Not yet recruitingOBSERVATIONAL

Treatment Patterns, Biochemical Profiles and Clinical Outcomes in Adults With X-Linked Hypophosphatemia

This study, called "Treatment Patterns, Biochemical Profiles and Clinical Outcomes in Adults With X-Linked Hypophosphatemia," aims to gain a better understanding of how X-linked Hypophosphatemia (XLH) impacts the body and daily life. XLH is a condition that affects how your body handles phosphate, which is important for strong bones. The study isn't testing a new treatment; instead, it observes adults who are already taking a medicine called burosumab. Researchers want to see how phosphate levels in these patients change over time and how these changes are linked to bone health, the overall effects of XLH, and any related health problems. By collecting this information, doctors hope to learn more about the condition and how current treatments are working in the real world.

At a glance

Status

Not yet recruiting

Sponsor

Novo Nordisk A/S

Enrolment target

100

Start

07 Jul 2026

Estimated completion

07 Jul 2030

X-linked Hypophosphatemia (XLH)

What is this study about?

This study is designed to help us learn more about a condition called X-linked Hypophosphatemia, or XLH. If you have XLH, your body doesn't handle phosphate properly, which can affect your bones and overall health. Phosphate is a mineral essential for strong bones and teeth, and when levels are too low, it can lead to various symptoms.

The main goal of this study is to understand how XLH affects adults in their daily lives and how their bodies respond to treatment. We're particularly interested in how phosphate levels change in adults who are already taking a specific medication for XLH called burosumab. We want to see if these changes in phosphate levels are connected to how strong their bones are, how much XLH affects their daily life, and any other health conditions linked to XLH.

It's important to know that this study isn't about giving you a new treatment or testing a new medicine. Instead, it's an 'observational' study. This means we'll be carefully watching and collecting information from people who are already on burosumab as part of their regular care. By doing this, we hope to gather valuable insights that can help doctors better understand and manage XLH for many years to come.

Key takeaways

This study observes adults already being treated for XLH with burosumab.
It aims to understand how burosumab affects phosphate levels and overall health in XLH.
No new treatments or medicines are being tested in this study.
Taking part involves sharing information from your routine medical check-ups.
The findings could help improve future understanding and care for people with XLH.
You can withdraw from the study at any time without affecting your medical treatment.

Who may be eligible?

To join this study, you need to be an adult between 18 and 64 years old. You must have been officially diagnosed with XLH, either through genetic testing or other medical checks, and have reliable documents proving this diagnosis.

A key requirement is that you must already be taking burosumab for your XLH and have been on this treatment for at least three months (90 days) beforehand. You'll also need to be willing and able to come to all the study visits for assessments, which will be scheduled as part of your normal medical care.

You can't take part if you've already been screened for this study before. Also, if you're currently in another study where you're trying out a new treatment or medicine, you won't be able to join this one. However, if you're in a study that just collects information, like a patient register, that might be okay. Lastly, if you have any other health conditions that your doctor thinks might make it unsafe for you to participate or make it hard for you to follow the study plan, then you wouldn't be able to join.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you between 18 and 64 years old?
Do you have a confirmed diagnosis of XLH?
Have you been taking burosumab for at least 3 months (90 days)?
Are you able and willing to attend your regular hospital appointments?
Are you currently NOT participating in a study testing a new medicine?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you won't be given any new medication, as this study is just observing your existing treatment. Your involvement will mainly include attending your routine hospital appointments and allowing the study team to collect information from these visits. This information will include details about your phosphate levels, bone health, and how XLH affects you.

There are no extra visits specifically for this study beyond your usual medical check-ups. The study will simply gather data from your standard care. The total length of your participation would depend on how long you remain on burosumab and how long the researchers plan to collect data, but you'll be told a clear timeline if you decide to join.

Potential risks and benefits

Since this is an observational study and you won't be given any new treatments, there are no medical risks directly from the study itself. Any risks or benefits would be related to your ongoing burosumab treatment, which your doctor has already discussed with you. A potential benefit of taking part is that the information collected from you and other participants could help doctors better understand XLH and improve care for people with the condition in the future. You are always free to change your mind and withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (23)

Yale University School of Medicine
Verified postcode
New Haven, United States
Indiana University Hospital
Verified postcode
Indianapolis, United States
The Ohio State University Wexner Medical Center (OSUWMC) - CarePoint East
Verified postcode
Columbus, United States
Vanderbilt University Medical Center
Verified postcode
Nashville, United States
Northern Sydney Cancer Centre-Royal North Shore Hospital (RNSH)
Verified postcode
St Leonards, Australia
Westmead Hospital
Verified postcode
Westmead, Australia
Monash University - Monash Medical Centre (MMC)
Verified postcode
Clayton, Australia
Bone Research and Education Centre
Verified postcode
Oakville, Canada
CHU de Bicetre
Approximate
Le Kremlin, France
Hopital Cochin - APHP
Verified postcode
Paris, France
Charite Campus Virchow
Verified postcode
Berlin, Germany
Endokrinologikum Goettingen
Verified postcode
Göttingen, Germany

Common questions

What is X-linked Hypophosphatemia (XLH)?

XLH is a condition that affects how your body handles phosphate, a mineral important for strong bones and teeth. It can lead to low phosphate levels, affecting bone health and other areas of your body.

Am I being given a new medicine in this study?

No, this study is not testing a new medicine. It's observing people who are already taking burosumab as part of their regular treatment for XLH.

Why are phosphate levels important for this study?

Phosphate levels are key in managing XLH. This study wants to see how these levels change in people on burosumab and how they relate to bone health and other XLH-related problems.

Will I have extra doctor's appointments if I join?

No, you won't have extra appointments. The study will collect information from your routine medical visits for your XLH care.

What kind of information will be collected about me?

The study will collect information from your medical records about your phosphate levels, bone health, and how XLH generally affects you, among other things, all as part of your standard care.

How to find out more

Novo Nordisk

clinicaltrials@novonordisk.com (+1) 866-867-7178 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.