CMAPS
Research type
Research Study
Full title
A Collaborative Multi-omic Approach to Pediatric Scleroderma (CMAPS) to molecularly characterize and stratify patients.
IRAS ID
364929
Contact name
Christian M. Hedrich
Contact email
Sponsor organisation
University of Liverpool
Duration of Study in the UK
4 years, 0 months, 0 days
Research summary
Juvenile onset scleroderma (including systemic sclerosis (SSc) and localized scleroderma (LS)) is a rare autoimmune condition that affects children, leading to increased skin thickness and damage to internal organs. A recent study supports that interstitial lung disease (ILD), the leading cause of SSc-associated death in adults, is just as prevalent in jSSc. There is limited knowledge of the cellular and molecular pathways that are involved in this disease process, making it extremely difficult for physicians to treat patients effectively, leading to trial and error with medications that can result in serious side effects. In addition, delaying treatment in growing children can also result in permanent damage and life-long disabilities.
To combat this problem, we would like to run a study that analyses blood samples, nasal epithelia samples, and skin samples from patients with jSSc and jLS. By using advanced technology, we can analyse specific genes and cells in the skin that are playing a major role in the disease process. Examining how certain cells are arranged in the skin, how they communicate with each other, and what changes are occurring in those cells due to disease, may help us find the key players that are driving inflammation and fibrosis. We will then look in the blood to see if we can see the same signals we have identified in the skin – in the future, this means we can take a blood sample rather than a skin biopsy which is more acceptable for patients.
Understanding the molecular and cellular processes of juvenile onset scleroderma and being able to identify specific profiles will improve patient outcomes by helping physicians develop personalized treatment plans for their patients.
REC name
London - Hampstead Research Ethics Committee
REC reference
26/PR/0185
Date of REC Opinion
19 Mar 2026
REC opinion
Further Information Favourable Opinion