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What It's really like: teens with XLH share their journey as bones stop growing

NCT ID NCT05181839

Knowledge-focused Sponsor: Kyowa Kirin Pharmaceutical Development Ltd Source: ClinicalTrials.gov ↗

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 13, 2026 · Last updated Jul 14, 2026 · Updated 1 time

Summary

This study follows about 25 adolescents aged 12–17 with X-Linked Hypophosphatemia (XLH) who are being treated with burosumab and are nearing the end of their skeletal growth. Researchers will track symptoms, activity levels, and overall burden through surveys and interviews over 26 weeks after growth plates close. The goal is to understand how their lived experience changes—whether they continue or stop burosumab—and to identify what support they and their caregivers need during this transition.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
burosumab
What this could lead to
If successful, this could help doctors and families understand what adolescents with XLH go through during this key growth stage, potentially guiding better support and treatment decisions.
What could go wrong
This is a small observational study (25 people) that describes experiences rather than testing a new treatment. It may not apply to all patients, and results depend on honest reporting from participants.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

25 people

The number who actually took part.

Started

Nov 2021

Finished

May 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

The population for this study is adolescents with XLH approaching end of skeletal growth, who are being treated with burosumab for at least 12 months as part of routine clinical care at specialist paediatric centres within the UK, France, Germany, Spain and the Netherlands.

Ages

12 to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

X-linked dominant hypophosphatemic rickets X-linked hypophosphatemic rickets

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

X-linked hypophosphatemia

Contacts and locations

Locations

  • APHP Paris - Assistance Publique Hopitaux de Paris

    Paris, France

  • Alder Hey Children's Hospital

    Liverpool, United Kingdom

  • Birmingham Women's and Children's Hospital

    Birmingham, United Kingdom

  • Bristol Royal

    Bristol, United Kingdom

  • Centre Hospitalier Universitair de Lille

    Lille, France

  • Charité - Universitätsmedizin Berlin

    Berlin, State of Berlin, 10117, Germany

  • Great Ormond Street Hospital

    London, United Kingdom

  • Hospices Civils De Lyon

    Lyon, France

  • Hospital General Universitario Santa Lucía

    Cartagena, Murcia, 30202, Spain

  • Hospital Saint Joan de Deu

    Barcelona, Catalona, 08907, Spain

  • Hospital Universitari Vall d'Hebron

    Barcelona, Catalonia, 08035, Spain

  • Hospital Universitario Virgen a de las Nieves

    Granada, Granada, 18014, Spain

  • Royal Manchester Childrens Hospital

    Manchester, Greater Manchester, M13 9WL, United Kingdom

  • University Medical Center Groningen - Beatrix Children's Hospital

    Groningen, Netherlands

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