First Patient Dosed in Phase 3 Gorlin Syndrome Trial

According to a story from, the biopharmaceutical company PellePharm, Inc. recently announced that it has dosed the first patient in the company’s Phase 3 clinical trial. This trial is testing patidegib topic gel, which is being developed as a treatment for basal cell carcinoma, a form of skin cancer that commonly appears in patients with Gorlin syndrome. PellePharm specializes in the development of treatments for rare diseases affecting the skin.

About Gorlin Syndrome

Gorlin syndrome, which is also known as Nevoid basal-cell carcinoma syndrome, is a rare genetic medical condition which inflicts defects in various areas of the body. It is perhaps most characterized by the develop of multiple basal cell carcinoma tumors on the persons skin, which appears in around 90 percent of cases. The syndrome has been linked to mutations affecting the PTCH gene, which normally suppresses tumor growth. Other symptoms include odontogenic keratocyst, cardiac fibromas, deformations of the ribs and vertebrae, ovarian fibromas in women, skeletal abnormalities, intracranial calcification, and distinct facial features. The severity of symptoms can vary widely between patients. There are no disease modifying therapies for Gorlin syndrome, and treatment focuses on alleviating symptoms. To learn more about Gorlin syndrome, click here.

About The Trial

While basal cell carcinoma is usually not life threatening, the fact remains that, in severe cases, Gorlin syndrome patients can often face the appearance of many of these tumors at once. Some patients may have hundreds of carcinoma tumors in their lifetimes which can only be removed with surgeries that can leave scars and disfigurement. The goal of the introduction of patidegib is to provide an alternative treatment that can reduce a patient’s number of tumors and help get rid of them without surgery.

This trial will compared patidegib topical gel alongside a vehicle gel over a 12 month period. This gel is to be applied to the face two times each day during this period. The main endpoint will be the number of surgically eligible basal cell carcinomas that develop through the duration of treatment. This trial is continuing to recruit new participants and is expected to include a total of 150 patients with Gorlin syndrome.

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