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Diacerein orphan drug development for epidermolysis bullosa simplex: A phase 2/3 randomized, placebo-controlled, double-blind clinical trial

V. Wally, A. Hovnanian, J. Ly, H. Buckova, V. Brunner, T. Lettner, M. Ablinger, TK. Felder, P. Hofbauer, M. Wolkersdorfer, FB. Lagler, W. Hitzl, M. Laimer, S. Kitzmüller, A. Diem, JW. Bauer,

. 2018 ; 78 (5) : 892-901.e7. [pub] 20180202

Language English Country United States

Document type Clinical Trial, Phase II, Clinical Trial, Phase III, Journal Article, Randomized Controlled Trial

Persistent link   https://www.medvik.cz/link/bmc19000934

BACKGROUND: Epidermolysis bullosa simplex (EBS) is a rare genetic, blistering skin disease for which there is no cure. Treatments that address the pathophysiology of EBS are needed. OBJECTIVE: Compare the impact of 1% diacerein cream with placebo in reducing the number of blisters in EBS. METHODS: In a randomized, placebo-controlled, phase 2/3 trial we used a 1% diacerein topical formulation to treat defined skin areas in 17 patients. In a 2-period crossover trial, patients were randomized to either placebo or diacerein for a 4-week treatment and a 3-month follow-up in period 1. After a washout, patients were crossed over during period 2. The prespecified primary end point was the proportion of patients with a reduction of number of blisters by more than 40% from baseline in selected areas over the treatment episode. RESULTS: Of the patients receiving diacerein, 86% in episode 1 and 37.5% in episode 2 met the primary end point (vs 14% and 17% with placebo, respectively). This effect was still significant after the follow-up. Changes in absolute blister numbers were significant for the diacerein group only. No adverse effects were observed. LIMITATIONS: Low patient numbers and no invasive data acquisition because of clinical burden in children. CONCLUSION: This trial provides evidence of the impact of 1% diacerein cream in the treatment of EBS.

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$a Wally, Verena $u EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology, University Hospital Salzburg, Paracelsus Medical University, Salzburg, Austria. Electronic address: v.wally@salk.at.
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$a Diacerein orphan drug development for epidermolysis bullosa simplex: A phase 2/3 randomized, placebo-controlled, double-blind clinical trial / $c V. Wally, A. Hovnanian, J. Ly, H. Buckova, V. Brunner, T. Lettner, M. Ablinger, TK. Felder, P. Hofbauer, M. Wolkersdorfer, FB. Lagler, W. Hitzl, M. Laimer, S. Kitzmüller, A. Diem, JW. Bauer,
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$a BACKGROUND: Epidermolysis bullosa simplex (EBS) is a rare genetic, blistering skin disease for which there is no cure. Treatments that address the pathophysiology of EBS are needed. OBJECTIVE: Compare the impact of 1% diacerein cream with placebo in reducing the number of blisters in EBS. METHODS: In a randomized, placebo-controlled, phase 2/3 trial we used a 1% diacerein topical formulation to treat defined skin areas in 17 patients. In a 2-period crossover trial, patients were randomized to either placebo or diacerein for a 4-week treatment and a 3-month follow-up in period 1. After a washout, patients were crossed over during period 2. The prespecified primary end point was the proportion of patients with a reduction of number of blisters by more than 40% from baseline in selected areas over the treatment episode. RESULTS: Of the patients receiving diacerein, 86% in episode 1 and 37.5% in episode 2 met the primary end point (vs 14% and 17% with placebo, respectively). This effect was still significant after the follow-up. Changes in absolute blister numbers were significant for the diacerein group only. No adverse effects were observed. LIMITATIONS: Low patient numbers and no invasive data acquisition because of clinical burden in children. CONCLUSION: This trial provides evidence of the impact of 1% diacerein cream in the treatment of EBS.
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$a Hovnanian, Alain $u INSERM UMR 1163, Laboratory of Genetic Skin Diseases, Imagine Institute, Paris, France; University Paris Descartes-Sorbonne Paris Cité, Paris, France; Department of Genetics, Necker Hospital, AP-HP, Paris, France.
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$a Ly, Juliette $u INSERM UMR 1163, Laboratory of Genetic Skin Diseases, Imagine Institute, Paris, France; University Paris Descartes-Sorbonne Paris Cité, Paris, France; Department of Genetics, Necker Hospital, AP-HP, Paris, France.
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$a Buckova, Hana $u Department of Pediatric Dermatology, Children's Hospital, Brno, Czech Republic.
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$a Brunner, Victoria $u EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology, University Hospital Salzburg, Paracelsus Medical University, Salzburg, Austria.
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$a Lettner, Thomas $u EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology, University Hospital Salzburg, Paracelsus Medical University, Salzburg, Austria.
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$a Ablinger, Michael $u EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology, University Hospital Salzburg, Paracelsus Medical University, Salzburg, Austria.
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$a Felder, Thomas K $u Department of Laboratory Medicine, Paracelsus Medical University, Salzburg, Austria.
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$a Hofbauer, Peter $u Landesapotheke Salzburg, Department of Production, Hospital Pharmacy, Salzburg, Austria.
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$a Wolkersdorfer, Martin $u Landesapotheke Salzburg, Department of Production, Hospital Pharmacy, Salzburg, Austria.
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$a Lagler, Florian B $u Institute for Inborn Errors of Metabolism, Paracelsus Medical University, Salzburg, Austria; Department of Pediatrics, Paracelsus Medical University, Salzburg, Austria.
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$a Hitzl, Wolfgang $u Research Office Biostatistics, Paracelsus Medical University, Salzburg, Austria.
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$a Laimer, Martin $u Department of Dermatology, University Hospital Salzburg, Paracelsus Medical University, Salzburg, Austria.
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$a Kitzmüller, Sophie $u EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology, University Hospital Salzburg, Paracelsus Medical University, Salzburg, Austria.
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$a Diem, Anja $u EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology, University Hospital Salzburg, Paracelsus Medical University, Salzburg, Austria.
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