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

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).

Timeline

Start

2015-03-11

Primary completion

2017-07-05

Completion

2017-07-05

Outcome

Missed primary endpoint

registry p = 0.985; Hazard Ratio (HR) 1.004 (95% CI 0.651 to 1.549) NCT02384460 ↗

registry p = 0.39; Odds Ratio (OR) 0.733 (95% CI 0.365 to 1.474) NCT02384460 ↗

Publications

• Murrell DF, Paller AS, Bodemer C, Browning J, Nikolic M, Barth JA, Lagast H, Krusinska E, Reha A; ESSENCE Study Group. Wound closure in epidermolysis bullosa: data from the vehicle arm of the phase 3 ESSENCE Study. Orphanet J Rare Dis. 2020 Jul 21;15(1):190. doi: 10.1186/s13023-020-01435-3.
• Paller AS, Browning J, Nikolic M, Bodemer C, Murrell DF, Lenon W, Krusinska E, Reha A, Lagast H, Barth JA; ESSENCE Study Group. Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study). Orphanet J Rare Dis. 2020 Jun 23;15(1):158. doi: 10.1186/s13023-020-01419-3.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	allantoin	Small molecule	6 %	Topical

Indications