Gorlin syndrome below the surface.

Gorlin syndrome is a rare genetic condition that causes mutations in a tumor-suppressor gene. It is characterized by a wide variety of developmental issues and a predisposition to developing certain forms of cancer, particularly a type of skin cancer known as basal cell carcinoma (BCC). Individuals with Gorlin syndrome can develop hundreds of BCCs in their lifetime, especially on the face and sun-exposed areas.

Gorlin syndrome can be frustrating to live with as it currently has no cure and only severe treatment options. But right now, research is underway on a topical gel (applied to the skin) that might reduce the disease burden of basal cell carcinomas from Gorlin syndrome on your face. And you may be able to take part.

About the study.

We are looking for adults 18 years of age and older who have been diagnosed with Gorlin syndrome, to take part in this clinical research study. The purpose of the study is to determine the safety and effectiveness of an investigational topical gel for the reduction of disease burden of facial basal cell carcinomas in people living with Gorlin syndrome.

Individuals will be evaluated to determine their eligibility to participate in this study. Each participant who qualifies will receive a topical gel; half of participants will receive the investigational topical gel containing the active ingredient, and the other half will receive a placebo gel that does not contain the active ingredient. All participants in the study will receive study-related medical exams, and study-related laboratory tests at no cost. Compensation for travel may also be available.

Study participation.

This study consists of a 45-day screening period, a 12-month treatment period, and a 30-day follow-up period. If you are eligible and choose to participate, you will be asked to attend 14 study visits and have one scheduled phone call during this 14-month time frame. Over the course of the study, the study doctor will perform a number of tests and assessments, including digital imaging to monitor your BCCs and biopsies of facial BCCs.

Who may qualify.

The Pelle-926-301 Study may be appropriate for those who meet the following criteria:

Are 18 years of age and older
Have been diagnosed with Gorlin syndrome
Have 10 or more clinically typical basal cell carcinomas present within the 24 months before Randomization – at least three of these BCCs must be on the face