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

NCT06050122 | Active Not Recruiting Phase 3 FDA Drug

• 2 other identifiers: SGT-610-012023-507528-21-00
• Study Type: interventional
• Target: 140
• Locations: 7 countries
• Sites: 40

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.

Conditions

Gorlin Syndrome

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (2)

Patidegib Gel 2%

EXPERIMENTAL

Patidegib Gel 2% (w/w), applied topically to the face twice daily for 12 months

Drug: Patidegib Topical Gel

Vehicle Gel

PLACEBO COMPARATOR

Vehicle Gel, applied topically to the face twice daily for 12 months

Drug: Patidegib Topical Gel with no active patidegib

Interventions

Patidegib Topical Gel DRUG

Patidegib Gel 2% is a smooth, clear, colorless-to-yellow, viscous, hydro-alcoholic gel for topical administration.

Also known as: Patidegib, IPI-926, Saridegib, SGT-610

Patidegib Gel 2%

Patidegib Topical Gel with no active patidegib DRUG

Vehicle Gel consists of the excipients of Patidegib Gel with no active patidegib. It is a smooth viscous gel for topical administration.

Also known as: Vehicle Gel, Placebo

Vehicle Gel

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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, 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.
• Inefficacy of previous Hedgehog inhibitor therapy.

Sponsors & Collaborators

• Sol-Gel Technologies, Ltd.: lead

Study Sites (40)

Northwest Arkansas Clinical Trials Center

Rogers, Arkansas, 72758, United States

Location

Center for Dermatology Clinical Research

Fremont, California, 94538, United States

Location

University of California Los Angeles (UCLA) - Center for Inflammatory Bowel Diseases - Westwood Location

Los Angeles, California, 90024, United States

Location

The Dermatology Center of Newport

Newport Beach, California, 92660, United States

Location

Stanford University - Lucille Packard's Children's Hospital

Redwood City, California, 94063, United States

Location

Yale University

New Haven, Connecticut, 06519, United States

Location

Dermatology Associates of Tallahassee

Tallahassee, Florida, 32308, United States

Location

University of South Florida Health

Tampa, Florida, 33612, United States

Location

The University of Chicago Medicine

Chicago, Illinois, 60637, United States

Location

The Johns Hopkins Hospital

Baltimore, Maryland, 21287-0006, United States

Location

University of Michigan Health - Michigan Medicine - University Hospital

Ann Arbor, Michigan, 48109, United States

Location

Minnesota Clinical Study Center

New Brighton, Minnesota, 55112, United States

Location

MediSearch Clinical Trials

Saint Joseph, Missouri, 64506, United States

Location

Schweiger Dermatology P.C.

Verona, New Jersey, 07044, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Duke University Health System

Durham, North Carolina, 27710, United States

Location

Apex Clinical Research Center

Canton, Ohio, 44718, United States

Location

Cleveland Clinic - Main Campus

Cleveland, Ohio, 44195, United States

Location

Apex Clinical Research Center

Mayfield Heights, Ohio, 44124, United States

Location

SSM Health Dermatology

Oklahoma City, Oklahoma, 73102, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Penn State Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

North Texas Center for Clinical Research

Frisco, Texas, 75034, United States

Location

The University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Hopital Avicenne

Bobigny, France

Location

Centre Hospitalier Universitaire De Nantes

Nantes, France

Location

Centre Hospitalier Lyon Sud

Pierre-Bénite, France

Location

Ludwig-Maximilians-Universitaet Muenchen (LMU) - Klinik und Poliklinik fuer Dermatologie und Allergologie

Munich, Bavaria, Germany

Location

Charité Universitätsmedizin Berlin - Campus Charité Mitte

Berlin, Germany

Location

DRK Gemeinnützige Krankenhaus GmbH Sachsen

Chemnitz, Germany

Location

ASST Spedali Civili di Brescia

Brescia, Italy

Location

AOU Maggiore della Carita

Novara, Italy

Location

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome, Italy

Location

Maastricht University Medical Center

Maastricht, Netherlands

Location

Hospital Clinic Barcelona

Barcelona, Spain

Location

Hospital de la Santa Creu i Sant Pau

Barcelona, Spain

Location

Hospital Universitartio 12 De Octubre

Madrid, Spain

Location

The University of Birmingham

Birmingham, United Kingdom

Location

Barts Health NHS Trust - The Royal London Hospital

London, United Kingdom

Location

Northern Care Alliance NHS Foundation Trust

Salford, United Kingdom

Location

MeSH Terms

Conditions

Basal Cell Nevus Syndrome

Interventions

IPI-926

Condition Hierarchy (Ancestors)

Odontogenic Cysts; Jaw Cysts; Bone Cysts; Cysts; Neoplasms; Carcinoma, Basal Cell; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms, Basal Cell; Neoplastic Syndromes, Hereditary; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Jaw Diseases; Stomatognathic Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 15, 2023
• First Posted: September 22, 2023
• Study Start: March 17, 2024
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): August 1, 2026
• Last Updated: October 14, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

DE (3); NL (1); FR (3); ES (3); GB (3); US (24); IT (3)