A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth

X-Linked Hypophosphatemia Clinical Trial

Official title:

An Observational, Prospective, European, Multicentre, Mixed Methods Study to Describe the Lived Experience of X-Linked Hypophosphatemia (XLH) for Adolescents at End of Skeletal Growth

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05181839
• Other study ID #: 2020-70-EU-CRY
• Status: Completed
• Start date: November 24, 2021
• Est. completion date: May 22, 2024

Study information

• Verified date: June 2024
• Source: Kyowa Kirin Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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 skeletal growth.

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-treated adolescents with XLH at the end of skeletal growth. The study will involve two observation periods around the confirmed date of end of skeletal growth (index date)

The purpose of this study is to describe the lived experience of XLH for adolescents who are being treated with burosumab at the end of skeletal growth, with a focus on adolescent reported symptoms, activity duration and intensity, and wider burden, and to describe change over time for those who continue and discontinue burosumab at the end of skeletal growth. The study will also explore the experiences of carers at the time the adolescent reaches the end of skeletal growth.

The key objectives of this study are to:

1. Describe the lived experience of adolescents with XLH who are being treated with burosumab within the 12 weeks prior to reaching the end of skeletal growth.

2. Describe the lived experience of adolescents with XLH during the 26 weeks immediately after the end of skeletal growth, overall and according to whether they continue or discontinue burosumab treatment.

3. Describe within-person changes in the lived experience of adolescents with XLH after reaching end of skeletal growth, in relation to their own pre-end of skeletal growth period.

4. Explore the supportive care needs and burden on carers at the time the adolescent with XLH reaches end of skeletal growth.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: May 22, 2024
• Est. primary completion date: May 22, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 17 Years

Eligibility

Inclusion Criteria:

• 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 Inclusion Criteria:

• 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 Criteria:

• 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.

Related Conditions & MeSH terms

• Familial Hypophosphatemic Rickets
• Hypophosphatemia
• X-Linked Hypophosphatemia

Intervention

Drug:
• Burosumab
To describe the lived experience of XLH for adolescents who are being treated with burosumab at the end of skeletal growth with a focus on adolescent-reported symptoms, activity duration and intensity, and wider burden, describing change over time for those who continue and discontinue burosumab after the end of skeletal growth.

Locations

Country	Name	City	State
France	Centre Hospitalier Universitair de Lille	Lille
France	Hospices Civils De Lyon	Lyon
France	APHP Paris - Assistance Publique Hopitaux de Paris	Paris
Germany	Charité - Universitätsmedizin Berlin	Berlin
Netherlands	University Medical Center Groningen - Beatrix Children's Hospital	Groningen
Spain	Hospital Saint Joan de Deu	Barcelona	Catalona
Spain	Hospital Universitari Vall d'Hebron	Barcelona	Catalonia
Spain	Hospital General Universitario Santa Lucía	Cartagena	Murcia
Spain	Hospital Universitario Virgen a de las Nieves	Granada
United Kingdom	Birmingham Women's and Children's Hospital	Birmingham
United Kingdom	Bristol Royal	Bristol
United Kingdom	Alder Hey Children's Hospital	Liverpool
United Kingdom	Great Ormond Street Hospital	London
United Kingdom	Royal Manchester Childrens Hospital	Manchester	Greater Manchester

Sponsors (1)

Lead Sponsor	Collaborator
Kyowa Kirin Pharmaceutical Development Ltd

Countries where clinical trial is conducted

France,  Germany,  Netherlands,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Describe the lived experience of adolescents with XLH who are being treated with burosumab within the 12 weeks prior to reaching the end of skeletal growth.		12 Weeks
Primary	Describe the lived experience of adolescents with XLH during the 26 weeks immediately after the end of skeletal growth, overall and according to whether they continue or discontinue burosumab treatment.		26 weeks
Primary	Describe within-person changes in the lived experience of adolescents with XLH after reaching end of skeletal growth, in relation to their own pre-end of skeletal growth period.		Up to 52 weeks
Primary	Explore the supportive care needs and burden on carers at the time the adolescent with XLH reaches end of skeletal growth.		will be completed between weeks 21 and 25