An Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas in Patients With Non-Gorlin High Frequency BCC

Study Overview
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Brief Summary
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This is a multicenter, randomized, double-blind, stratified, vehicle-controlled study of the efficacy and safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult participants with non-Gorlin HF-BCC (high-frequency basal cell carcinoma). Participants will be randomized (1:1) to receive either Patidegib Topical Gel, 2%, or Vehicle for 9 months. Randomization will be stratified by gender. The primary endpoint is the number of nSEB (surgically eligible basal cell carcinoma) that develop on the face over the 9 month period. The primary end point will be assessed by imaging and tracking of BCCs consistently throughout the study in order to identify nSEBs.

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

Conditions
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Recurrent Basal Cell Carcinoma

Intervention / Treatment
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* Drug: Patidegib Topical Gel, 2%
* Drug: Patidegib Topical Gel, Vehicle


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: The subject must have had at least 10 (with at least 3 on the face) clinically typical BCCs present within 24 months prior to Screening and Randomization (Baseline/Day 1). Additionally, the subject must have at least 2 BCCs with longest diameter <5 mm present on the face prior to Screening and Randomization (Baseline/Day 1). The subject must be willing to abstain from application of a non-study topical medication (prescription or over the counter) to facial skin for the duration of the trial except as prescribed by the Investigator. Exclusion Criteria: The subject has been previously diagnosed with Gorlin syndrome On medical history, family history or clinical examination there is a suspicion that the patient has Gorlin syndrome. Patients with a family history of medulloblastoma The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study IP. The subject has uncontrolled systemic disease. The subject has been treated for invasive cancer within the past 5 years excluding non-melanoma skin cancer, Stage I cervical cancer, ductal carcinoma in situ of the breast, or chronic lymphocytic leukemia (CLL) Stage 0.

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: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Participants will be randomized (1:1) to receive either Patidegib Topical Gel, 2%, or Vehicle for 9 months. Randomization will be stratified by gender.
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Patidegib Topical Gel, 2%<br>Participants will be randomized (1:1) to receive Patidegib Topical Gel, 2% for 9 months | Drug: Patidegib Topical Gel, 2%<br>* Patidegib Topical Gel, 2%<br>* Other names: IP;|
| Active Comparator: Patidegib Topical Gel, Vehicle<br>Participants will be randomized (1:1) to receive Patidegib Topical Gel, Vehicle for 9 months | Drug: Patidegib Topical Gel, Vehicle<br>* Patidegib Topical Gel, Vehicle<br>* Other names: IP, Vehicle;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of new surgically eligible BCCs (nSEBs) |  | Baseline through Month 9 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of treatment emergent adverse events assessed with means and standard errors or proportions |  | Baseline through Month 9 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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High Frequency Basal Cell Carcinoma, non-gorlin, HF-BCC, Bazex-Dupré-Christol Syndrome, Rombo Syndrome, Oley Syndrome, Xeroderma Pigmentosum (XP) Syndrome, BCC, SEB, Hedgehog inhibitor, Basal Cell Nevus Syndrome, Basal Cell Carcinoma, Surgically Eligible Basal Cell Carcinoma, patidegib, HFBCC, HHI