Conditions: Epidermolysis Bullosa
Keywords: Epidermolysis Bullosa Simplex Recessive Dystrophic Junctional non-Herlitz Amicus Therapeutics Scioderm, Inc. SD-101 6% SD-101-6.0 Allantoin 6% SD-101 3% SD-101-3.0 Allantoin 3% Zorblisa
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 48
Healthy volunteers: no

Brief summary:
The purpose of this study was to assess whether the topical use of SD-101 cream (3% or 6%) was effective in treating wounds in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz Epidermolysis Bullosa (EB).

Detailed description:
This was a Phase 2b, multi-center, randomized, double-blind, vehicle-controlled study to assess the efficacy and safety of SD-101 dermal cream (3% or 6%) on skin lesions in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.

SD-101 cream \[containing 3% or 6% or vehicle (0%)\] was applied topically, once a day to the entire body for a period of 90 days. Eligible participants had a target wound assessed at baseline. Selected target wound had to be at least a certain age and within a prespecified size range at study entry. 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 at Week 2, Month 1, Month 4, and Month 5 (at 14, 30, 60, and 90 days, respectively) to have the target wound previously identified at baseline re-assessed for the level of healing. In addition, changes in itching, pain, body surface area coverage of blisters and lesions, and scaring of the healed target wound were also assessed at each visit.

Arms:
arm 1: SD-101 dermal cream (6%) applied topically once daily over the entire body for 90 days. arm 2: SD-101 dermal cream (3%) applied topically once daily over the entire body for 90 days. arm 3: Vehicle dermal cream (SD-101 0%) applied topically once daily over the entire body for 90 days.

Interventions: intervention 1: SD-101 dermal cream (3%) intervention 2: SD-101 Dermal Cream (6%) intervention 3: Vehicle (SD-101 0%)
Intervention descriptions: intervention 1: SD-101 is a white, crystalline powder that is formulated within an odorless, soft, white cream base. SD-101-3.0 cream contains allantoin, a diureide glyoxylic acid, at a concentration of 3% and other excipients. intervention 2: SD-101 is a white, crystalline powder that is formulated within an odorless, soft, white cream base. SD-101-6.0 cream contains allantoin, a diureide glyoxylic acid, at a concentration of 6% and other excipients. intervention 3: A white, crystalline powder that is formulated within an odorless, soft, white cream base. It contains no allantoin, only excipients.

Protocol document: