CCP-020, or diacerein 1 percent ointment, is a treatment that Castle Creek Pharmaceuticals is developing for epidermolysis bullosa simplex (EBS).

A medication applied directly to the skin, it contains diacerein, a drug used to treat joint and skin inflammation. Diacerein is administered orally when used to treat other inflammatory diseases but is  incorporated into an ointment for the treatment of EBS.

How CCP-020 works

The distinctive feature of EBS is fragile, blister-prone skin.

Scientists know that inflammatory mediators, or proteins promoting inflammation, are activated in the skin cells of EBS patients. This suggests that inflammation plays an important role in the disease.

Diacerein is a small molecule that suppresses inflammation by blocking the inflammatory mediators interleukin-1B (IL-1B) and caspase-1 (CASP1). Inhibiting these proteins may decrease inflammation,  strengthening skin tissue and helping support healing.

CCP-020 in clinical trials

On March 6, 2017, Castle Creek announced the results of a Phase 2 clinical trial of CCP-20 as an EBS treatment. Seventeen patients had CCP-020 applied to their skin once a day for four weeks.

Blistering dropped by 60 percent in patients using CCP-20, versus 15 percent in those treated with a placebo. At the end of the three-month follow-up period, 67 percent of those in the placebo group returned to the blistering levels they had before the trial, compared with only 12.5 percent in the treatment group. The results suggested that CCP-20 could have long-term benefits in the treatment of EBS.  Patients also tolerated CCP-20 well, with no treatment-related adverse effects.

Castle Creek has also started a Phase 2/3 clinical trial (NCT03154333) to evaluate the safety and effectiveness of CCP-20 in EBS.

On June 5, 2017, it announced that it had enrolled the trial’s first patient. The goal is to enroll 80 people with EBS. Participants will be randomized to receive either CCP-20 or a placebo once a day for eight weeks.

Investigators will record and evaluate changes in itching, pain, and mobility during the study. The trial investigators are continuing to recruit participants.

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