RecruitingOBSERVATIONAL

Registry for Patients With X-Linked Hypophosphatemia

This international study is collecting important information about X-Linked Hypophosphatemia (XLH) – a condition that affects how your body handles phosphate. It's a 'registry', meaning it gathers details from people with XLH over many years, rather than testing new treatments. The main goal is to build a clearer picture of how XLH affects people's health over time, both in children and adults. Researchers want to learn more about the best ways to treat XLH and how the condition changes as someone gets older. By understanding this better, doctors hope to improve care for everyone living with XLH. Your participation would involve sharing your existing health information, with your permission, to help researchers learn from real-world experiences. It's about observing, not about changing your current medical care.

At a glance

Status

Recruiting

Sponsor

Kyowa Kirin Pharmaceutical Development Ltd

Enrolment target

1,343

Start

12 Sep 2017

Estimated completion

01 Jul 2029

X-Linked Hypophosphatemia

What is this study about?

This study is called a 'registry,' which acts like a secure collection of information from many different people with a specific health condition. In this case, it's focusing on X-Linked Hypophosphatemia, often shortened to XLH. XLH is a rare genetic condition that primarily affects how your body uses and absorbs phosphate, a mineral crucial for strong bones and teeth. Because it's an X-linked condition, it usually affects males more severely, but females can also have it.

The main purpose of this registry is simple: to learn as much as possible about XLH in real-life situations. The researchers want to understand how the condition naturally progresses over many years in both children and adults. They also want to see how different treatments are being used and how effective they are in the long run. By collecting this information from around the world, doctors hope to get a much clearer picture of XLH, which can then help them improve care and develop better guidance for people with the condition in the future.

It's important to know that this is not a study where you'll be given a new medicine or treatment to try. Instead, it's about observing and recording what's already happening with your health and treatment. Your participation helps build a valuable resource for medical professionals and scientists, contributing to a better understanding of XLH for generations to come. Some of the information collected will also be used to help meet requirements from health authorities about the safety of certain XLH medicines.

Key takeaways

This is a study to learn more about XLH in real life.
It collects information from your existing medical records.
You won't receive new treatments or extra tests.
Anyone with XLH can join, regardless of age or current treatment.
Your participation helps improve understanding and care for XLH.
You can leave the study whenever you want to.

Who may be eligible?

To be able to join this registry, you need to meet a few simple requirements. Firstly, you must have X-Linked Hypophosphatemia (XLH). This diagnosis will be confirmed by your doctor based on your health history, medical tests, or genetic results.

There are no age limits – people of all ages, from babies to adults, are welcome to take part. Also, it doesn't matter if you are currently receiving treatment for your XLH or not.

However, you cannot join this registry if you are currently taking part in another clinical trial where you are being given an experimental treatment or medicine. Once your involvement in that trial finishes, and all follow-up appointments are completed, you would then be able to join this XLH registry.

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.

Do I have a confirmed diagnosis of X-Linked Hypophosphatemia (XLH)?
Am I willing to share my medical information from my routine doctor visits?
Am I currently NOT taking part in another medical trial where I am trying an experimental drug?
Do I or my legal guardian have the capacity to understand and agree to participate?

Answer every question to see your result.

What does participation involve?

If you decide to join this registry, it's quite straightforward. Primarily, it involves sharing your existing health information with the research team. This data will come from your regular medical appointments and records. You won't generally need extra visits just for this study; the information will be gathered from your routine care.

There are no specific study medications involved, as this isn't a trial testing a new drug. The study will simply record what treatments you are already receiving as part of your usual care for XLH. There are no extra tests or assessments beyond what your doctor would typically do for your condition.

This is a long-term observational study, meaning researchers will collect information over many years to see how XLH develops and how treatments affect people over time. There isn't a set total duration for your participation; it will continue as long as you wish to be involved and the registry is active. You are free to withdraw at any time.

Potential risks and benefits

In terms of benefits, participating in this registry directly helps advance our understanding of XLH, potentially leading to improved care for yourself and others with the condition in the future. You won't receive immediate personal health benefits or changes to your current treatment as a result of joining. The main risk is a small possibility of your personal health information being accidentally accessed, but strict measures are in place to protect your privacy and keep your data secure and anonymous where possible. Remember, your participation is completely voluntary, and you can withdraw from the registry at any time without affecting your medical care.

Locations (118)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Hopital Universitaire des Enfants Reine Fabiola
Verified postcode
Brussels, Belgium· Recruiting
Universitair Ziekenhuis Brussel
Verified postcode
Brussels, Belgium· Recruiting
Cliniques Universitaires Saint-Luc
Verified postcode
Brussels, Belgium· Recruiting
UZA
Verified postcode
Edegem, Belgium· Recruiting
Universitair Ziekenhuis Gent
Verified postcode
Ghent, Belgium· Recruiting
UZ Leuven
Verified postcode
Leuven, Belgium· Recruiting
CHU de Liège
Verified postcode
Liège, Belgium· Recruiting
SHATPD "Prof Ivan Mitev" EAD
Verified postcode
Sofia, Bulgaria· Active not recruiting
UMHAT "SofiaMed", OOD
Verified postcode
Sofia, Bulgaria· Active not recruiting
Children Hospital
Verified postcode
Brno, Czechia· Active not recruiting
Fakultni nemocnice v Motole
Verified postcode
Prague, Czechia· Active not recruiting
Aalborg University Hospital
Verified postcode
Aalborg, Denmark· Active not recruiting

Common questions

What is X-Linked Hypophosphatemia (XLH)?

XLH is a rare genetic condition that mostly affects how your body handles phosphate, an important mineral for healthy bones and teeth. This can lead to problems like bowed legs, bone pain, and dental issues.

Will I have to take new medicine or get extra tests?

No, this is not a study that tests new medicines or treatments. You won't need any extra tests or appointments beyond your usual medical care for XLH.

Who is running this study?

This is an international collaboration involving many medical centres and researchers globally, aiming to gather comprehensive data on XLH.

How long will I be in the study?

This is a long-term study, gathering information over many years. You can choose to leave the study at any time without it affecting your medical care.

Will my personal details be kept private?

Yes, all your personal information is kept strictly confidential. The data is anonymised as much as possible to protect your privacy.

How to find out more

Clinical Trial Cordinator

KKI-IMA-XLH-Registry@kyowakirin.com +49 15114861167 Official trial record

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