Extrait

Background and study aims This research study concerns individuals with the inherited blistering skin disease known as dystrophic epidermolysis bullosa (DEB). This a very severe condition in which skin injury leads to blisters and chronic wounds that remain inflamed and take longer to heal and sometimes never heal. The entire skin and internal organs can be affected. The increased skin fragility leads to scarring and an increased risk of skin cancer at a young age. Currently we have no effective treatments for DEB and we are unable to prevent the disease complications. Our aim is to show that skin cells called fibroblasts can improve wound healing in people with DEB. We know that these cells make collagen VII, the protein that is genetically abnormal in these people. We have shown in previous studies that skin fibroblasts from other healthy people are able to compensate for this and stimulate production of some of the collagen VII that is missing in your skin. Who can participate? Patients aged 16-70 diagnosed with dominant or recessive DEB. What does the study involve? If you take part, first of all, you will have several of your skin wounds assessed and photographed. You will have a physical examination of your skin and you will be asked to donate a small blood sample. The sample will be taken in the same way as any standard blood test. A urine test to exclude pregnancy will be needed for women. On your next visit, if your wounds are not showing signs of spontaneous healing, you will be invited to participate in the next part of the trial which involves injections into your skin. We would like to inject two preparations into your skin. One will be a solution containing the fibroblast cells, the other will be the solution but without the fibroblast cells. The solution lacking the fibroblasts is called a “placebo”. We feel it is important to compare the effects of fibroblasts and placebo on wound healing in your skin so that we can learn more about how your skin heals. Up to 10 skin wounds may be treated and we plan to do this on one visit to the hospital. This may require up to 60 injections in your skin. This may sound like a lot of injections but they should not cause you much discomfort. Each injection may cause brief stinging for a few seconds, similar to having a blood test. The size of the needle used for injecting the fibroblast cells or placebo solution is in fact slightly thinner than most blood test needles. If you find the injection process at all uncomfortable, we can interrupt the injections and numb the skin with a topical anaesthetic cream. After the injections you will be able to go home immediately. The choice of either fibroblasts or placebo will be selected in a random manner. On average, half the wounds will be treated with cells and half with placebo, but you will not be told how each individual wound has been treated. This step will then allow us to follow up the healing process in a “blinded” manner. Only at the end of the trial you will learn how each wound was treated. After the initial injection day, you will be asked to attend for six further clinic visits over the subsequent six months. On these visits, the wounds selected during your first visit will be photographed and their size measured. We will ask you to keep a wound diary and take weekly photos of the selected wounds with cameras provided by us. Training and technical support will also be provided. The measurements will be done with a computer software programme and this will be a painless procedure. We will also take a skin swab from the wounds to test for any infection; this is also a painless procedure. On two of these visits you will be asked to give two further small blood samples. We are also keen to assess the impact of the cell injections on your skin and we will ask you to complete a questionnaire about this. What are the possible benefits and risks of participating? It is likely that participation in this research project will not be of immediate clinical benefit to you. However, the results should be able to tell us whether skin fibroblasts have the potential to speed up wound healing and improve skin function as well as the quality of your life by reducing the wound pain. At the same time by following non-treated wounds with serial photographs we will be able to assess and document their healing process. The final results of the clinical trial will be discussed with you in detail by a member of the research team. The injection of fibroblasts or inactive solution into the skin could cause pain for a few seconds. We plan to minimise the pain by anaesthetising the skin using a topical anaesthetic cream if you find the pain of the injections too uncomfortable. Several studies have shown that injected skin fibroblasts are well tolerated but there is the potential to cause minor redness and irritation that should resolve spontaneously over a few days. Venous sampling for blood tests could result in pain, bruising and/or infection at the injection site. Infection can be treated with a short course of oral antibiotics. Where is the study run from? It is being organized by Guy’s and St Thomas’ NHS Trust in London (UK). When is the study starting and how long is it expected to run for? The study began in January 2011 is open to participants until November 2011. Who is funding the study? The study is being funded by the Dystrophic Epidermolysis Bullosa Research Association (DEBRA) (UK), Technology and Strategy Board (UK) and Intercytex Ltd (UK) - investigational product manufacturer and supplier. Who is the main contact? Dr Gabriela Petrof [email protected]

Critère d'inclusion

• Dystrophic epidermolysis bullosa (DEB)

Liens

• ictrp
• isrctn