ESSENCE Study: Efficacy and Safety of SD-101 Cream in Patients With Epidermolysis Bullosa

• Time to complete target wound closure [ Time Frame: Within 3 months ]
  
• The proportion of patients experiencing complete closure of their target wound. [ Time Frame: Within 3 months ]
  

• Proportion of patients experiencing complete closure of their target wound. [ Time Frame: Within 2 months ]
  
• Proportion of patients experiencing complete closure of their target wound. [ Time Frame: Within 1 month ]
  
• Change in lesional skin [ Time Frame: Month 3 ]
  Based on BSAI at month 3, compared to baseline
  
  
• Change of Total Body Wound Burden based on BSAI [ Time Frame: Month 3 ]
  Assessed at month 3; compared to baseline
  
  
• Change in itching [ Time Frame: Day 7 ]
  Assessed at Day 7, compared to baseline
  
  
• Change in pain [ Time Frame: Day 7 ]
  Assessed at Day 7, compared to baseline
  
  

• Change of Total Body Wound Burden based on BSAI [ Time Frame: 2 weeks; months 1 and 2 ]
  Assessed at Week 2 and Months 1 and 2; compared to baseline
  
  
• Percent change from baseline of Total Body Wound Burden based on BSAI [ Time Frame: 2 weeks; Months 1, 2, and 3 ]
  Assessed at week 2 and Months 1, 2, and 3
  
  
• Change in lesional skin [ Time Frame: week 2; months 1 and 2 ]
  based on BSAI at Week 2 and Months 1 and 2, compared to baseline
  
  
• Percent change in lesional skin based on BSAI [ Time Frame: 2 weeks; Months 1, 2, and 3 ]
  Assessed at week 2 and Months 1, 2, and 3 compared to baseline
  
  
• Presence of scarring of healed target wound [ Time Frame: At visit where target wound has healed ]
  
• Change in target wound characteristics (i.e. Inflammation, blistering, granulation tissue, erythema, exudate) at week 2, Months 1, 2, and 3, compared to baseline [ Time Frame: 2 weeks; months 1, 2, and 3 ]
  
• Change in itching and pain [ Time Frame: Days 1 to 6; week 2; months 1, 2 and 3 ]
  Assessed at Days 1 to 6, Week 2 and Months 1, 2 and 3; compared to baseline
  
  
• Proportion of patients experiencing target wound closure within week 2 [ Time Frame: Week 2 ]
  

Epidermolysis Bullosa (EB) is a rare group of inherited disorders that typically manifest at birth as blistering and lesion formation on the skin and, in some cases, the epithelial lining of other organs, in response to little or no apparent trauma. In consequence, the skin is extremely fragile which can result in shearing of the skin, causing a high risk of infection. All forms of EB are both debilitating and life threatening. In some EB subtypes, high mortality occurs before the age of 1 (Junctional Herlitz), and others in adolescence to early adulthood, typically due to infection or failure to thrive. In addition, children surviving into their 20's and 30's are also at risk for development of a virulent form of squamous cell carcinoma, which is in many cases fatal.

There are no standard of care products available to treat the dermal manifestations of EB, and there is no approved drug for EB in either Europe or the United States. There have been numerous studies published on potential treatments for skin manifestations associated with EB, including vitamin E therapy, systemic phenytoin, topical nonsteroidal agents, cyproheptadine, tetracycline, and dapsone. No controlled studies showed clinical benefit of any therapy. Newer exploratory treatments including skin grafts, bioengineered skin products, and gene therapy have been unsuccessful to date.

This is a Phase 3, multi-center, randomized, double-blind, placebo-controlled, study to assess the efficacy and safety of SD-101-6.0 cream vs. placebo (SD-101-0.0) on lesions in patients with Simplex, Recessive Dystrophic, or Junctional non-Herlitz Epidermolysis Bullosa.

SD-101-6.0 cream or placebo (SD-101-0.0) will be applied topically, once a day to the entire body for a period of 90 days. Patients will have 1 target wound selected at baseline by the investigator. Selected target wound must be of at least a certain age. Photographic confirmation of the target wound location will be collected at baseline, and the picture saved from the first visit will be used to confirm location of the target wound at subsequent visits.

The patient will return to the study site for Visit 2 (14 days ±5 days from baseline), Visit 3 (30 days ±7 days from baseline), Visit 4 (60 days ±7 days from baseline), and Visit 5 (90 days ±7 days from baseline) to have the target wound, previously identified at baseline, re-assessed for the level of healing. In addition, itching, pain, body surface area (BSA), target wound closure, and scarring of healed target wound will also be assessed at each visit. The ARANZ SilhouetteStar™ will be used to measure the target wound at all visits.