Epidermolysis Bullosa (EB) refers to a group of rare conditions which are characterized by fragile skin. Basically, the disease is caused by separation between the layers of the skin. When EB patients experience injury, their wounds can become chronic, and blisters often develop. Other common complications include cardiomyopathy, anemia, dysphagia, osteoporosis, and syndactyly to name just a few. EB patients are also at risk for being diagnosed with squamous cell carcinoma. Some of the subtypes of EB include Junctional (JEB), Simplex (EBS), and Dystrophic (DEB). So far, 18 different genes have been identified as causing the condition.
Unfortunately, there are currently no FDA approved treatments for any of the EB subtypes, which in total, affect around 60,000 people in the United States and European Union. But, recent investigations have shown that inflammation plays an important role in this disease. This newfound information has opened a new potential door in the search for effective therapies.
There is now a Phase 2 clinical trial in the works, investigating a potential treatment which targets the inflammatory properties of EB.
The Phase II Trial
The organization responsible for conducting this trial is TWi Biotechnology. They began enrollment for the study in October of 2018 and after just three months they have announced completed enrollment, meaning they are able to move forward with the investigation.
This trial will be examining the tolerability, safety, and efficacy of AC-203 as a potential treatment for EB, EBS, JEB, and DEB. It is double-blind, multi-center, and placebo-controlled.
AC-203 is a topical form of diacerin which has proven effective in reducing inflammation in other conditions such as arthritis and diabetes. This drug has Orphan Drug Designation for EB and has already been approved (since the 1990s) for many chronic rheumatic diseases in Italy, Spain, France, and other countries.
This trial of AC-203 includes 9 participants who were randomized at two different trial sites. It will last 8 weeks, during which patients will receive topical treatment of AC-203 or placebo once daily. Following this period, patients will be monitored for a 4 week follow-up period. Efficacy will be measured by changes in parts of the skin that have faced injury. Erythema, blisters, curst, and erosion will be analyzed. Additionally, the patient’s pain and itchiness will be considered. Daily images of the injured areas will be documented by participants and these images will be utilized in the analysis.
Results from this trial should be reported within the second quarter of this year.
Full information of the trial can be found on ClinicalTrials.gov
You can read more about this trial, its speedy enrollment, and the potential of this treatment here.