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Anticipated DEBRA funding of three clinical trials for severe RDEB

Expert evaluation for the DEBRA International Clinical Research Call 2015 has led to funding recommendation of three clinical trials targeting priority medical needs in RDEB.

In 2015, DEBRA International held a Call for proposals to its Clinical Research Programme for major funding support of clinical research and clinical trials targeting therapy development and symptom-relief for EB.

Eighteen expression-of-interest outline applications were made to Stage 1 of the Call. Following evaluation of these outline proposals, nine applicants were invited to submit full proposals to Stage 2, and these addressed a wide variety of therapeutic approaches targeting symptom relief or the underlying causes of EB.

The criteria for the Call were that a proposal address either late-stage preclinical research, or clinical trials, of treatments where a substantial body of evidence of proof of concept already existed.

Assessment of these proposals against the criteria of scientific quality and ultimate patient benefit was undertaken by both external peer-review, and consideration by an Expert Panel specially convened for the Call. Members of the Expert Panel were drawn from DEBRA International's MSAP, external academic and clinical experts in particular therapeutic technologies, and included representatives from the biopharma industry to provide a wider perspective on the likely feasibility of translating the proposed treatments into eventual clinical practice.

As a result of review, three proposals were recommended for funding by the Expert Panel, and these recommendations were subsequently considered by the Boards of Trustees of DEBRA Austria and DEBRA UK, national member organisations of DEBRA International, which had committed funding to support the Call. DEBRA Austria has approved support for two early-stage clinical trials, while DEBRA UK will support a further clinical trial.

Whilst the call was open to (pre)clinical research for all types of EB, all three clinical trials approved for funding focus on priority unmet medical needs in severe RDEB.

One targets a grafting (ex-vivo) gene therapy to address the underlying genetic fault in RDEB. The other two address the severe consequences of RDEB through repurposing drugs (i.e. investigating the suitability of drugs originally investigated or approved for other conditions), which has the advantages that there is usually already a body of safety evidence, at least in other patient groups. One of these trials targets the fibrosis that causes disabling scarring and contractures, and the other targets the life-threatening squamous cell carcinoma (SCC): both these complications result from the chronic wounds and inflammation in RDEB skin.

If successful, these early-stage trials would lead to expanded, later-stage trials, to confirm safety and establish stronger evidence of efficacy, and eventually, it is hoped, to treatments that could improve quality of life and potentially life expectancy for people with RDEB.

At present, discussions are underway with the applicants to finalise amendments to the proposals as recommended by the Expert Panel, with the aim of refining protocols and investigations to improve the projects' value, and likelihood of success. On completion also of contractual negotiations, it is anticipated that awards of approximately €500K will be made to each.

Dr. Clare Robinson, DEBRA International Head of Research

Read previous articles about this topic:

word cloud around the DEBRA "Clinical Research Call 2015"

Group picture of the Expert panel for the Clinical Research Call 2015

Expert panel specially convened for the DEBRA Clinical Research Call 2015