Active not recruitingOBSERVATIONAL

A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA)

The TRACK-FA study is investigating how Friedreich Ataxia (FA) affects the brain and spinal cord over two years. Researchers will use special brain and spinal cord scans, along with other health and memory tests, to see how the disease progresses in about 200 people with FA. They will compare these findings to about 100 healthy volunteers. There are currently no cures for FA, but new treatments are being developed. This study aims to find better ways to measure if these new treatments are working in future clinical trials. By understanding how FA changes the body, especially the nervous system, this study hopes to speed up the discovery of effective therapies. It's a collaborative effort across several international research centres.

At a glance

Status

Active not recruiting

Sponsor

Monash University

Enrolment target

300

Start

10 Feb 2021

Estimated completion

01 Oct 2025

Friedreich Ataxia

What is this study about?

Friedreich Ataxia (FA) is a condition that affects many parts of the body, but mainly the central nervous system, which includes your brain and spinal cord. People with FA often experience problems with balance and coordination, difficulty speaking, swallowing, and sometimes issues with vision and hearing. Other symptoms can include heart problems, diabetes, and tiredness. The symptoms usually start between the ages of 5 and 15, and they can worsen over time.

Currently, there isn't a cure for FA or any treatments that can slow down its progression. However, scientists are working hard to develop new medicines. A big challenge in testing these new treatments is finding reliable ways to measure if they are actually working. This is where the TRACK-FA study comes in. It aims to develop better ways to track the changes in the brain and spinal cord caused by FA, using advanced scans. These measurements could then be used in future studies to see if new treatments are making a difference.

By carefully studying a large group of people with FA and comparing them to healthy individuals over two years, researchers hope to create a valuable collection of information. This database will help scientists around the world better understand FA and more effectively test potential new treatments. It's a team effort involving several universities and research groups, all working together to improve the lives of people with Friedreich Ataxia.

Key takeaways

A two-year study tracking changes in the brain and spinal cord in people with Friedreich Ataxia.
Aims to find better ways to measure disease progression for future drug trials.
Involves regular MRI scans, clinical tests, and blood samples.
Does not include any experimental treatments.
Compares people with FA to healthy volunteers.
Your participation helps speed up the search for new FA treatments.

Who may be eligible?

To join the TRACK-FA study, you must be at least 5 years old. If you have Friedreich Ataxia, you’ll need to have a confirmed genetic test result showing the specific changes linked to FA. Your FA symptoms should have started by the age of 25, and you should have had the condition for no more than 25 years. Also, your current FA symptoms, based on a special rating scale, should fall within a certain range.

There are also reasons why someone might not be able to join. For example, if you are under 5, can't give your consent to take part, or have certain metal implants in your body (like a pacemaker) that would make MRI scans unsafe. Pregnant individuals cannot participate. It’s also important that you don't have other serious medical conditions or another neurological problem that might interfere with the study's results.

If you're a healthy volunteer, you must not have any diagnosed mental health or neurological conditions. You also shouldn't have any other ongoing medical conditions that could affect the study. If you have a sibling with FA, you cannot join as a healthy volunteer unless your genetic status (whether you carry a gene for FA) is already known.

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.

Are you at least 5 years old?
If you have FA, do you have a confirmed genetic diagnosis?
If you have FA, did your symptoms start by age 25 and have you had FA for 25 years or less?
Do you have any metal implants (like a pacemaker) that would make MRI scans unsafe?
Are you pregnant or could you be pregnant?
Are you a healthy volunteer without any diagnosed neurological or psychiatric conditions?

Answer every question to see your result.

What does participation involve?

If you decide to join the TRACK-FA study, you'll be involved for a total of two years. During this time, you will have three main assessment visits: one at the beginning, another after 12 months, and a final one after 24 months. Each visit will involve several assessments.

These assessments include advanced scans of your brain and spinal cord (MRI scans) to see how things are changing. You will also undergo various clinical tests to check your balance, coordination, and other physical abilities related to FA. There will be memory and thinking tests, and you may also be asked to provide blood samples for further research. There are no medications involved in this study, as it's purely observational to understand the disease progress. The researchers will not tell you to change any existing treatments you are on.

Potential risks and benefits

Participating in research studies like TRACK-FA has potential benefits, though it won't directly treat your FA. You will receive detailed health assessments and scans, which could provide valuable information about your condition. Your contribution will also be crucial for advancing scientific understanding of FA, helping to develop better ways to measure the disease and speeding up the search for effective treatments for others in the future. The risks of participating are generally low; they primarily include the small risks associated with MRI scans (such as claustrophobia or discomfort from noise) and blood draws (like bruising or mild pain). You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (7)

University of Florida
Verified postcode
Gainesville, United States
Center for Magnetic Resonance Research, University of Minnesota
Verified postcode
Minneapolis, United States
Children's Hospital of Philadelphia
Verified postcode
Philadelphia, United States
Monash Biomedical Imaging, Monash University
Verified postcode
Clayton, Australia
Lab of Neuroimaging and Dept of Neurology, University of Campinas (UNICAMP)
Verified postcode
São Paulo, Brazil
McGill University
Verified postcode
Montreal, Canada
Department of Neurology, RWTH Aachen University
Verified postcode
Aachen, Germany

Common questions

What is a 'natural history' study?

It means the study tracks how a condition normally progresses over time without giving any experimental treatments. It helps us understand the disease better.

Will I get any medicine or treatment in this study?

No, this study does not involve any experimental medications or treatments. It's about observing how Friedreich Ataxia affects the body over time.

How long will I need to be involved?

If you join, you will be part of the study for two years, with three main visits scheduled at the beginning, 12 months, and 24 months.

Are the scans safe?

Yes, MRI scans are generally very safe. However, they use strong magnets, so people with certain metal implants cannot have them. The study team will check for this.

What happens to my information after the study?

Your information will be anonymised (so you can't be identified) and added to a secure database. This data will then be used by researchers to learn more about FA and develop new treatments.