Extrait

Background and study aims: Recessive Dystrophic Epidermolysis Bullosa (RDEB) is a severe inherited skin disease caused by lack of collagen VII - the protein that 'sticks' the top and bottom layer of the skin together. It is a severe condition leading to skin fragility, blisters and wounds which are slow to heal or leave non-healing open wounds. There is desperate need to develop new treatments for RDEB and attempts are being made to develop clinical trials using different types of therapies. This current research project aims to find out if using a particular type of cell is safe to use and can improve the skin disease in this genetic disease. Who can participate? Children with a diagnosis of RDEB, aged 1 to 17 years. What does the study involve? The study will consist of a total of 7 visits. The first visit will consist of a screening consultation with a study doctors, during the one the study will be thoroughly explained and questions answered. If the parent and child decide to participate, an informed consent form will be signed. Six further visits will be scheduled after this, during which different procedures will be carried out: cell infusions, blood tests and skin biopsies. Also, various assessments of the wounds, pain and quality of life will be done using different scoring systems. What are the possible benefits and risks of participating? If the treatment works, the skin disease may become milder with fewer blisters and wounds that hopefully heal faster. However, we don't know how long the effects will last. We will follow the child in the context of the clinical trial for 6 months and then continue to collect information about the skin disease during the routine clinical appointments for up to 1 year. If the cell treatment proves safe and study participants benefit from it, there will be the possibility to administer further cell treatments. We also hope that the information we gather from this clinical trial will contribute to future studies for therapies of individuals with RDEB. Mesenchymal Stem Cells (MSCs) have been used in clinical trials for other medical conditions with no severe side effects recorded and recent studies in using MSCs for children with RDEB in other countries have reported no serious adverse reactions. Although not expected, the infusion of any blood product carries a small risk of complications such as allergic reaction, infection or other unpredicted reactions that could potentially require medical care and hospitalisation. Blood taking and skin biopsies 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? The study will take place at Great Ormond Street Hospital for Children in London, UK. When is the study starting and how long is it expected to last for? We are aiming to start the study on the 14/10/2012 . Study participants will be followed up in the context of the clinical trial for 6 months and then continue to collect information about the skin disease during the routine clinical appointments for up to 1 year. Who is funding the study? Dystrophic Epidermolysis Bullosa Research Association (DebRA) and sponsored by King's College London. Who is the main contact? Professor John McGrath [email protected]

Critère d'inclusion

• Recessive Dystrophic Epidermolysis Bullosa

Liens

• isrctn
• ictrp