PROMWiSe V0.2
Research type
Research Study
Full title
Measuring What Matters in Wolfram Syndrome: A group concept mapping exercise to facilitate identification of the most appropriate PROMs for use in clinical trials and practice for children and adults living with Wolfram Syndrome.
IRAS ID
238627
Contact name
Anita Slade
Contact email
Sponsor organisation
University of Birmingham
Clinicaltrials.gov Identifier
ERN_18-1380, Ethics Number
Duration of Study in the UK
0 years, 5 months, 30 days
Research summary
Background: Wolfram Syndrome (WS) is a rare progressive genetic disease that effects children from an early age and often results in premature death from neurological complications in young adulthood. People with the syndrome have a range of issues including diabetes, visual impairment, neurological impairment and psychological issues related to the condition such as depression. As part of a larger clinical trial we wish to identify a range of patient reported outcome measures (PROMs) which are age appropriate for the issues that people living with WS identify as problematic. This will allow us to measure the impact of therapeutic interventions aimed at slowing down or improving the progression of WS, and the extent to which interventions improve patients quality of life. However, because its a rare condition we don't know which PROMS would be best to use and which issues are important to people living with Wolfram syndrome. Previous patient partner involvement (PPI) work with adults, children and their parents suggested that there were a diverse range of issues that needed to be addressed and not all of the reviewed PROMs addressed all of their issues. Therefore, we want to do a group concept mapping exercise with children, young adults and and adults living with Wolfram syndrome to identify which symptoms have the most impact on their life. This will allow us to identify the most suitable and age appropriate PROMs to measure changes in their symptoms and the impact on their quality of life.
REC name
West Midlands - South Birmingham Research Ethics Committee
REC reference
19/WM/0220
Date of REC Opinion
19 Aug 2019
REC opinion
Further Information Favourable Opinion