The first patient has been enrolled in a new Phase 1 study evaluating the pharmacokinetics (PK) and safety of Castle Creek Pharmaceuticals’ diacerein 1% ointment (CCP-020) under maximum use conditions in patients with epidermolysis bullosa (EB) at the UNC-Chapel Hill Dermatology & Skin Cancer Center.

Diacerein is a slow-acting, small molecular weight compound of the class anthraquinone with potent anti-inflammatory properties. It is believed to block an inflammatory signaling pathway associated with the most common subtype, EB simplex, resulting in strengthening epidermal tissue and healing of skin blisters.

The open-label multi-center, global Phase 1 study is expected to enroll 16-20 people living with EB age 6 months and older in Europe and age 4 years and older within the U.S. Patients who enroll in the study will be treated with CCP-020 for 10 days and will be asked to provide blood samples while receiving CCP-020. Patients who participate in the PK study may be eligible to enroll in a separate open-label extension study, where they will receive CCP-020 for two additional treatment cycles.

“There has been no treatment available and most of the EB community is excited to participate in anything that will give researchers knowledge of the disease as a whole,” says Dr. Aída Lugo-Somolinos, principal investigator at UNC-Chapel Hill, in a news release. “People with localized EB lesions on the hands or feet, or generalized lesions on arms, legs, and torso are encouraged to enroll in this study as long as the lesions cover more than 2 percent of the total body surface area.” Dr Lugo-Somolinos adds, "Not only evaluating efficacy is important but also we have a responsibility to find a product that is safe to use in open skin. To evaluate safety, a shorter but very important PK study is enrolling patients right now.”

In addition to the PK study, the company is currently evaluating CCP-020 in the ongoing DELIVERS study for the treatment of patients with EB simplex. CCP-020 is an investigational drug believed to block an inflammatory signaling pathway associated with EBS resulting in strengthening epidermal tissue and healing of skin blisters. 

“We remain committed to supporting people who are impacted by EB through research that can lead to insights on the disease and innovative treatment approaches,” says Amir Tavakkol, PhD, executive vice president and chief development officer at Castle Creek Pharmaceuticals, in a news release. “By collecting pharmacokinetic data and profiling drug absorption and safety and tolerability, this Phase 1 study will play an integral role in the continued development of CCP-020.” 

For more information about the Phase 1 study, including a list of participating sites and their contact information, visit https://clinicaltrials.gov/ct2/show/NCT03472287.