Diacerein orphan drug development for epidermolysis bullosa simplex: A phase 2/3 randomized, placebo-controlled, double-blind clinical trial
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F65269705%3A_____%2F18%3A00068575" target="_blank" >RIV/65269705:_____/18:00068575 - isvavai.cz</a>
Result on the web
<a href="https://www.sciencedirect.com/science/article/pii/S0190962218301300?via%3Dihub" target="_blank" >https://www.sciencedirect.com/science/article/pii/S0190962218301300?via%3Dihub</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1016/j.jaad.2018.01.019" target="_blank" >10.1016/j.jaad.2018.01.019</a>
Alternative languages
Result language
angličtina
Original language name
Diacerein orphan drug development for epidermolysis bullosa simplex: A phase 2/3 randomized, placebo-controlled, double-blind clinical trial
Original language description
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.
Czech name
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Czech description
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Classification
Type
J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database
CEP classification
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OECD FORD branch
30216 - Dermatology and venereal diseases
Result continuities
Project
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Continuities
I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace
Others
Publication year
2018
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
Journal of the American Academy of Dermatology
ISSN
0190-9622
e-ISSN
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Volume of the periodical
78
Issue of the periodical within the volume
5
Country of publishing house
US - UNITED STATES
Number of pages
17
Pages from-to
892-"+"
UT code for WoS article
000430421900023
EID of the result in the Scopus database
2-s2.0-85045583406