X-Linked HypophosphataemiaNCT04819490
A UK Multicentre, Health-Related Quality of Life Study for Children and Adolescents With XLH
An observational study of X-Linked Hypophosphataemia, run by Kyowa Kirin Pharmaceutical Development Ltd at 6 sites in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 to 18 years
- Conditions the study is for
- X-Linked Hypophosphataemia
- How many people
- 32 participants
- Where
- 6 sitesUnited Kingdom
- Study dates
- 1 Apr 2021to 29 Jul 2022
- How long will I take part?
- 6 Months
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: 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.
Who may be able to join
- 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
Who cannot join
- 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.
- 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.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United Kingdom6 sites
Royal Hospital for Sick Children
Edinburgh, EH9 1LF
NHS Greater Glasgow & Clyde- South Glasgow University Hosp Division
Glasgow, G12 0XH
Leeds Teaching Hospitals NHS Trust, Trust Headquarters, St. James's University Hospital
Leeds, LS9 7TF
Evelina Childrens Hospital
London, SE1 7EH
Royal Manchester Children's Hospital
Manchester, M13 9WL
Sheffield Childrens Hospital
Sheffield, S10 2RX
About this study
Official title: A UK Multicentre, Non-interventional, Observational, Health-Related Quality of Life Study for Children and Adolescents With X-linked Hypophosphataemia
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.
Read the detailed 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:
- Routinely collected data from the XLH Registry on patient demographics and clinical data, as well as PedsQL HRQoL data.
- HRQoL data prospectively collected at enrolment, 6-month and 12-month follow-up by the patient and/or their parent / legal guardian.
- RSS scores within the XLH Registry will be used where available, or RSS will be centrally calculated retrospectively from radiographs of the affected wrist and/or knee. All RSS calculations will be centrally reviewed.
- Conditions
- X-Linked Hypophosphataemia
- Keywords
- Crysvita, Burosumab, X-linked Hypophosphataemia (XLH), PHEX Gene Mutation
- Medical subject headings
- Familial Hypophosphatemic Rickets
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: Changes from baseline, 6 months and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Mar 2021
- Study start1 Apr 2021
- Primary completion29 Jul 2022
- Study completion29 Jul 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for X-Linked HypophosphataemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Other
- Enrolment
- 32 participants (actual)
- Target duration
- 6 Months
Sponsor and investigators
- Lead sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Kyowa Kirin Medical Affairs Division, Study director, Kyowa Kirin, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04819490
- Sponsor's protocol ID
- 2020-55-UK-CRY
- Status last verified
- Jul 2024
- First submitted
- 18 Mar 2021
- Last updated
- 25 Jul 2024
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A UK Multicentre, Non-interventional, Observational, Health-Related Quality of Life Study for Children and Adolescents With X-linked Hypophosphataemia
- ClinicalTrials.gov ID
- NCT04819490
- Acronym / sponsor ID
- 2020-55-UK-CRY
- Conditions
- X-Linked Hypophosphataemia
- Keywords
- Crysvita, Burosumab, X-linked Hypophosphataemia (XLH), PHEX Gene Mutation
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Kyowa Kirin Pharmaceutical Development LtdIndustry (pharma, biotech, devices)
- Study start
- 1 Apr 2021 (actual)
- Primary completion
- 29 Jul 2022 (actual)
- Study completion
- 29 Jul 2022 (actual)
- First posted
- 29 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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:
- Routinely collected data from the XLH Registry on patient demographics and clinical data, as well as PedsQL HRQoL data.
- HRQoL data prospectively collected at enrolment, 6-month and 12-month follow-up by the patient and/or their parent / legal guardian.
- RSS scores within the XLH Registry will be used where available, or RSS will be centrally calculated retrospectively from radiographs of the affected wrist and/or knee. All RSS calculations will be centrally reviewed.
Study design
- Observational model
- Other
- Time perspective
- Other
- Target follow-up
- 6 Months
Who may qualify
- Ages
- 4 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- 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.
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (3 listed)
- 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.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
To derive health state utilities based on Rickets Severity Score (RSS), in the study population
Time frame: Changes from baseline, 6 months and 12 months
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.
To derive health state utilities based on EQ-5D-Y in the study population
Time frame: Changes from baseline, 6 months and 12 months
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.
To derive health state utilities based on Child Health Utility 9 Dimensions [CHU-9D], in the study population
Time frame: Changes from baseline, 6 months and 12 months
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kyowa Kirin Medical Affairs Division study director
Kyowa Kirin, Inc.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of X-Linked Hypophosphataemia, run by Kyowa Kirin Pharmaceutical Development Ltd at 6 sites in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To derive health state utilities based on Rickets Severity Score (RSS), in the study population (time frame: Changes from baseline, 6 months and 12 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Jul 2024.