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

Gorlin Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06050122
• Other study ID #: SGT-610-01
• Secondary ID: 2023-507528-21-0
• Status: Recruiting
• Phase: Phase 3
• Start date: March 17, 2024
• Est. completion date: December 2025

Study information

• Verified date: April 2024
• Source: Sol-Gel Technologies, Ltd.
• Contact: Patient Referral Support Service
• Phone: +1 833 958 0014
• Email: clinicalinfo[@]sol-gel.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

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 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 140
• Est. completion date: December 2025
• Est. primary completion date: December 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Key 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 and non-melanoma skin cancer, Stage I cervical cancer, or ductal carcinoma in situ of the breast.

5. Previous Hedgehog inhibitor therapy was considered to have failed for reasons other than unfavorable side effect(s).

Related Conditions & MeSH terms

• Basal Cell Nevus Syndrome
• Carcinoma, Basal Cell
• Gorlin Syndrome
• Syndrome

Intervention

Drug:
• Patidegib Topical Gel
Patidegib Gel 2% is a smooth, clear, colorless-to-yellow, viscous, hydro-alcoholic gel for topical administration.
• Patidegib Topical Gel with no active patidegib
Vehicle Gel consists of the excipients of Patidegib Gel with no active patidegib. It is a smooth viscous gel for topical administration.

Locations

Country	Name	City	State
United States	Center for Dermatology Clinical Research	Fremont	California
United States	North Texas Center for Clinical Research	Frisco	Texas
United States	Mercy Health System	Lake Geneva	Wisconsin
United States	Apex Clinical Research Center	Mayfield Heights	Ohio
United States	Minnesota Clinical Study Center	New Brighton	Minnesota
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	Northwest Arkansas Clinical Trials Center	Rogers	Arkansas

Sponsors (2)

Lead Sponsor	Collaborator
Sol-Gel Technologies, Ltd.	Premier Research Group plc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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