Clinical trial NCT06050122: Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults with Gorlin Syndrome
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Gorlin Syndrome
Lead sponsor: Sol-Gel Technologies, Ltd.
Sponsor class: INDUSTRY
First posted: 2023-09-22
Enrollment: 140
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

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).

Eligibility criteria: 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.

Primary outcomes:
measure: Number of new BCCs on the face at Month 12
timeFrame: Month 12
Secondary outcomes:
Other outcomes:
Locations:
city: Rogers
country: United States
facility: Northwest Arkansas Clinical Trials Center
state: Arkansas
city: Fremont
country: United States
facility: Center for Dermatology Clinical Research
state: California
city: Los Angeles
country: United States
facility: University of California Los Angeles (UCLA) - Center for Inflammatory Bowel Diseases - Westwood Location
state: California
city: Newport Beach
country: United States
facility: The Dermatology Center of Newport
state: California
city: Redwood City
country: United States
facility: Stanford University - Lucille Packard's Children's Hospital
state: California
city: New Haven
country: United States
facility: Yale University
state: Connecticut
city: Tallahassee
country: United States
facility: Dermatology Associates of Tallahassee
state: Florida
city: Tampa
country: United States
facility: University of South Florida Health
state: Florida
city: Chicago
country: United States
facility: The University of Chicago Medicine
state: Illinois
city: Baltimore
country: United States
facility: The Johns Hopkins Hospital
state: Maryland
city: Boston
country: United States
facility: Harvard Medical School - Mass Gen (MGH) - Cancer Center - Center for Pediatric Hematology-Oncology
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Health - Michigan Medicine - University Hospital
state: Michigan
city: New Brighton
country: United States
facility: Minnesota Clinical Study Center
state: Minnesota
city: St Joseph
country: United States
facility: MediSearch Clinical Trials
state: Missouri
city: Verona
country: United States
facility: Schweiger Dermatology P.C.
state: New Jersey
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Durham
country: United States
facility: Duke University Health System
state: North Carolina
city: Canton
country: United States
facility: Apex Clinical Research Center
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic - Main Campus
state: Ohio
city: Mayfield Heights
country: United States
facility: Apex Clinical Research Center
state: Ohio
city: Oklahoma City
country: United States
facility: SSM Health Dermatology
state: Oklahoma
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Hershey
country: United States
facility: Penn State Hershey Medical Center
state: Pennsylvania
city: Frisco
country: United States
facility: North Texas Center for Clinical Research
state: Texas
city: Houston
country: United States
facility: The University of Texas MD Anderson Cancer Center
state: Texas
city: Lake Geneva
country: United States
facility: Mercy Health System
state: Wisconsin
city: Bobigny
country: France
facility: Hopital Avicenne
city: Nantes
country: France
facility: Centre Hospitalier Universitaire De Nantes
city: Pierre-Bénite
country: France
facility: Centre Hospitalier Lyon Sud
city: Muenchen
country: Germany
facility: Ludwig-Maximilians-Universitaet Muenchen (LMU) - Klinik und Poliklinik fuer Dermatologie und Allergologie
state: Bayern
city: Berlin
country: Germany
facility: Charité Universitätsmedizin Berlin - Campus Charité Mitte
city: Chemnitz
country: Germany
facility: DRK Gemeinnützige Krankenhaus GmbH Sachsen
city: Brescia
country: Italy
facility: ASST Spedali Civili di Brescia
city: Novara
country: Italy
facility: AOU Maggiore della Carita
city: Rome
country: Italy
facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
city: Maastricht
country: Netherlands
facility: Maastricht University Medical Center
city: Łódź
country: Poland
facility: Dermoklinika Centrum Medyczne
city: Barcelona
country: Spain
facility: Hospital Clinic Barcelona
city: Barcelona
country: Spain
facility: Hospital de la Santa Creu i Sant Pau
city: Madrid
country: Spain
facility: Hospital Universitartio 12 De Octubre
city: Birmingham
country: United Kingdom
facility: The University of Birmingham
city: London
country: United Kingdom
facility: Barts Health NHS Trust - The Royal London Hospital
city: Salford
country: United Kingdom
facility: Northern Care Alliance NHS Foundation Trust
Study officials:
References: