PEBLES
Research type
Research Study
Full title
Prospective Epidermolysis Bullosa Longitudinal Evaluation Study (PEBLES): Natural history and clinical endpoints in epidermolysis bullosa
IRAS ID
142032
Contact name
Jemima Mellerio
Contact email
Research summary
Epidermolysis bullosa (EB) is a general term used to describe a group of inherited skin disorders in which there is a tendency for the skin and mucous membranes to break down following minor injury. One of the most severe forms of EB is recessive dystrophic EB (RDEB), which can affect many systems in the body.
In order to undertake clinical trials of new, emerging therapies in RDEB, it is necessary to have a clear understanding about the natural history and course of the disease so that any modification by a therapy can be reliably assessed.
Our proposed study will examine and record data from a large series of RDEB patients (approximately 120) who are seen at the adult and paediatric EB centres in London. Data collected from patients will include: demographics, clinical examination findings, test results (e.g. blood tests, bone scans, echocardiograms), and data on any treatments or procedures they have had. All of this information would normally be collected as part of the patients’ regular clinical care. The data captured will give a picture of the clinical and laboratory status of patients at different ages from birth throughout life. Subsequently, for the 2 year duration of the project, individual patients will have the same information recorded (every 6 months in children up to 10 years, annually thereafter) to provide a longitudinal record of how the EB has progressed for that individual. This data will provide insight into the natural course of RDEB over time, and also identify which clinical features are meaningful as endpoints for clinical trials of new EB therapies.REC name
London - Bromley Research Ethics Committee
REC reference
15/LO/0001
Date of REC Opinion
24 Sep 2014
REC opinion
Favourable Opinion