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

NCT ID: NCT06050122

Last Updated: 2025-10-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

140 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-03-17

Study Completion Date

2026-08-31

Brief Summary

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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

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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).

Conditions

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Gorlin Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

PREVENTION

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Patidegib Gel 2%

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

Group Type EXPERIMENTAL

Patidegib Topical Gel

Intervention Type DRUG

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

Vehicle Gel

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

Group Type PLACEBO_COMPARATOR

Patidegib Topical Gel with no active patidegib

Intervention Type DRUG

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

Interventions

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Patidegib Topical Gel

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

Intervention Type 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.

Intervention Type DRUG

Other Intervention Names

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Patidegib IPI-926 Saridegib SGT-610 Vehicle Gel Placebo

Eligibility Criteria

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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.

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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sol-Gel Technologies, Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Northwest Arkansas Clinical Trials Center

Rogers, Arkansas, United States

Site Status

Center for Dermatology Clinical Research

Fremont, California, United States

Site Status

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

Los Angeles, California, United States

Site Status

The Dermatology Center of Newport

Newport Beach, California, United States

Site Status

Stanford University - Lucille Packard's Children's Hospital

Redwood City, California, United States

Site Status

Yale University

New Haven, Connecticut, United States

Site Status

Dermatology Associates of Tallahassee

Tallahassee, Florida, United States

Site Status

University of South Florida Health

Tampa, Florida, United States

Site Status

The University of Chicago Medicine

Chicago, Illinois, United States

Site Status

The Johns Hopkins Hospital

Baltimore, Maryland, United States

Site Status

University of Michigan Health - Michigan Medicine - University Hospital

Ann Arbor, Michigan, United States

Site Status

Minnesota Clinical Study Center

New Brighton, Minnesota, United States

Site Status

MediSearch Clinical Trials

Saint Joseph, Missouri, United States

Site Status

Schweiger Dermatology P.C.

Verona, New Jersey, United States

Site Status

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Site Status

Duke University Health System

Durham, North Carolina, United States

Site Status

Apex Clinical Research Center

Canton, Ohio, United States

Site Status

Cleveland Clinic - Main Campus

Cleveland, Ohio, United States

Site Status

Apex Clinical Research Center

Mayfield Heights, Ohio, United States

Site Status

SSM Health Dermatology

Oklahoma City, Oklahoma, United States

Site Status

Oregon Health and Science University

Portland, Oregon, United States

Site Status

Penn State Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

North Texas Center for Clinical Research

Frisco, Texas, United States

Site Status

The University of Texas MD Anderson Cancer Center

Houston, Texas, United States

Site Status

Hopital Avicenne

Bobigny, , France

Site Status

Centre Hospitalier Universitaire De Nantes

Nantes, , France

Site Status

Centre Hospitalier Lyon Sud

Pierre-Bénite, , France

Site Status

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

Munich, Bavaria, Germany

Site Status

Charité Universitätsmedizin Berlin - Campus Charité Mitte

Berlin, , Germany

Site Status

DRK Gemeinnützige Krankenhaus GmbH Sachsen

Chemnitz, , Germany

Site Status

ASST Spedali Civili di Brescia

Brescia, , Italy

Site Status

AOU Maggiore della Carita

Novara, , Italy

Site Status

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome, , Italy

Site Status

Maastricht University Medical Center

Maastricht, , Netherlands

Site Status

Hospital Clinic Barcelona

Barcelona, , Spain

Site Status

Hospital de la Santa Creu i Sant Pau

Barcelona, , Spain

Site Status

Hospital Universitartio 12 De Octubre

Madrid, , Spain

Site Status

The University of Birmingham

Birmingham, , United Kingdom

Site Status

Barts Health NHS Trust - The Royal London Hospital

London, , United Kingdom

Site Status

Northern Care Alliance NHS Foundation Trust

Salford, , United Kingdom

Site Status

Countries

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United States France Germany Italy Netherlands Spain United Kingdom

Other Identifiers

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2023-507528-21-00

Identifier Type: OTHER

Identifier Source: secondary_id

SGT-610-01

Identifier Type: -

Identifier Source: org_study_id

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