UK HRQoL study for children and adolescents with XLH v1.0

• Research type

  Research Study

• Full title

  A UK multicentre, non-interventional, observational, Health-Related Quality of Life study for children and adolescents with X-Linked Hypophosphataemia

• IRAS ID

  292647

• Contact name

  Zulf Mughal

• Contact email

  zulf.mughal@cmft.nhs.uk

• Sponsor organisation

  Kyowa Kirin International

• Clinicaltrials.gov Identifier

  NCT04819490, clinicaltrials.gov

• Duration of Study in the UK

  1 years, 7 months, 23 days

• Research summary

  This study aims to provide Health-Related Quality of Life (HRQoL) data from children and adolescents with growing skeleton with XLH in the United Kingdom (UK), including those treated with burosumab or alternative XLH treatment, as part of an updated submission to the Scottish Medicines Consortium (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 be used to derive HRQoL utility estimates in children and adolescents with growing skeletons for Rickets Severity Score health states.

• REC name

  Yorkshire & The Humber - Leeds West Research Ethics Committee

• REC reference

  21/YH/0099

• Date of REC Opinion

  22 Apr 2021

• REC opinion

  Favourable Opinion