Gorlin Syndrome Alliance Hosts Discussion About Patidegib Clinical Trial

Gorlin Syndrome Alliance Hosts Discussion About Patidegib Clinical Trial

Dr. Sean Christensen, Assistant Professor of Dermatologic Surgery at Yale University School of Medicine, joined Julie Breneiser, president of the Gorlin Syndrome Alliance, patient, and mother of two affected children, for a live discussion about the Patidegib Clinical Trial involving a topical hedgehog inhibitor. If you were unable to join in on the conversation, you can watch the video on demand below. 


Some of our viewers had questions following the discussion, so we reached out to our contacts and received the following answers for you.

Question: I have an upcoming dermatologist appointment on September 7th.  He only finds skin cancers on my face or shoulders. I’m very interested in the trial and want to see if I qualify. But should I go forth with my Sept appointment if I don’t have the screening by then?

Please schedule a screening visit with the nearest site and check for eligibility into the trial. In regards to the September visit, the patient should consult and follow the advice of their dermatologist.

Question: Are all of the sites college/university/teaching hospitals?

Not all the sites are college/university/teaching hospitals.

Question: How does a topical cream get to the P1? Is this just a preventative cream for the time used on that spot, since there are no margins with BCCNS. As a genetic disease, just as happens with surgery, BCCS grow back in the same areas.

This is a topical hedgehog inhibitor which is in the phase 3 clinical trial currently. In this study, patients apply the topical gel twice daily on the face not on individual spots. The drug is in experimental phase and efficacy/safety is not determined.

Question: Is genetic testing part of the trial?


Question: With the travel reimbursements, do we need to go to the closest site, or can we go anywhere? I almost think I’d rather fly direct 1 hour or so to Philadelphia or somewhere than drive 2.5 hr in my car to Cleveland for visits if they are only going to be 1-2 hours

Patients can go to any site.

Question: What quantity is considered “High-dose” Nicotinamide and why a 3 month wait?

High dose Nicotinamide is based on PI’s discretion. A 3 month wait is to remove an effect of Nicotinamide on the study medication.

Question: Why did they go with the 2% and stop the 4%?

Both 2% and 4% were evaluated in a phase 2 study. 2% was chosen to study in a larger phase 3 study based on efficacy and safety data.