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

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.

Who Can Participate?

Eligibility

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 connor.whatley@duke.edu.

Age Range

18-110

Looking for Healthy Participants

What is Involved?

Description

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.

Locations

Duke University Hospital

Visit Timing

Weekdays

Compensation

Yes

Spanish Materials Available

Yes

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

Meenal

Kheterpal

Protocol Number

PRO00114454

NCT ID

NCT06050122

Phase

III

Enrollment Status

Open to Enrollment

ClinicalTrials.gov