Natural History of Wilson Disease
This research is all about understanding Wilson Disease better. It's a rare genetic condition where copper builds up in the body, potentially damaging organs. We are collecting information and samples from people with Wilson Disease to learn how the condition progresses over time and how different treatments work. The main goals are to find the most accurate ways to diagnose Wilson Disease and the best ways to monitor treatment effectiveness. This will help doctors make better decisions about care now, and also support the development of new treatments in the future. If you have Wilson Disease, your health information could help researchers improve life for many others living with this condition.
At a glance
What is this study about?
This research project is designed to help us learn more about Wilson Disease, which is a rare, inherited condition where your body stores too much copper. Over time, this excess copper can cause serious problems for organs like your liver and brain. Right now, we want to gather information from people living with Wilson Disease to understand how the condition naturally behaves and progresses over many years.
We also want to find the best ways to pinpoint when someone has Wilson Disease and how to tell if their treatment, such as 'chelation therapy' or zinc, is working well. By looking at different tests and measurements from many patients, we hope to identify clear signs that can help doctors confirm a diagnosis and adjust treatments more effectively.
Ultimately, this study aims to create better tools for monitoring Wilson Disease. We're looking to see if a simple score or measurement might serve as a reliable indicator of how well someone is doing on their treatment. This could make it easier to test new treatments in the future and ensure that patients receive the most beneficial care possible.
Key takeaways
- It's a research project to understand Wilson Disease better.
- Aims to find best ways to diagnose and monitor treatments.
- Involves collecting health information from patients.
- No new treatments or extra clinic visits for participants.
- Your data could help improve future care for Wilson Disease patients.
Who may be eligible?
To be part of this study, you must have already been diagnosed with Wilson Disease by a doctor. This is the most important requirement.
If you are an adult, you need to be willing and able to give your permission to join the study. If you are under 18, your parent or guardian will need to give permission, and you might also need to agree to take part yourself, depending on your age and local rules.
You cannot join the study if doctors have definitely said you do not have Wilson Disease, or if you or your guardian are not willing to provide permission to participate.
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 Wilson Disease?
- Am I, or my guardian, willing to give permission to participate?
- Am I currently receiving care for Wilson Disease?
- Have I been told I do not have Wilson Disease?
What does participation involve?
This study involves sharing your health information and possibly some samples, which will be collected during your regular check-ups for Wilson Disease. You won't need extra hospital visits specifically for this study. The research team will gather data from your existing medical records over time.
There are no new medicines or treatments given as part of this study; you will continue with your usual care as recommended by your doctor. The duration of your participation will depend on how long you continue to be followed at a specialist clinic for your Wilson Disease. It is mainly about collecting information over time.
Potential risks and benefits
Locations (6)
- Yale UniversityVerified postcodeNew Haven, United States· Recruiting
- Advent HealthVerified postcodeOrlando, United States· Recruiting
- Baylor College of MedicineVerified postcodeHouston, United States· Recruiting
- Seattle Children's HospitalVerified postcodeSeattle, United States· Recruiting
- Universitätsklinikum HeidelbergVerified postcodeHeidelberg, Germany· Recruiting
- Royal Surrey Country HospitalVerified postcodeGuildford, United Kingdom· Recruiting
Common questions
What is Wilson Disease?
It's a rare genetic condition where your body can't remove extra copper, causing it to build up and potentially harm organs like your liver and brain.
Will I get any new treatments in this study?
No, this study doesn't involve any new treatments or extra medication. You'll continue with your usual care for Wilson Disease.
How long will I be in the study?
Participation duration depends on how long you are followed at your specialist clinic for Wilson Disease, as the study collects information from your ongoing care.
Do I need to visit the hospital more often?
No, you won't need extra visits for this study. Information will be collected during your standard check-ups and from your existing medical records.
Can I leave the study at any time?
Yes, you can choose to leave the study at any point, and it won't affect your medical treatment or care.
How to find out more
Ricarda Tomlin
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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