We believe that the study name ESSENCE evokes the characteristics of endurance and dignity possessed by people living with EB. We see the essence of a person as his or her identity, that which persists and defines someone independently of the pain or health challenges that characterizes life with EB. The personalized, patient-focused approach we take at Amicus Therapeutics acknowledges individuality. Patients are the very essence of ESSENCE.
What is the ESSENCE Phase 3 Study?
The ESSENCE Study is a Phase 3 clinical trial of SD-101, an investigational topical medicine being developed by Amicus, together with Scioderm, to treat skin blistering and lesions associated with EB. The ESSENCE Study is currently underway in the U.S. and Europe, with sites planned in additional geographies.
The double-blind, placebo-controlled trial will evaluate the safety and efficacy of SD-101 in patients with various subtypes of EB (Simplex, Recessive Dystrophic and Junctional [non-Herlitz]).
Participants will be randomized 1:1 to two treatment groups receiving either SD-101 6% or placebo applied over their entire body once daily for three months.
The primary efficacy endpoint will be complete closure of a target wound. Improvement in itching, pain, and full-body wound and lesion coverage will also be assessed. Investigators will also assess safety.
It is expected that everyone completing the three-month study will be offered the option to receive active SD-101 in an open-label extension trial.