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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Total Enrollment

25 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-11-24

Study Completion Date

2024-05-22

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

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

NCT04819490

X-Linked Hypophosphataemia

COMPLETED

Efficacy and Safety of Burosumab Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With XLH

NCT02915705

X-Linked Hypophosphatemia

COMPLETED PHASE3

X-linked Hypophosphatemia and FGF21

NCT03596554

X-linked Hypophosphatemia

COMPLETED

A Study to Assess the Safety and Efficacy of Leuprorelin in Central Precocious Puberty in Chinese Participants

NCT02427958

Central Precocious Puberty

COMPLETED PHASE4

Effect of Cinacalcet on Parathyroid Hormone Secretion in Children and Adolescents With Hypophosphatemic Rickets

NCT00195936

Hypophosphatemic Rickets, X-Linked Dominant

COMPLETED PHASE1

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

X-Linked Hypophosphatemia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

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.

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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).

* 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.

Minimum Eligible Age

12 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No