NCT02384460

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

A Phase 3, Multi-center, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of SD-101 Cream in Patients With Epidermolysis Bullosa

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 169 (actual)
• Sponsor: Scioderm, Inc. · Industry
• Sex: All
• Age: 1 Month
• Healthy volunteers: Not accepted

Summary

The aim was to assess the efficacy and safety of SD-101-6.0 cream versus Placebo (SD-101-0.0) cream in the treatment of skin lesions in participants with Epidermolysis Bullosa. Funding Source - United States Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD).

Detailed description

This was a Phase 3, multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy and safety of SD-101-6.0 cream versus placebo (SD-101-0.0) cream on skin lesions in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz Epidermolysis Bullosa. Epidermolysis Bullosa is a rare group of inherited disorders that typically manifest at birth as blistering and lesion formation on the skin in response to little or no apparent trauma. In this study, SD-101-6.0 cream or placebo (SD-101-0.0) cream was to be applied topically, once a day to the entire body for a period of 90 days. Participants had 1 target wound selected at baseline by the investigator. The selected target wound was required to have been present for at least 21 days. Photographic confirmation of the target wound location was collected at baseline, and the picture saved from the first visit was used to confirm location of the target wound at subsequent visits. The participant returned to the study site for Visit 2 (approximately 14 days from baseline), Visit 3 (approximately 30 days from baseline), Visit 4 (approximately 60 days from baseline), and Visit 5 (approximately 90 days from baseline) to have the target wound assessed for the level of healing. In addition, itching, pain, body surface area, target wound closure, and scarring of healed target wound were assessed at each visit. The ARANZ SilhouetteStar™ was used to measure the target wound at all visits.

Conditions

• Epidermolysis Bullosa

Interventions

Type	Name	Description
DRUG	SD-101-6.0 cream	applied topically once a day for 90 days
DRUG	Placebo (SD-101-0.0) cream	applied topically once a day for 90 days

Timeline

• Start date: 2015-03-11
• Primary completion: 2017-07-05
• Completion: 2017-07-05
• First posted: 2015-03-10
• Last updated: 2020-04-09
• Results posted: 2018-10-16

Locations

38 sites across 13 countries: United States, Australia, Austria, France, Germany, Israel, Italy, Lithuania, Netherlands, Poland, Serbia, Spain, United Kingdom