Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at UCLA
Dates
study started
completion around

Description

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.

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

Details

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).

Keywords

Gorlin Syndrome, Basal Cell Carcinoma, Basal Cell Nevus Syndrome, Syndrome, Veratrum Alkaloids, Patidegib Topical Gel, Patidegib Topical Gel with no active patidegib, Patidegib Gel 2%, Vehicle Gel

Eligibility

You can join if…

Open to people ages 18 years and up

  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.

You CAN'T join if...

  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.

Locations

  • University of California Los Angeles (UCLA) - Center for Inflammatory Bowel Diseases - Westwood Location accepting new patients
    Los Angeles California 90024 United States
  • The Dermatology Center of Newport accepting new patients
    Newport Beach California 92660 United States
  • Stanford University - Lucille Packard's Children's Hospital accepting new patients
    Redwood City California 94063 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Sol-Gel Technologies, Ltd.
ID
NCT06050122
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 140 study participants
Last Updated