Calling people living with severe EB Simplex in France!

Our DEBRA UK-funded clinical trial of the psoriasis drug, apremilast for EB Simplex (EBS) generalised severe, is underway at four centres in France but the researchers need more people to take part. Please speak to your EB specialist doctor or nurse if you live in France and would like to be involved. If you have any friends or family living with severe EBS in France, please share this article to raise awareness of the opportunity to get involved in the GEBULO trial.

Hear from Dr Christine Chiaverini who is leading this trial of apremilast in EBS in our research blog.

Six hospital visits will be needed over the course of the six-month trial, and these can be at the University Hospital of Nice, University Hospital of Toulouse, Hospital Necker Enfants Malades, or Hospital Saint Louis.

Apremilast tablets will be provided to adults and children aged six years or older with a confirmed genetic diagnosis of EBS due to changes in the genes for keratin-5 or keratin-14. The EBS symptoms must be severe enough that at least four new blisters appear daily and there are exclusion criteria that mean not everyone will be able to take part. The total number of new blisters on a person’s body will be counted daily by themselves or their family member during dressing changes for a week before each visit and recorded. The average number of new blisters per day will be compared between the beginning and end of three study periods. These are an eight-week period of treatment with apremilast, a period of four weeks without apremilast, then another eight-week period of apremilast tablets. As well as comparing the average number of daily new blisters, levels of pain, itch, quality of life and side effects of the treatment will be assessed and recorded.

This is an open-label study which means that it is not ‘blinded’. Everyone taking part will know who is taking apremilast and when they are taking it or not taking it. There will not be a placebo control because each person’s individual EB symptoms will be compared in the periods when they are taking apremilast to the period when they are not. This study design is partly because of the nature of EB as a rare condition where it is difficult to recruit a large number of participants, but also means that everyone taking part will receive the drug being tested.

Apremilast is a tablet, sold under the brand name Otezla, that is currently used to treat inflammatory autoimmune conditions including psoriasis in the UK, United States and European Union. It works by calming down specific parts of the immune system process called inflammation, that happens in response to skin damage. There are some known side effects of taking apremilast, including upset stomach which may affect about a quarter of people and can be severe in the first few weeks of taking it. Side effects will be carefully monitored in the trial and, as in any clinical trial, people taking part can stop at any point for any reason, even after signing all the consent forms.

You can read more about the details of this clinical trial in the BMJ Open medical journal and in a plain language article as well as on our website.