RecruitingOBSERVATIONAL

Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

This study is looking at Friedreich ataxia, a rare genetic disease that affects the nervous system and heart. It's a long-term project that brings together information from two earlier studies, making it bigger and more powerful. The main goal is to understand how the disease changes over time, which is called its 'natural history'. By doing this, scientists hope to find better ways to design clinical trials and develop new medicines and treatments. People of any age with a confirmed diagnosis of Friedreich ataxia can take part. If you join, you would have yearly check-ups for up to 25 years. These check-ups would involve looking at your medical records, doing neurological tests, and assessing your movement, arm function, and how your quality of life is affected.

At a glance

Status

Recruiting

Sponsor

Friedreich's Ataxia Research Alliance

Enrolment target

3,000

Start

28 Jun 2023

Estimated completion

28 Jan 2048

Friedreich Ataxia

What is this study about?

This study is all about understanding Friedreich ataxia better. Friedreich ataxia is a rare condition that you're born with, affecting your nerves and heart. Because it's quite rare, doctors and scientists need to collect a lot of information to truly understand how it progresses over time and what symptoms people experience. This knowledge is crucial for developing safe and effective treatments in the future.

This study, called UNIFAI, is a huge group effort, bringing together researchers from all over the world. It combines information from two big studies that have been running for a long time in different parts of the world. By joining forces, they can learn even more. They will follow people with Friedreich ataxia for many years, carefully observing how the condition affects their bodies and daily lives. This long-term monitoring helps them track changes and spot important patterns.

The information gathered will be incredibly valuable. It helps researchers understand what to look for when they're testing new medicines and how to measure if a new treatment is actually working. Essentially, it lays the groundwork for future breakthroughs that could improve the lives of people with Friedreich ataxia.

Key takeaways

This study helps understand Friedreich ataxia better for future treatments.
It's a long-term 'observation' study, not testing new drugs.
Participation involves yearly clinic visits for up to 25 years.
You'll have physical tests and answer questions about your health.
It's open to people of all ages with a confirmed diagnosis.

Who may be eligible?

This study welcomes both men and women of all ages. To join, you must have a confirmed diagnosis of Friedreich ataxia, meaning genetic tests have shown you have the condition. If you wish to take part, you'll need to give your informed consent, which means you understand what the study involves and agree to participate.

If you are under 18, both you and a parent or legal guardian will need to sign consent forms. If an adult isn't able to make decisions for themselves, their legal representative can provide consent for them.

You cannot join the study if you're unable or unwilling to give your consent, or if you have other ongoing medical problems that would make it difficult to take part in the study or complete the assessments. The study doctors also need to believe you'll be able to stick to the study's requirements.

Quick self-check

Do I have a confirmed genetic diagnosis of Friedreich ataxia?
Am I able and willing to give informed consent (or have it given by a legal representative)?
Are there any other serious health conditions present that might interfere with participating?
Am I able to attend yearly study visits?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you won't be given any new medications or treatments. Instead, the study is focused on observing your health over time. You would have a study visit once a year, and these visits could continue for up to 25 years.

During these yearly visits, the study team will collect information from your medical records and discuss your health history. They will also carry out various assessments. These include neurological exams to check your reflexes and coordination, tests to see how you walk, measures of how well you use your arms, and questionnaires about your overall quality of life. Some clinics might also gather extra information about your speech, vision, tiredness, balance, and thinking abilities.

Potential risks and benefits

Participating in this study won't offer a direct medical benefit as no new treatments are being given. However, the information gathered will be incredibly valuable for the entire Friedreich ataxia community, helping researchers understand the condition better and develop future treatments. The risks are generally low, mainly involving the time commitment for yearly visits and the minor discomfort of some physical assessments. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (34)

UCLA Ataxia Center
Los Angeles, United States· Recruiting
University of Colorado
Denver, United States· Recruiting
University of Florida - Neurology
Gainesville, United States· Recruiting
USF Ataxia Research Center
Tampa, United States· Recruiting
Emory University Hospital - Neurology
Atlanta, United States· Recruiting
University of Iowa, Stead Family Children's Hospital
Iowa City, United States· Recruiting
Ohio State University - Neurology
Columbus, United States· Recruiting
Children's Hospital of Philadelphia
Philadelphia, United States· Recruiting
St. Jude Children's Research Hospital
Memphis, United States· Recruiting
Murdoch Childrens Research Institute
Parkville, Australia· Recruiting
Medical University Innsbruck, Department of Neurology
Innsbruck, Austria· Recruiting
Université Libre de Bruxelles, Hôpital Erasme, Dpt of Neurology
Brussels, Belgium· Recruiting

+22 more sites — see the official record for the full list.

Common questions

What is Friedreich ataxia?

It's a rare genetic condition that affects the nervous system and heart, causing problems with movement, balance, and other body functions.

Why is this study important if no new drugs are given?

It helps researchers understand how Friedreich ataxia progresses over many years, which is essential for designing future treatment studies and finding new medicines.

How often do I need to visit the clinic?

You would have one study visit each year.

How long will I be in the study?

You could be in the study for up to 25 years, with annual visits.

Can children participate?

Yes, people of all ages can participate, as long as they and their legal guardian give consent.

How to find out more

Cait Monette

cait.monette@cureFA.org 16513291892 Official trial record

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