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

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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Gorlin Syndrome
• Intervention / Treatment: Drug: Patidegib Topical Gel; Drug: Patidegib Topical Gel with no active patidegib

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 Type: Interventional
• Enrollment (Estimated): 140
• Phase: Phase 3

Contacts and Locations

Study Locations

• France
  • Bobigny, France
    • Hôpital Avicenne
  • Nantes, France
    • Centre Hospitalier Universitaire de Nantes
  • Pierre-Bénite, France
    • Centre Hospitalier Lyon Sud
• Germany
  • Berlin, Germany
    • Charité Universitätsmedizin Berlin - Campus Charité Mitte
  • Chemnitz, Germany
    • DRK Gemeinnützige Krankenhaus GmbH Sachsen
  • Bavaria
    • Munich, Bavaria, Germany
      • Ludwig-Maximilians-Universitaet Muenchen (LMU) - Klinik und Poliklinik fuer Dermatologie und Allergologie
• Italy
  • Brescia, Italy
    • ASST Spedali Civili di Brescia
  • Novara, Italy
    • Aou Maggiore Della Carita
  • Rome, Italy
    • Fondazione Policlinico Universitario Agostino Gemelli IRCCS
• Netherlands
  • Maastricht, Netherlands
    • Maastricht University Medical Center
• Spain
  • Barcelona, Spain
    • Hospital de la Santa Creu i Sant Pau
  • Barcelona, Spain
    • Hospital Clinic Barcelona
  • Madrid, Spain
    • Hospital Universitartio 12 De Octubre
• United Kingdom
  • Birmingham, United Kingdom
    • The University of Birmingham
  • London, United Kingdom
    • Barts Health NHS Trust - The Royal London Hospital
  • Salford, United Kingdom
    • Northern Care Alliance NHS Foundation Trust
• United States
  • Arkansas
    • Rogers, Arkansas, United States, 72758
      • Northwest Arkansas Clinical Trials Center
  • California
    • Fremont, California, United States, 94538
      • Center for Dermatology Clinical Research
    • Los Angeles, California, United States, 90024
      • University of California Los Angeles (UCLA) - Center for Inflammatory Bowel Diseases - Westwood Location
    • Newport Beach, California, United States, 92660
      • The Dermatology Center of Newport
    • Redwood City, California, United States, 94063
      • Stanford University - Lucille Packard's Children's Hospital
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Yale University
  • Florida
    • Tallahassee, Florida, United States, 32308
      • Dermatology Associates of Tallahassee
    • Tampa, Florida, United States, 33612
      • University of South Florida Health
  • Illinois
    • Chicago, Illinois, United States, 60637
      • The University of Chicago Medicine
  • Maryland
    • Baltimore, Maryland, United States, 21287-0006
      • The Johns Hopkins Hospital
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan Health - Michigan Medicine - University Hospital
  • Minnesota
    • New Brighton, Minnesota, United States, 55112
      • Minnesota Clinical Study Center
  • Missouri
    • Saint Joseph, Missouri, United States, 64506
      • MediSearch Clinical Trials
  • New Jersey
    • Verona, New Jersey, United States, 07044
      • Schweiger Dermatology P.C.
  • New York
    • New York, New York, United States, 10065
      • Memorial Sloan Kettering Cancer Center
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Health System
  • Ohio
    • Canton, Ohio, United States, 44718
      • Apex Clinical Research Center
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic - Main Campus
    • Mayfield Heights, Ohio, United States, 44124
      • Apex Clinical Research Center
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73102
      • SSM Health Dermatology
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health and Science University
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Penn State Hershey Medical Center
  • Texas
    • Frisco, Texas, United States, 75034
      • North Texas Center for Clinical Research
    • Houston, Texas, United States, 77030
      • The University of Texas MD Anderson Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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, 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 Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Patidegib Gel 2%; Patidegib Gel 2% (w/w), applied topically to the face twice daily for 12 months	Drug: Patidegib Topical Gel; Patidegib Gel 2% is a smooth, clear, colorless-to-yellow, viscous, hydro-alcoholic gel for topical administration.; Other Names: IPI-926; Patidegib; Saridegib; SGT-610
Placebo Comparator: Vehicle Gel; Vehicle Gel, applied topically to the face twice daily for 12 months	Drug: 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.; Other Names: Placebo; Vehicle Gel

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of new BCCs on the face at 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	Month 12

Collaborators and Investigators

• Sponsor: Sol-Gel Technologies, Ltd.
• Collaborators: Premier Research Group plc

Study record dates

Study Major Dates

• Study Start (Actual): March 17, 2024
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): August 1, 2026

Study Registration Dates

• First Submitted: September 15, 2023
• First Submitted That Met QC Criteria: September 15, 2023
• First Posted (Actual): September 22, 2023

Study Record Updates

• Last Update Posted (Estimated): October 14, 2025
• Last Update Submitted That Met QC Criteria: October 12, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Bone Diseases; Musculoskeletal Diseases; Stomatognathic Diseases; Neoplasms; Genetic Diseases, Inborn; Jaw Diseases; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Carcinoma; Congenital Abnormalities; Abnormalities, Multiple; Cysts; Neoplastic Syndromes, Hereditary; Bone Diseases, Developmental; Neoplasms, Basal Cell; Odontogenic Cysts; Jaw Cysts; Bone Cysts; Carcinoma, Basal Cell; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Basal Cell Nevus Syndrome; Substandard Drugs; Pharmaceutical Preparations; IPI-926
• Other Study ID Numbers: SGT-610-01; 2023-507528-21-00 (Other Identifier: EU CT)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No