Last updated on February 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 and suprabasal epidermis. Diacerein 1% Ointment is a topical ointment that is being developed for the treatment of EBS with the potential to block activity of interleukin-1 (IL-1), a pro-inflammatory cytokine, and reduce the auto-inflammatory effects in the skin of patients with EBS. 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, randomized, double-blind, parallel-group Phase 2 study. 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 18 international investigational centers.

The objective of this study is to compare the efficacy of Diacerein 1% Ointment to Control Ointment when applied once-daily in subjects with EBS using an Investigator Global Assessment to determine overall treatment success. The study will also compare the effects of Diacerein 1% Ointment to Control Ointment in subjects with EBS in terms of safety and tolerability, patient-reported effects on pruritus, pain, mobility, as well as reduction in body surface area of EBS lesions, and reduction in lesion surface area of the referenced lesion.

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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John Browning, MD

Texas Dermatology and Laser Specialists
San Antonio, TX United States
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Cristina Has, MD

University Medical Center Freiburg
Freiburg, Germany
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Amy Paller

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

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

EB House Austria
Salzburg, Austria
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Emmanuelle Bourrat

Hopital Saint Louis
Paris, France
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Christine Bodemer

Hopital Necker-Enfants Malades
Paris, France
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Eli Sprecher

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

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