Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults with Gorlin Syndrome
- Conditions
- Gorlin Syndrome
- Interventions
- 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).
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 140
- 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.
Key
- 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.
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
- Secondary Outcome Measures
Name Time Method
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
Harvard Medical School - Mass Gen (MGH) - Cancer Center - Center for Pediatric Hematology-Oncology
🇺🇸Boston, Massachusetts, United States
University of Michigan Health - Michigan Medicine - University Hospital
🇺🇸Ann Arbor, Michigan, United States
Minnesota Clinical Study Center
🇺🇸New Brighton, Minnesota, United States
MediSearch Clinical Trials
🇺🇸St Joseph, Missouri, United States
Memorial Sloan Kettering Cancer Center
🇺🇸New York, New York, United States
Duke University Health System
🇺🇸Durham, North Carolina, United States
Apex Clinical Research Center
🇺🇸Mayfield Heights, Ohio, United States
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
🇮🇹Rome, Italy
Schweiger Dermatology P.C.
🇺🇸Verona, New Jersey, United States
Cleveland Clinic - Main Campus
🇺🇸Cleveland, Ohio, United States
The University of Texas MD Anderson Cancer Center
🇺🇸Houston, Texas, United States
Centre Hospitalier Lyon Sud
🇫🇷Pierre-Bénite, France
Charité Universitätsmedizin Berlin - Campus Charité Mitte
🇩🇪Berlin, Germany
Hospital Clinic Barcelona
🇪🇸Barcelona, Spain
SSM Health Dermatology
🇺🇸Oklahoma City, Oklahoma, United States
Oregon Health and Science University
🇺🇸Portland, Oregon, United States
Penn State Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
North Texas Center for Clinical Research
🇺🇸Frisco, Texas, United States
Mercy Health System
🇺🇸Lake Geneva, Wisconsin, United States
Hopital Avicenne
🇫🇷Bobigny, France
Centre Hospitalier Universitaire De Nantes
🇫🇷Nantes, France
Ludwig-Maximilians-Universitaet Muenchen (LMU) - Klinik und Poliklinik fuer Dermatologie und Allergologie
🇩🇪Muenchen, Bayern, Germany
DRK Gemeinnützige Krankenhaus GmbH Sachsen
🇩🇪Chemnitz, Germany
ASST Spedali Civili di Brescia
🇮🇹Brescia, Italy
AOU Maggiore della Carita
🇮🇹Novara, Italy
Maastricht University Medical Center
🇳🇱Maastricht, Netherlands
Dermoklinika Centrum Medyczne
🇵🇱Łódź, Poland
Hospital de la Santa Creu i Sant Pau
🇪🇸Barcelona, Spain
Hospital Universitartio 12 De Octubre
🇪🇸Madrid, Spain
The University of Birmingham
🇬🇧Birmingham, United Kingdom
Barts Health NHS Trust - The Royal London Hospital
🇬🇧London, United Kingdom
Northern Care Alliance NHS Foundation Trust
🇬🇧Salford, United Kingdom