AC-203-EBS-007
Research type
Research Study
Full title
An International, Multicenter, Randomized, Double-Blind, Parallel Group, Vehicle-Controlled, Phase 2/3 Study with Open-Label Extension Evaluating the Efficacy and Safety of Diacerein 1% Ointment for the Treatment of Generalized Epidermolysis Bullosa Simplex (EBS)
IRAS ID
1009244
Contact name
Anna Martinez
Contact email
Sponsor organisation
TWi Biotechnology, Inc
Clinicaltrials.gov Identifier
Research summary
Epidermolysis bullosa simplex (EBS) is a genetic skin disease caused by mutations in genes encoding specific structural proteins in the skin. EBS belongs a general class of inherited blistering skin diseases. The investigational drug to treat this condition is called AC-203 (study drug) which is a diacerein ointment. The most frequently reported side effect of oral diacerein was diarrhea with associated reports of loose stools and abdominal pain. This is due to the mild laxative effect of the active ingredient.
The main purpose of this study is to learn how well diacerein ointment works to reduce your signs and symptoms of EBS and how safe diacerein is compared with a vehicle ointment. A vehicle is an ointment that looks like the diacerein ointment and has the same composition as the diacerein ointment but does not contain diacerein.
Approximately 80-100 participants, aged 6 months and older will take part in this research study. This study will be conducted across 30 sites worldwide. The study is divided into Part A and Part B. The duration of study participation for Part A is anticipated to be approximately ~16 to 20 weeks per patient consisting of a Screening Period of up to 4 weeks, a Double-Blind Vehicle-Controlled Treatment Period of 8 weeks and a No Treatment Follow-up Period of 8 weeks. The Open-Label Extension Treatment Period for Part B will be 24 weeks.
In this study, we will evaluate the efficacy and safety of diacerein 1% ointment after 8 weeks of treatment in adult and paediatric patients (aged 6 months and older) with generalized EBS. In the open label extension, we will continue to evaluate the long-term safety of the study drug.
REC name
South Central - Berkshire Research Ethics Committee
REC reference
23/SC/0425
Date of REC Opinion
29 Jan 2024
REC opinion
Further Information Favourable Opinion