SGT-610-01 (Gorlin Syndrome/Facial Basal Cell Carcinoma)

What is the Purpose of this Study?

We are doing this study to find out if an experimental drug called patidegib (the study drug) is a safe and effective option for people diagnosed with Gorlin syndrome. We want to know if it can help lower the number of basal cell carcinomas on the face.

What is the Condition Being Studied?

Facial Basal Cell Carcinomas in Gorlin Syndrome

Who Can Participate in the Study?

Adults ages 18+ who:

  • Are diagnosed with PTCH1 mutation
  • Have at least 10 basal cell carcinomas on the face

For more information, contact the study team at

Age Group

What is Involved?

This study is divided into 3 periods:

  • Screening period
  • Study drug period
  • Safety follow-up period

During the screening period, you will have the following tests and procedures done:

  • Physical exam
  • Blood draws
  • Skin exam

If you are eligible to join the study based on screening, you will proceed to the study drug period. This period will last up to 12 months. The study drug is a topical ointment that you will apply to your face 2 times a day and at least 6 hours apart. While you are using the study drug, you will visit our clinic 5 times for check-ups and have at least 8 phone calls with the study team.

After you stop taking the study drug, we will keep in touch with you for about a month to see how you are doing.

Study Details

Full 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
Principal Investigator
Associate Professor of Dermatology
Protocol Number
IRB: PRO00114454
NCT: NCT06050122
Phase III
Enrollment Status