Trial Evaluating Diacerein 1% Ointment as EBS Treatment Enrolls First Patient

The first patient has enrolled in a clinical trial evaluating both the efficacy and safety of diacerein 1% ointment (CCP-020) in treating epidermolysis bullosa simplex (EBS), Castle Creek Pharmaceuticals recently announced. The company’s Phase 2/3 DELIVERS clinical trial (NCT03154333) is currently recruiting participants.

CCP-020 is a small molecule, engineered to target and block caspase-1 (CASP1) and interleukin-1 (IL-1) beta —  two key factors mediating inflammation associated with EBS. By lessening the inflammatory process underlying the disease, CCP-020 may contribute to strengthening epidermal tissue and support healing.

“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,” Amy Paller, MD, director of the Northwestern University Skin Disease Research Center and DELIVERS lead researcher, said in a press 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 Phase 2/3 DELIVERS trial is expected to enroll 80 patients with a confirmed diagnosis of EBS from multiple locations in the U.S., Europe, Israel, and Australia.

Researchers will evaluate the safety and efficacy of CCP-020 compared with a control ointment when applied once a day for a total of eight weeks. Patients will register their outcomes, including pain, pruritus (severe itching), and mobility on a weekly basis using an electronic diary.

“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,” said 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,”

“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,” Kopelan added.

A previous Phase 2 clinical trial showed that EBS patients treated with CCP-020 had an average blistering reduction of 60 percent after four weeks of treatment.

“At Castle Creek we are focused on addressing complex health problems that often affect smaller, underserved and highly specialized patient communities,” said Greg Licholai, MD, president and chief scientific officer at Castle Creek Pharmaceuticals. “As we continue the development of CCP-020, we have an opportunity to fulfill our mission with a new treatment option for people living with EBS and the potential to significantly improve standard of care and patient health and quality of life.”