The first patient has enrolled in Castle Creek Pharmaceuticals (CCP) Phase 2/3 DELIVERS clinical trial evaluating the safety and efficacy of diacerein 1% ointment (CCP-020) for the treatment of epidermolysis bullosa simplex (EBS).
CCP-020 is engineered to block an important inflammatory signaling pathway associated with EBS, which could strengthen epidermal tissue and support healing. In a previously completed Phase 2 clinical trial that included 17 patients with EBS, patients treated with CCP-020 showed an average 60 percent reduction in blistering after four weeks.
The randomized, double-blind, parallel group DELIVERS study is designed to enroll about 80 participants who have a clinical and laboratory confirmed diagnosis of EBS at research sites in the U.S., Europe, Israel and Australia. Researchers will compare the safety and efficacy of CCP-020 to a control ointment when applied once-daily for eight weeks. Participants will report outcomes including pruritus, pain, and mobility week-to-week using an electronic diary.
“Currently there are no treatment options that target the underlying cause of EBS, the most common form of a family of rare conditions leading to extremely fragile skin," says Amy Paller, MD, director of the Northwestern University Skin Disease Research Center and lead investigator on the DELIVERS study, in a news release. "The topical formulation of diacerein 1% that we are investigating is potentially disease-modifying, which would represent an important advance in treatment for patients." The National Institutes of Health (NIH) estimates EBS affects one in 30,000 to 50,000 people.
"Generalized EBS can be quite debilitating from infancy into adulthood because many patients experience open, blistering wounds that need frequent bandage changes and present a risk of infection," says Brett Kopelan, executive director of the nonprofit organization debra of America. "There is no treatment or cure. Pain management, wound care, and protective bandaging are the only treatment options. Supportive care, often by multiple providers including physicians, nurses, nutritionists, and psychological counselors, is all these patients have available right now. We are excited that Castle Creek is addressing EBS, which is the most common type of epidermolysis bullosa but is often the most underserved in terms of research into a treatment. We look forward to working hand-in-hand to educate the population about this trial."
The Dystrophic Epidermolysis Bullosa Research Association of America (debra of America) is the only U.S. nonprofit providing all-inclusive support to the EB Community, through funding research for a cure and by providing free programs and services for those with EB.