Topical diacerein 1% met its primary efficacy endpoint in a study of patients with epidermolysis bullosa simplex (EBS), according to final results from a phase 2 study presented at the 25th Annual Congress of the European Academy of Dermatology and Venereology in Vienna, Austria.
Johann Bauer, MD, MBA, HCM, head of the University Clinic for Dermatology of the SALK/Paracelsus Medical University and the principal investigator in the trial, presented the results that evaluated the efficacy and safety of topical diacerein 1%, an inhibitor of IL-1β, compared with placebo in patients with EBS.
Data from the completed multicenter, randomized, double-blind, placebo-controlled trial included 17 patients with EBS who were treated for a 4-week period followed by a 3-month follow-up phase in 2 subsequent years, with a crossover after the first year. The patient age range was 4 to 12 years with approximately equal numbers of men and women.
Results demonstrated that 60% of patients in the topical diacerein 1% treatment group achieved the primary endpoint (at least a 40% reduction in the number of blisters) at 4 weeks. Only 15% of placebo-treated patients showed a similar benefit. Continued benefit of topical diacerein 1% was also demonstrated after cessation of treatment through the follow-up period of 12 weeks. Topical diacerein 1% was well tolerated by patients with no treatment-related adverse events reported.
“We are happy that this study confirms our initial experimental results and phase 1 data. The superior efficacy of diacerein coupled with an excellent tolerability profile may indicate a potential new and safe approach for treating patients with EBS,” said Dr Bauer.
Topical diacerein 1% met its primary efficacy endpoint in a study of patients with epidermolysis bullosa simplex (EBS), according to final results from a phase 2 study presented at the 25th Annual Congress of the European Academy of Dermatology and Venereology in Vienna, Austria.
Johann Bauer, MD, MBA, HCM, head of the University Clinic for Dermatology of the SALK/Paracelsus Medical University and the principal investigator in the trial, presented the results that evaluated the efficacy and safety of topical diacerein 1%, an inhibitor of IL-1β, compared with placebo in patients with EBS.
Data from the completed multicenter, randomized, double-blind, placebo-controlled trial included 17 patients with EBS who were treated for a 4-week period followed by a 3-month follow-up phase in 2 subsequent years, with a crossover after the first year. The patient age range was 4 to 12 years with approximately equal numbers of men and women.
Results demonstrated that 60% of patients in the topical diacerein 1% treatment group achieved the primary endpoint (at least a 40% reduction in the number of blisters) at 4 weeks. Only 15% of placebo-treated patients showed a similar benefit. Continued benefit of topical diacerein 1% was also demonstrated after cessation of treatment through the follow-up period of 12 weeks. Topical diacerein 1% was well tolerated by patients with no treatment-related adverse events reported.
“We are happy that this study confirms our initial experimental results and phase 1 data. The superior efficacy of diacerein coupled with an excellent tolerability profile may indicate a potential new and safe approach for treating patients with EBS,” said Dr Bauer.
Topical diacerein 1% met its primary efficacy endpoint in a study of patients with epidermolysis bullosa simplex (EBS), according to final results from a phase 2 study presented at the 25th Annual Congress of the European Academy of Dermatology and Venereology in Vienna, Austria.
Johann Bauer, MD, MBA, HCM, head of the University Clinic for Dermatology of the SALK/Paracelsus Medical University and the principal investigator in the trial, presented the results that evaluated the efficacy and safety of topical diacerein 1%, an inhibitor of IL-1β, compared with placebo in patients with EBS.
Data from the completed multicenter, randomized, double-blind, placebo-controlled trial included 17 patients with EBS who were treated for a 4-week period followed by a 3-month follow-up phase in 2 subsequent years, with a crossover after the first year. The patient age range was 4 to 12 years with approximately equal numbers of men and women.
Results demonstrated that 60% of patients in the topical diacerein 1% treatment group achieved the primary endpoint (at least a 40% reduction in the number of blisters) at 4 weeks. Only 15% of placebo-treated patients showed a similar benefit. Continued benefit of topical diacerein 1% was also demonstrated after cessation of treatment through the follow-up period of 12 weeks. Topical diacerein 1% was well tolerated by patients with no treatment-related adverse events reported.
“We are happy that this study confirms our initial experimental results and phase 1 data. The superior efficacy of diacerein coupled with an excellent tolerability profile may indicate a potential new and safe approach for treating patients with EBS,” said Dr Bauer.
