yellow, viscous, hydro-alcoholic gel for topical administration.<br>* Other names: SGT-610;|
| Placebo Comparator: Vehicle Gel<br>Vehicle Gel, applied topically to the face twice daily for 12 months | Drug: Patidegib Topical Gel with no active patidegib<br>* Vehicle Gel consists of the excipients of Patidegib Gel with no active patidegib. It is a smooth viscous gel for topical administration.<br>* Other names: Placebo;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of new BCCs on the face at Month 12 | New BCCs, confirmed through dermoscopy, at Month 12 after twice-daily treatment with either Patidegib Gel 2% or Vehicle Gel for 12 months | Month 12 |