A UK Multicentre, Health-Related Quality of Life Study for Children and Adolescents With XLH (NCT04819490) | Clinical Trial Compass
CompletedNot Applicable
A UK Multicentre, Health-Related Quality of Life Study for Children and Adolescents With XLH
United Kingdom32 participantsStarted 2021-04-01
Plain-language summary
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.
Who can participate
Age range4 Years – 18 Years
SexALL
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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:
✓. RSS calculated at enrolment during a routine clinic visit. OR
✓. Historical radiographs of the affected wrist and/or knee available in the medical chart within the 6 months prior to enrolment, to allow RSS to be centrally calculated retrospectively.
✓. 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
✕. Does not have the cognitive capacity to provide informed consent, or their legally designated representative (i.e. parent / legal guardian) does not have the cognitive capacity to provide informed consent.
✕. Is not expected to have open growth plates for the duration of the study.
✕
What they're measuring
1
To derive health state utilities based on Rickets Severity Score (RSS), in the study population
Timeframe: Changes from baseline, 6 months and 12 months
2
To derive health state utilities based on EQ-5D-Y in the study population
Timeframe: Changes from baseline, 6 months and 12 months
3
To derive health state utilities based on Child Health Utility 9 Dimensions [CHU-9D], in the study population
Timeframe: Changes from baseline, 6 months and 12 months
. Currently participating in an interventional clinical trial. Participation in a Compassionate Use Programme, Pre-commercial Programme, or Investigator Initiated Study does not preclude a patient from participation in this study.