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

Purpose

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.

Condition

  • Gorlin Syndrome

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Prevention
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
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:
    • Patidegib
    • IPI-926
    • 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:
    • Vehicle Gel
    • Placebo

Recruiting Locations

Harvard Medical School - Mass Gen (MGH) - Cancer Center - Center for Pediatric Hematology-Oncology
Boston, Massachusetts 02114
Contact:
Patient Referral Service - United States
+1 984 275 0103

More Details

Status
Recruiting
Sponsor
Sol-Gel Technologies, Ltd.

Study Contact

Patient Referral Support Service (US toll-free number)
984-275-0103
clinicalinfo@sol-gel.com

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).