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This study aims to provide Health-related Quality of Life (HRQoL) data from children and adolescents with growing skeletons in the United Kingdom (UK), including those treated with burosumab or alternative XLH treatment, as part of an updated submission to the SMC in early 2023. This study will utilise data from a subset of UK sites already within the XLH Registry (including participating Scottish sites) and collect additional HRQoL data within these sites (that are otherwise not included in the wider XLH Registry protocol). The HRQoL data will enable the calculation of HRQoL to derive the HRQoL utility estimates in children and adolescents with growing skeletons for the RSS health states, hence addressing an area of uncertainty.
Full description
This is a multicentre, non-interventional HRQoL study of children and adolescents with growing skeletons using routinely collected XLH Registry data and prospectively collected HRQoL data. This study will aim to enrol approximately 50 patients from approximately 5 UK centres (currently participating in the XLH Registry) over a 6-month period. Each patient will be followed for up to 12 months after enrolment. As stipulated, the data collection should not require additional patient visits or data collection within the health service.
Three types of data will be used in this study:
Enrollment
Sex
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Volunteers
Inclusion criteria
Aged ≥4 years and <18 years.
Enrolled in the XLH Registry via one of the participating UK centres.
Have at least one of the following:
Receiving any XLH treatment (burosumab, oral phosphate and active vitamin D, or other XLH treatment) within 30 days prior to informed consent for this study
Exclusion criteria
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Data sourced from clinicaltrials.gov
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