Study of health outcomes and experiences of young people with XLH
Research type
Research Study
Full title
An observational, prospective, European, multicentre, mixed methods study to describe the lived experience of X-Linked Hypophosphatemia (XLH) for adolescents at end of skeletal growth
IRAS ID
297382
Contact name
Vrinda Saraff
Contact email
Sponsor organisation
Kyowa Kirin International
Clinicaltrials.gov Identifier
NA, NA
Duration of Study in the UK
2 years, 4 months, 31 days
Research summary
This observational, prospective, European, multicentre, mixed methods study aims to describe the lived experience of X- Linked Hypophosphatemia (XLH) for adolescents at end of skeletal growth. In addition, gain understanding of the impact of caring for an adolescent with XLH at the end of growth.
Up to 30 adolescents diagnosed with XLH, who have been treated for at least 12 months with burosumab and up to 15 parent / carers of the same adolescents will be invited to participate, as part of routine care, from specialist paediatric centres in the UK and potentially France, Germany, Spain and the Netherlands. A mix of quantitative and qualitative data will be captured, during two observation periods of the study, before and after end of growth, as determined by the participants physician.
A mixed methods research approach is the most appropriate to meet the research aims. Data from the adolescents will be collected quantitative and qualitative data using a bespoke designed app, wearable device (sport watch) from the hospital notes and via interview. The quantitative data will assess participant reported symptoms, activity intensity and duration, school and activity participation, healthcare resource utilisation and health-related quality of life during a 4-week period prior to end of growth and for 6 months after the end of growth. Interviews with the adolescents at the beginning and end of the study will provide further insights into their experiences and changes with the end of growth. Analysis of the quantitative and qualitative findings will indicate if changes in treatment at the end of growth are associated with change in the symptoms and impact of XLH. No additional visits will be required during the study and their treatment of XLH will be as prescribed by their treating physician after the end of growth and may be the continuation of burosumab or a change in treatment.
REC name
South West - Cornwall & Plymouth Research Ethics Committee
REC reference
21/SW/0081
Date of REC Opinion
9 Aug 2021
REC opinion
Further Information Favourable Opinion