An observational study in X-Linked Hypophosphataemia, sponsored by Kyowa Kirin Pharmaceutical Development Ltd. Completed at 6 sites in United Kingdom. Open to participants aged 4 Years to 18 Years. Per ClinicalTrials.gov, last updated 2024-07-25.
Sponsored by Kyowa Kirin Pharmaceutical Development Ltd (part of Kyowa Kirin Co., Ltd.) · Observational
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.
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:
49 studies on the registry are indexed under Familial Hypophosphatemic Rickets; 8 are open to participants now.
This study's enrollment of 32 is below the median of 50 across 16 observational studies indexed under Familial Hypophosphatemic Rickets.
Browse Familial Hypophosphatemic Rickets studies →Kyowa Kirin Pharmaceutical Development Ltd is the lead sponsor of 13 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
The study will aim to enrol approximately 50 patients from at least 5 UK centres (currently participating in the XLH Registry).
Subjects will be receiving any XLH treatment (burosumab, oral phosphate and active vitamin D or another XLH treatment) within 30 days or prior to informed consent.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Have at least one of the following:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
To derive health state utilities based on Rickets Severity Score (RSS), in the study population
The RSS is a quantitative method to assess rickets severity in the wrists and knees based on the degree of metaphyseal fraying, concavity, and the proportion of the growth plate affected. It is a 10-point scale, where 10 represents the most extreme degree of rickets severity. In nutritional rickets, RSS may range from 0.5 to 10.
Time frame: Changes from baseline, 6 months and 12 months
To derive health state utilities based on EQ-5D-Y in the study population
The EQ-5D-Y is a two-part instrument: the EQ-5D descriptive system and the EQ visual analogue scale (VAS). The descriptive system covers five health domains: mobility, looking after myself, doing usual activities, having pain or discomfort and feeling worried, sad or unhappy. Each domain has 3 response categories: no problems, some problems and a lot of problems. The response categories can be reflected by a 1-digit number (1-3) and combined for the five dimensions into a 5-digit number to describe the health state of the patient. The EQ VAS records the patient's self-rated health on a vertical VAS of 0-100 where 100="The best health you can imagine" and 0="The worst health you can imagine". The VAS can be used as a quantitative measure of health outcome that reflects the patient's own judgement; results can be presented as both a measure of the central tendency and a measure of dispersion.
Time frame: Changes from baseline, 6 months and 12 months
To derive health state utilities based on Child Health Utility 9 Dimensions [CHU-9D], in the study population
CHU-9D is a generic child HRQoL questionnaire consisting of nine items, each with five response categories. Each item covers a different domain: worry, sadness, pain, tiredness, annoyance, school, sleep, daily routine and activities.
Time frame: Changes from baseline, 6 months and 12 months
This study is completed, as verified in Jul 2024. You cannot join it, but the record below documents what was studied.
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Familial Hypophosphatemic Rickets →
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