Registry of Patients With Hypophosphatasia
This is a long-term study collecting information from people of all ages who have Hypophosphatasia (HPP), a rare condition affecting bones and teeth. It's happening in different countries, including the UK. The main goal is to build a clearer picture of HPP – how it develops, what symptoms people experience, and how it impacts their lives. Researchers are also looking at how effective and safe a specific treatment called Asfotase alfa is for people with HPP. This study is observational, meaning participants are not given new treatments as part of the study; instead, information is collected about the care they are already receiving or would receive. It helps medical professionals better understand HPP and improve care for those living with it.
At a glance
What is this study about?
This study is called a 'registry' for people with a rare condition known as Hypophosphatasia (HPP). HPP affects the development of bones and teeth, causing them to be softer and weaker than they should be. Because HPP is rare, doctors want to learn as much as possible about how it affects people over time, from babies to adults.
The registry is designed to follow people with HPP in the UK and other countries for many years. It's like building a large, detailed library of information about HPP. The researchers won't be giving new medicines or treatments as part of this study. Instead, they will gather details about people's health, their symptoms, how HPP has progressed, and what treatments they are receiving, including one called Asfotase alfa.
By collecting this information, doctors hope to gain a much better understanding of HPP. This will help them to improve how the condition is diagnosed, how it progresses, and ultimately, how to provide the best possible care and support for people living with it.
Key takeaways
- It's a long-term study collecting information about Hypophosphatasia (HPP).
- It aims to better understand HPP and its impact on people of all ages.
- It observes outcomes for people receiving a treatment called Asfotase alfa.
- No new treatments or extra appointments are involved.
- Your medical care will not be affected by joining or leaving the study.
- Your information helps improve future care for people with HPP.
Who may be eligible?
To join this study, you must have a confirmed diagnosis of Hypophosphatasia (HPP). This diagnosis needs to be supported by a blood test showing low levels of a substance called alkaline phosphatase for your age and sex, or by a genetic test that identifies changes in the ALPL gene linked to HPP.
The study welcomes people of all ages, both male and female. If you are a child, your parent or legal guardian would need to agree for you to take part. All participants, or their legal representatives if they are unable, must be able to understand the study information and give their permission to join, either by reading or having it explained to them in their local language.
Essentially, if you or someone you care for has HPP and meets these basic requirements, you might be able to take part. However, you cannot join this study if you are already participating in another clinical trial sponsored by the company Alexion, though joining this registry won't stop you from participating in future trials they might run.
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 Hypophosphatasia (HPP)?
- Is my alkaline phosphatase level below normal, or do I have a confirmed ALPL gene change?
- Am I (or my legal representative) able to understand study information?
- Am I not currently in another Alexion clinical trial?
What does participation involve?
If you decide to join this study, you won't be given any new medication or experimental treatments. Instead, the study will simply collect information about your health as part of your regular medical care for HPP. This means researchers will gather details from your medical records regarding your HPP diagnosis, symptoms, how the condition has progressed over time, and any treatments you are receiving, specifically Asfotase alfa if applicable.
Participation is long-term, meaning information will be collected over many years. There are no extra visits or assessments specifically required for this study beyond what your doctors would normally do for your HPP care. You or your legal representative will be asked to sign an informed consent form and permission to share your medical records. The overall duration of your participation will depend on how long the registry continues to collect data and your willingness to remain involved.
Potential risks and benefits
Locations (64)
- Research SiteVerified postcodeCentennial, United States
- Research SiteVerified postcodeHartford, United States
- Research SiteVerified postcodeTampa, United States
- Research SiteVerified postcodeDecatur, United States
- Research SiteVerified postcodeBaltimore, United States
- Research SiteVerified postcodeBoston, United States
- Research SiteVerified postcodeBoston, United States
- Research SiteVerified postcodeMinneapolis, United States
- Research SiteVerified postcodeKansas City, United States
- Research SiteVerified postcodeSt Louis, United States
- Research SiteVerified postcodeHackensack, United States
- Research SiteVerified postcodeMineola, United States
Common questions
What is Hypophosphatasia (HPP)?
HPP is a rare condition that affects bones and teeth, making them softer and weaker than normal.
Is this study giving new medicine?
No, this study only collects information about your health and the care you are already receiving; it doesn't give new medications.
How long will I be in the study?
This is a long-term study, so information will be collected over many years, following your regular care.
Do I have to attend extra appointments?
No, there are no extra appointments required specifically for this study beyond your usual medical care.
Can I leave the study if I change my mind?
Yes, you can leave the study at any time, and it won't affect your medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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