NCT03154333

Safety and Efficacy of Diacerein 1% Ointment for Subjects With Epidermolysis Bullosa Simplex (EBS)

An International, Multicenter, Randomized, Double-Blind, Parallel-Group Phase 2 Study Evaluating the Safety and Efficacy of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 54 (actual)
• Sponsor: Castle Creek Pharmaceuticals, LLC · Industry
• Sex: All
• Age: 4 Years
• Healthy volunteers: Not accepted

Summary

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 purpose of this study is to compare the efficacy of diacerein 1% ointment to vehicle ointment when applied once-daily for 8 weeks in subjects with EBS.

Detailed description

The study is an international, multicenter, randomized, double-blind, vehicle-controlled, parallel group study to evaluate the safety and efficacy of diacerein 1% ointment for the treatment of subjects with EBS. Participants were assigned to study groups receiving either diacerein 1% ointment or vehicle ointment for 8 weeks, followed by an 8 week follow-up period. Approximately 80 subjects were to be randomized to one of the 2 treatment groups. The primary objective of this study is to compare the efficacy of diacerein 1% ointment to vehicle 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 vehicle ointment in subjects with EBS in changes in Investigator Global Assessment (IGA) scores, pain, pruritus, mobility, and safety and tolerability.

Conditions

• Epidermolysis Bullosa Simplex

Interventions

Type	Name	Description
DRUG	diacerein 1% ointment	diacerein 1% ointment administered topically
DRUG	A placebo ointment	vehicle ointment administered topically

Timeline

• Start date: 2017-06-01
• Primary completion: 2018-10-15
• Completion: 2018-10-31
• First posted: 2017-05-16
• Last updated: 2019-11-05
• Results posted: 2019-11-05

Locations

22 sites across 8 countries: United States, Australia, Austria, France, Germany, Israel, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study