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18.10.2016

Expansion to new sites worldwide of phase 3 clinical trial of topical cream SD-101 for all EB types

As an update on the DEBRA news item of 17 August 2015, the number of sites at which people with any type of EB being enrolled into the ‘ESSENCE’ clinical study of the cream SD-101 has increased to 25 around the world, with still more planned.

Earlier-stage clinical studies of the topical cream were carried out in 2012 and 2014 by the company Scioderm, now acquired by the company Amicus Therapeutics. Regulatory authorities in the USA and Europe, whose role is to oversee approval of new medicines for safety and effectiveness, gave permission for the phase 3 trial based on the results of the earlier phase 2b trial. Recruiting enough patients, both adults and children, to the phase 3 clinical trial will be important for gathering the information to demonstrate conclusively whether the SD-101 cream has significant patient benefit and is safe. The results of the trial can then be considered by regulators in deciding whether to give approval for routine clinical use.  

Detailed information on the trial may be found at:   https://www.clinicaltrials.gov/ct2/show/study/NCT02384460?term=sd101&show_locs=Y#locn , and Amicus provides the following information, and links to further details:

 

Amicus Therapeutics is expanding the number of international sites for participation in the ESSENCE Study.  The ESSENCE Study is a Phase 3 clinical trial of SD-101, an investigational topical cream to treat skin blistering and lesions associated with Epidermolysis Bullosa (EB).

 

The double-blind, placebo-controlled, trial is evaluating the safety and efficacy of SD-101 in patients aged 1 month and older with various subtypes of EB (Simplex, Recessive Dystrophic and Junctional [non-Herlitz]).

 

Currently, there are 14 sites in the United States and eight in Europe including Austria, France (2 sites), Germany, Italy, Netherlands, Poland and the UK.  A new site in Australia has just opened and is now enrolling study participants, bringing the number of sites in Australia up to three.   Over the coming months, several more sites are expected to join the ESSENCE Study in Europe and other regions, while even more locations are being explored as well.  Travel and lodging assistance is being provided to participants in the ESSENCE Study.

 

The ESSENCE Study (at all sites) provides individuals with EB who have chronic wounds the opportunity to help in the possible development of an investigational treatment for EB wounds that could potentially help alleviate some of the suffering faced by people with EB.. 

For more information on how to qualify and participate, as well as to find the latest contacts and locations, go to  www.ebstudy.com  or e-mail info@ebstudy.com.

 

Dr. Clare Robinson, Head of Research DEBRA International

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DEBRA International provides information and news that may be of interest to people with EB, but does not recommend or endorse products, or participation in any particular clinical trial. We strongly recommend that anyone considering a treatment or clinical trial should also speak with their doctor, or medical team, who helps them manage their EB.