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A UK Multicentre, Health-Related Quality of Life Study for Children and Adolescents With XLH

NCT04819490 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 32

Last updated 2024-07-25

No results posted yet for this study

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.

Conditions

  • X-Linked Hypophosphataemia

Sponsors & Collaborators

  • Kyowa Kirin Pharmaceutical Development Ltd

    lead INDUSTRY

Principal Investigators

  • Kyowa Kirin Medical Affairs Division · Kyowa Kirin, Inc.

Eligibility

Min Age
4 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-04-01
Primary Completion
2022-07-29
Completion
2022-07-29

Countries

  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04819490 on ClinicalTrials.gov