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

Phase 3

Recruiting

• Conditions: Gorlin Syndrome
• Interventions: Drug: Patidegib Topical Gel; Drug: Patidegib Topical Gel with no active patidegib

• Registration Number: NCT06050122
• Lead Sponsor: Sol-Gel Technologies, Ltd.

Brief Summary

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.

People who take part 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 those who used Patidegib Gel 2% or Vehicle Gel after 12 months.

Detailed Description

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 subjects with Gorlin syndrome.

Subjects will be randomized (1:1) to receive either Patidegib Gel 2% or Vehicle Gel for 12 months.

The assignment of subjects to the 2 groups will be stratified by sex assigned at birth, age (≥60 or \<60 years), and number of BCC lesions at the treatment area (face) (10-15, 16-30 or \>30) at Baseline.

All suspicious lesions will be imaged and tracked consistently throughout the study so that new BCCs that arise can be readily identified. New BCCs will be confirmed by dermoscopic analysis through the study. The BCC images will be reviewed and assessed by a Central Photo Review Board (CPRB).

Study Timeline

• Study First Submitted: 2023-09-15
• Study First Submitted QC: 2023-09-15
• Study First Posted: 2023-09-22
• Study Start: 2024-03-17
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-18
• Last Update Posted: 2025-03-21
• Primary Completion: 2025-12
• Study Completion: 2025-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 140

Inclusion Criteria

1. The subject must be at least 18 years old at the Screening Visit.
2. The subject must be confirmed to have a PTCH1 mutation.
3. The subject must have at least 10 BCCs on the face at Randomization (Baseline/Day 1).
4. 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

1. The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study Investigational Product (IP).
2. The subject is known to have hypersensitivity to any of the ingredients in the IP formulation.
3. The subject has uncontrolled systemic disease.
4. The subject has been treated for invasive cancer within the past 5 years excluding chronic lymphocytic leukemia Stage 0, non-melanoma skin cancer, successfully treated melanoma in situ and Stage I melanoma, Stage I cervical cancer, or ductal carcinoma in situ of the breast.
5. Inefficacy of previous Hedgehog inhibitor therapy.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Patidegib Gel 2%	Patidegib Topical Gel	Patidegib Gel 2% (w/w), applied topically to the face twice daily for 12 months
Vehicle Gel	Patidegib Topical Gel with no active patidegib	Vehicle Gel, applied topically to the face twice daily for 12 months

Primary Outcome Measures

Name	Time	Method
Number of new BCCs on the face at Month 12	Month 12	New BCCs at Month 12 after twice-daily treatment with either Patidegib Gel 2% or Vehicle Gel for 12 months, as assessed by the CPRB

Trial Locations

Locations (42)

Northwest Arkansas Clinical Trials Center; 🇺🇸 Rogers, Arkansas, United States

Center for Dermatology Clinical Research; 🇺🇸 Fremont, California, United States

University of California Los Angeles (UCLA) - Center for Inflammatory Bowel Diseases - Westwood Location; 🇺🇸 Los Angeles, California, United States

The Dermatology Center of Newport; 🇺🇸 Newport Beach, California, United States

Stanford University - Lucille Packard's Children's Hospital; 🇺🇸 Redwood City, California, United States

Yale University; 🇺🇸 New Haven, Connecticut, United States

Dermatology Associates of Tallahassee; 🇺🇸 Tallahassee, Florida, United States

University of South Florida Health; 🇺🇸 Tampa, Florida, United States

The University of Chicago Medicine; 🇺🇸 Chicago, Illinois, United States

The Johns Hopkins Hospital; 🇺🇸 Baltimore, Maryland, United States

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