A study to test the use of a visual tool in measuring disease activity in children with dermatomyositis

ISRCTN ISRCTN59325153
DOI https://doi.org/10.1186/ISRCTN59325153
IRAS number 333838
Secondary identifying numbers CPMS 60608, NIHR204944, IRAS 333838
Submission date
09/02/2024
Registration date
15/02/2024
Last edited
20/03/2025
Recruitment status
No longer recruiting
Overall study status
Ongoing
Condition category
Skin and Connective Tissue Diseases
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Record updated in last year

Plain English Summary

Background and study aims
Juvenile dermatomyositis (JDM) is a rare but serious disease which causes muscle and skin inflammation, affects blood vessels and can affect major organs. With treatment, the disease can enter remission, but course is unpredictable and persistent disease or disease flares occur.
Blood vessel abnormality (vasculopathy) is key to disease pathogenesis and persistent vasculopathy is associated with more severe disease, worse disease manifestations, and risk of life-threatening complications. Blood vessels at the base of the fingernail can be visualised when magnified and provide a valuable insight into vascular disease activity. There is an unmet need to better evaluate vasculopathy in clinical practice, to improve long-term disease outcome.
Nailfold capillary abnormalities have been found to relate to biomarkers of vascular disease activity in the blood stream of patients with JDM, but these vascular biomarkers are not yet available in routine clinical practice. The importance of measuring nailfold changes in clinic is well recognised but hampered by the lack of equipment that allows this to occur. Use of a handheld device, with images captured and sent to the specialist reporting centre can provide a step change in the management of patients with JDM.
This study will test the use of a visual tool (Dinolite CapillaryScope) that allows patients to see their blood vessels and help them know how active their disease is. If deemed useful it may provide an increased understanding of the need for medication need and motivation to improve adherence.

Who can participate?
Participants 0-18 years old, with juvenile dermatomyositis.

What does the study involve?
Participants will have nailfold capillaroscopy (NFC) performed on up to 8 fingers. Patients and parents will be asked to complete age-appropriate questionnaires on their opinions of NFC. Patients and parents will be invited to take part in an individual patient/family interview to further determine impact of NFC. This is optional. Healthcare professionals will complete a NFC data form at the time of NFC and time of receiving a report.

What are the possible benefits and risks of participating?
BENEFITS- If it is demonstrated that NFC is beneficial in clinic, it can easily and quickly be incorporated into all specialist centres that care for children/young people with JDM in the UK.
If deemed useful, Dinolite CapillaryScope and NFC will provide an increased understanding of the need for medication need and motivation to improve adherence. It could help patients have a sense of control, reduce anxiety, give a better understanding of symptoms, sense of being understood, improve confidence in management and a more hopeful outlook for future quality of life/well-being.

RISKS- The main burden is the extra time taken in clinic for NFC to be performed on children/young people with JDM. This needs to be evaluated against any benefit for patients/parents and clinicians.
Nailfold capillaroscopy is painless and adverse events are not anticipated. It is possible that patients participating may reflect on implications of their disease, with emotional consequences. If patients experience any unexpected outcomes such as increased anxiety or emotional distress about their disease, they will be supported by our study psychologist and members of the multi-disciplinary team at each participating centre.

Where is the study run from?
Alder Hey Children's Hospital (UK)

When is the study starting and how long is it expected to run for?
September 2023 to August 2025

Who is funding the study?
National Institute for Health and Care Research (NIHR) (UK)

Who is the main contact?
Dr Liza McCann (Liza.McCann@alderhey.nhs.uk)
Jessica Fitzgerald (jessica.fitzgerald@liverpool.ac.uk)

Contact information

Miss Jessica Fitzgerald
Public

Institute in the Park, Alder Hey Children's Hospital, Eaton
Liverpool
L12 2AP
United Kingdom

Phone +44 7785740950
Email Jessica.Fitzgerald@liverpool.ac.uk
Dr Liza McCann
Scientific, Principal Investigator

Alder Hey Children's Hospital, Eaton Road
Liverpool
L12 2AP
United Kingdom

ORCiD logoORCID ID 0000-0002-0372-5758
Phone +44 1512824521
Email liza.mccann@alderhey.nhs.uk

Study information

Study designInterventional non-randomized with qualitative follow up
Primary study designInterventional
Secondary study designNon randomised study
Study setting(s)Hospital
Study typeDiagnostic
Participant information sheet Not available in web format, please use the contact details to request a patient information sheet.
Scientific titleMYOSCOPE: Bringing a visual measure of disease activity into clinical practice to help children with dermatomyositis
Study acronymMYOSCOPE
Study hypothesisThe purpose of this study is to determine whether a low-cost handheld tool (Dinolite® CapillaryScope) can adequately capture blood vessel changes at the base of the fingernails in children/ young people with juvenile dermatomyositis (JDM) when it is used in clinic, and whether this benefits patients/parents in understanding their disease and need for medication, as well as benefiting healthcare professionals.
Ethics approval(s)

Approved 01/02/2024, East Midlands – Nottingham 2 (2 Redman Place, Stratford, London, E20 1JQ, United Kingdom; +44 20 7104 8057; nottingham2.rec@hra.nhs.uk), ref: 24/EM/0023

ConditionJuvenile Dermatomyositis (JDM)
InterventionPatients will be recruited from two UK centres. All patients wanting to participate in this study can do so. There is no randomisation. This study is predominantly cross-sectional.
During routine clinic visits, patients will have nailfold capillaroscopy (NFC) in up to 8 fingers (minimum 2 fingers), taking approximately 15 minutes. An age-adjusted questionnaire will explore feelings about NFC and if perceived to be helpful. Study participants will be invited to participate in a single individual/family interview to further explore impact of NFC. Options available will ensure families with digital poverty/language barriers can participate.
Clinicians will complete data at each patient visit (1-3 per patient) to document time taken for NFC, any difficulty, and whether NFC changed treatment decisions. After receiving a formal NFC report (within 1-2 weeks), clinicians will document if any further treatment changes were made. NFC will be compared to tools currently used to measure global and skin disease activity in JDM.
Intervention typeOther
Primary outcome measureFeasibility of performing NFC in clinic and benefit (or not) to patients, parents and healthcare professionals. This will be assessed qualitatively through questionnaires and interviews.
Secondary outcome measuresThere are no secondary outcome measures
Overall study start date01/09/2023
Overall study end date31/08/2025

Eligibility

Participant type(s)Patient
Age groupChild
Upper age limit18 Years
SexBoth
Target number of participantsPlanned Sample Size: 40; UK Sample Size: 40
Total final enrolment40
Participant inclusion criteria1. Medical diagnosis of Juvenile dermatomyositis (JDM) with or without overlap features.
2. Less than 18 years of age
3. Any time point of disease.
Participant exclusion criteria1. Unwillingness to participate.
Procedures are in place to ensure that patients are not excluded due to language barriers, geographical location, digital poverty or other factors.
No patient / parent will be coerced into involvement of this research project. Patients can choose to leave the research project at any time, without the need to explain their reasons.
Recruitment start date26/06/2024
Recruitment end date13/03/2025

Locations

Countries of recruitment

  • England
  • United Kingdom

Study participating centres

Alder Hey Children's NHS Foundation Trust
Alder Hey Hospital
Eaton Road
West Derby
Liverpool
L12 2AP
United Kingdom
Great Ormond Street Hospital for Children
Great Ormond Street
London
WC1N 3JH
United Kingdom

Sponsor information

Alder Hey Children's NHS Foundation Trust
Hospital/treatment centre

Alder Hey Hospital, Eaton Road, West Derby
Liverpool
L12 2AP
England
United Kingdom

Phone +44 151 252 5570
Email research@alderhey.nhs.uk
Website http://www.alderhey.nhs.uk/
ROR logo "ROR" https://ror.org/00p18zw56

Funders

Funder type

Government

NIHR Central Commissioning Facility (CCF)

No information available

Results and Publications

Intention to publish date31/08/2026
Individual participant data (IPD) Intention to shareYes
IPD sharing plan summaryStored in non-publicly available repository
Publication and dissemination planThe findings of the study will be disseminated via medical conferences and in peer-reviewed journals and shared internationally via scientific networks, newsletters and social media. Funding bodies will require acknowledgement in publications and will require reports of the study. Results will also be presented at future Patient and Public Involvement events and shared in patient/parent newsletters. A lay summary of study findings and publications relating to the study will be shown on the JDCBS website.
IPD sharing planThe datasets generated during and/or analysed during the current study will be stored in a non-publicly available repository (Data will be collected on site and entered on to an electronic spreadsheet. The spreadsheet will be encrypted and be stored on a password protected computer in a locked area and the file will be password protected).

Editorial Notes

20/03/2025: The following changes were made:
1. Total final enrolment added.
2. The recruitment end date was changed from 01/03/2025 to 13/03/2025.
12/07/2024: The recruitment start date was changed from 01/06/2024 to 26/06/2024.
13/05/2024: The recruitment start date was changed from 01/05/2024 to 01/06/2024.
08/04/2024: The recruitment start date was changed from 01/04/2024 to 01/05/2024.
09/02/2024: Trial's existence confirmed by the National Institute for Health and Care Research (NIHR) (UK).