Last updated on July 2018

Safety and Efficacy of Diacerein 1% Ointment Topical Formulation Compared to Placebo for Subjects With Epidermolysis Bullosa Simplex (EBS)


Brief description of study

Epidermolysis bullosa simplex (EBS) is a rare genetic skin disease characterized by fragility of the skin and mucous membranes resulting in painful blisters and erosions after minor trauma. The simplex form is classified by skin blister development in the basal epidermis. Diacerein 1% Ointment is a topical ointment that is being developed for the treatment of EBS. Diacerein in the topical formulation is hydrolyzed to rhein in the epidermis and dermis following administration. Diacerein and rhein have been shown to inhibit the in vitro and in vivo production and activity of interleukin-1 (IL-1) and other pro-inflammatory cytokines.The purpose of this study is to compare the efficacy of Diacerein 1% Ointment to Control Ointment when applied once-daily for 8 weeks in subjects with EBS.

Detailed Study Description

The proposed study is an international, multicenter, randomized, double-blind, vehicle-controlled, parallel group study to evaluate the safety and efficacy of topical Diacerein 1% Ointment for the treatment of subjects with EBS. Subject randomization will be stratified by genotype (KRT5 and/or KRT14 versus other genotypes) and age group (<8 and 8 years old). Participants will be subject to up to 6-week screening period, then be assigned to treat EBS lesions once daily with either Diacerein 1% ointment topical formulation or placebo control ointment for 8 weeks, followed by an 8 week follow-up period. Approximately 80 subjects are planned to be randomized to one of the 2 treatment groups in this study at approximately 20 international investigational centers.

The primary objective of this study is to compare the efficacy of Diacerein 1% Ointment to Control Ointment based on reduction in body surface area (BSA) of EBS lesions being treated when applied once-daily for 8 weeks in subjects with EBS. The secondary objectives are to compare the effects of Diacerein 1% Ointment to Control Ointment in subjects with EBS in changes in Investigator Global Assessment (IGA) scores, pain, pruritus, mobility, and safety and tolerability.

Clinical Study Identifier: NCT03154333

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Joyce Teng, MD

Stanford University
Palo Alto, CA United States
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Anna Bruckner, MD

Children's Hospital Colorado
Aurora, CO United States
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Amy Paller, MD

Northwestern University
Chicago, IL United States
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Jonathan Dyer, MD

University of Missouri Healthcare
Columbia, MO United States
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Aida Lugo-Somolinos, MD

University of North Carolina - Chapel Hill
Chapel Hill, NC United States
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Cristina Has, MD

University Medical Center Freiburg
Freiburg, Germany
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Amy Paller, MD

Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, IL United States
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Deirdre Murrell, MD

Premier Specialists Pty Ltd; The Church
Kogarah, Australia
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Martin Laimer, MD

EB House Austria
Salzburg, Austria
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Emmanuelle Bourrat, MD

Hopital Saint Louis
Paris, France
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Christine Bodemer, MD

Hopital Necker-Enfants Malades
Paris, France
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Eli Sprecher, MD

Tel Aviv Sourasky Medical Center
Tel Aviv, Israel
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Marcel Jonkman, MD

University Medical Center Groningen
Groningen, Netherlands
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Jemima Mellerio, MD

St. Thomas' Hospital - St Johns Institute of Dermatology
London, United Kingdom
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Harper Price

Phoenix Childrens Hospital
Phoenix, AZ United States
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Kristen Hook, MD

University of Minnesota
Minneapolis, MN United States
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Anne Lucky, MD

Cincinnati Children's Hospital Medical Center
Cincinnati, OH United States
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John Browning, MD

Children's Hospital of San Antonio ; Texas Dermatology and Laser Specialists
San Antonio, TX United States
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Christine Chiaverini, MD

CHU de NICE - Hopital de l'Archet II - Service de Dermatologie
Nice, France
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Jordan Slutsky

Stony Brook University Medical Center
Stony Brook, NY United States
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Lara Wine Lee

Medical University of South Carolina (MUSC)
Charleston, SC United States
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Jemima Mellerio

Great Ormond Street Hospital
London, United Kingdom
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