RecruitingOBSERVATIONAL

Alport Therapy Registry - European Initiative Towards Delaying Renal Failure in Alport Syndrome

This ongoing study, called 'Alport Therapy Registry' or 'Alport XXL', aims to understand how various treatments affect people with Alport syndrome. This is a genetic condition that causes kidney damage. Researchers are collecting health information from patients globally to see if certain medicines, like ACE-inhibitors or ARBs, can slow down kidney disease progression, prevent kidney failure, and improve life expectancy. The study started in Europe in 2006 and has now expanded worldwide. It's particularly interested in how early treatment in young patients affects their long-term health, comparing different drug combinations.

At a glance

Status

Recruiting

Sponsor

University Hospital Goettingen

Enrolment target

800

Start

01 Jul 1995

Estimated completion

01 Mar 2036

Alport Syndrome Hereditary Kidney Disease Pediatric Kidney Disease Thin Basement Membrane Disease Familial Benign Hematuria

What is this study about?

Alport syndrome is a serious inherited condition that affects the kidneys. It can lead to kidney failure quite early in life. At the moment, there aren't specific medications approved just for Alport syndrome, but doctors use other medicines that they hope will help. This study, known as the 'Alport Therapy Registry' or 'Alport XXL', is looking into how well these existing medicines work in real life. It's designed to see if different treatments can slow down the kidney problems, delay kidney failure, and help people live longer, healthier lives compared to those who haven't taken these medicines.

This isn't a study where doctors give new treatments to people. Instead, it's an 'observational' study. This means researchers are simply collecting information from people who are already receiving treatments for Alport syndrome as part of their usual care. They've been doing this in Europe since 2006 and more recently expanded globally. They're particularly keen to understand if starting treatment early, especially in children, makes a bigger difference.

The study looks at different stages of kidney problems in Alport syndrome, from early signs like blood in the urine, to more advanced stages. By collecting information from many people over time, they hope to get a clear picture of which treatments, started at which stage, are most effective. This information is really important for guiding future treatment decisions and improving the lives of people with Alport syndrome worldwide.

Key takeaways

This study collects information on existing treatments for Alport syndrome.
It aims to see if medicines can slow down kidney damage and improve life expectancy.
Participation is observational; no new treatments or extra appointments are involved.
Information is collected from people with Alport syndrome worldwide.
Results help doctors understand the best ways to treat Alport syndrome.

Who may be eligible?

To be part of this study, you need to have a confirmed diagnosis of Alport syndrome. This diagnosis can be made through a kidney biopsy (where a small sample of your kidney is taken for examination) or by a genetic test that identifies the specific gene changes causing your Alport syndrome, or both.

Any type of Alport syndrome is welcome, whether it’s called X-linked, autosomal, or digenic. This means people with changes in the COL4A3, COL4A4, or COL4A5 genes can participate.

However, some people cannot participate. If you're not comfortable giving your permission (informed consent) for your health information to be used, you won't be able to join. Also, if you’re suspected of having Alport syndrome but the diagnosis hasn't been fully confirmed yet, you wouldn't be able to join until it is.

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 Alport syndrome?
Has my Alport syndrome been diagnosed by a kidney biopsy or genetic test?
Am I willing to allow my existing health information to be used for research?
Am I receiving treatment for Alport syndrome?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, it simply means that your routine health information and treatment details related to your Alport syndrome will be collected and added to the study's registry. This includes details about any medicines you are already taking for your condition, such as ACE-inhibitors, ARBs, statins, or other listed treatments. You won't be asked to change any of your current medications or treatments, nor will you need to attend any extra appointments specifically for this study. Your participation mainly involves allowing researchers to gather data from your medical records over time to help them understand the long-term effects of different treatments. The duration of your participation would be ongoing, as it's a long-term observational registry.

Potential risks and benefits

Because this is an observational study, there are no new medical treatments or procedures involved, so no direct medical risks from participating. Your existing medical care will continue as usual. The main benefit is knowing that your health information could help researchers better understand Alport syndrome and improve treatments for others in the future. You have the right to withdraw your permission for your data to be used at any time, without it affecting your medical care.

Locations (1)

University Medical Center Göttingen
Verified postcode
Göttingen, Germany· Recruiting

Common questions

What exactly is Alport syndrome?

Alport syndrome is a genetic condition that causes kidney damage, and can also affect hearing and eyesight in some people.

Are they testing new drugs in this study?

No, this study is looking at how existing common medicines, like those for blood pressure, affect people with Alport syndrome in real life.

Will I have to take any extra pills or medicines?

No, you don't take any extra medicines for this study. It simply tracks the treatments you're already receiving as part of your usual care.

How long will my information be used?

This is a long-term study, so your information might be collected over many years to understand the long-term effects of treatments.

Can I stop participating if I change my mind?

Yes, you can withdraw your consent for your information to be used at any time without it affecting your medical care.

How to find out more

Oliver Gross, MD

gross.oliver@med.uni-goettingen.de +49-551-39- Official trial record

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