Biomarker and cohort study of localised scleroderma in childhood
Research type
Research Study
Full title
Biomarker and cohort study of localised scleroderma in childhood
IRAS ID
232334
Contact name
Despina Eleftheriou
Contact email
Sponsor organisation
Great Ormond Street Hospital NHS Foundation Trust
Duration of Study in the UK
3 years, 0 months, 1 days
Research summary
Scleroderma is a rare condition (<1 in 100,000 children) that results in hard, thickened areas of skin and sometimes affects internal organs. Our recent UK wide study of localised scleroderma (LS) in children highlighted the burden of morbidity and disfigurement in this disease. Disappointingly however there is almost no evidence base for treatment and clinical trials are urgently needed. A key limiting factor has been the lack of objective outcome measures to assess disease activity. In addition, there are no circulating blood markers that can help us reliably track disease activity in LS. Therefore there is an urgent need to identify biomarkers (blood tests) and other clinical measures of disease activity that can help us also develop robust outcome measures for clinical treatment trials. Without validated outcome measures (biomarkers/clinical assessments) it is currently difficult to ascertain treatment effects and therefore the evidence for efficacy for any treatment is lacking. The aims of this study are: (i) to create the infrastructure for collecting clinical data for children with the localised form of the disease (localised scleroderma; LS); (ii) identify biomarkers (blood tests) of disease activity that can be used in combination with other clinical measures as robust outcome measures for treatment trials.
REC name
London - Bloomsbury Research Ethics Committee
REC reference
17/LO/1628
Date of REC Opinion
18 Dec 2017
REC opinion
Further Information Favourable Opinion