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

To join this study, you need to be an adult, at least 18 years old. You must have been diagnosed with Gorlin syndrome and have a specific genetic change (called a PTCH1 mutation) confirmed by a doctor. You also need to have quite a few BCCs on your face – at least 10 – when you start the study. It's important that you're willing to only use the study gel on your face and avoid other skin creams or treatments, unless your study doctor says they are okay.

There are also some reasons why you wouldn't be able to join. For example, if you've recently used strong skin treatments on your face or other medications that might interfere with the study's results. If you're allergic to anything in the gel, or have another serious health condition that isn't under control, you wouldn't be able to participate. Also, if you've had certain types of cancer in the last five years, or if a previous similar treatment for Gorlin syndrome didn't work for you, you might not be eligible.

If you join the study, you'll be given either the active Patidegib gel or a 'dummy' gel. You won't know which one you're receiving, and neither will your study doctor. You'll need to apply the gel to your face twice a day every day for 12 months. Throughout this year, you'll have regular visits to the clinic where study staff will check your skin for any new spots, photograph your face, and assess how the gel is working. They will also ask about any side effects you might be experiencing and perform some health checks. It's important to attend all appointments so the researchers can track your progress and safety. There will be ongoing checks of your skin to record any new BCCs that appear.