Status:
COMPLETED
A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth
Lead Sponsor:
Kyowa Kirin Pharmaceutical Development Ltd
Conditions:
X-Linked Hypophosphatemia
Eligibility:
All Genders
12-17 years
Brief Summary
An observational, prospective, mixed-methods study involving the integration of quantitative and qualitative data exploring the lived experience of burosumab-treated adolescents with XLH at the end of...
Detailed Description
This an observational, prospective, European, multicentre, mixed methods study that will involve the integration of quantitative and qualitative data exploring the lived experience of burosumab-treate...
Eligibility Criteria
Inclusion
- Confirmed diagnosis of XLH (documented diagnosis of XLH in medical records, and evidence of at least one of the following: hypophosphataemia and/or impaired phosphate reabsorption due to elevated FGF23; PHEX mutation).
- Aged 12 to 17 years at start of study.
- Has open growth plates at enrolment and is estimated by their treating clinician to reach end of skeletal growth within the next 26 weeks (based on clinician's judgement in accordance with their normal approach used in routine practice).
- Has been receiving treatment with burosumab for at least study le (52 weeks).
- Provides informed consent to take part in the study (or provides assent, and carer provides consent, where applicable in accordance with specific country regulations).
- Carer
- A main carer of a study participant (i.e. a parent or guardian who provides day-today support or care for the adolescent with XLH who is taking part in this study).
- Provides informed consent to take part in the study (for self and/or on behalf of eligible adolescent, where applicable in accordance with specific country regulations).
Exclusion
- Unwilling and unable to participate in all aspects of the study (i.e. interviews, app, EQ- 5D-Y, wearable data collection) and /or does not agree to the collection of data from medical records.
- Missed two or more injections of burosumab in the past 6 months.
- Is planned to have any surgery during the study period.
Key Trial Info
Start Date :
November 24 2021
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
May 22 2024
Estimated Enrollment :
25 Patients enrolled
Trial Details
Trial ID
NCT05181839
Start Date
November 24 2021
End Date
May 22 2024
Last Update
September 4 2024
Active Locations (14)
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1
Centre Hospitalier Universitair de Lille
Lille, France
2
Hospices Civils De Lyon
Lyon, France
3
APHP Paris - Assistance Publique Hopitaux de Paris
Paris, France
4
Charité - Universitätsmedizin Berlin
Berlin, Germany, 10117