Active — Not RecruitingNot Applicable

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

United States, Australia, Brazil300 participantsStarted 2021-02-10

Plain-language summary

This is a natural history study prospectively investigating neuroimaging markers of disease progression in children and adults with Friedreich ataxia (FA). There will be three assessment periods (baseline, 12 and 24 months). The study will include approximately 200 individuals with FA and 100 matched controls recruited across the six international academic sites. Other assessments will include secondary clinical and cognitive markers, as well as exploratory blood markers.

Who can participate

Age range

5 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Age ≥ 5 years * Written informed consent provided * Individuals with FA must have a genetic confirmation of diagnosis and be biallelic for a GAA repeat length \> 55 in intron 1 of FXN and/or have a GAA repeat length \> 55 in intron 1 of FXN in one allele and another type of mutation that is inferred to cause loss of function in the second FXN allele * Individuals with FA must have an age of disease onset ≤ 25 years * Individuals with FA must have a disease duration ≤ 25 years * Individuals with FA must have a Friedreich Ataxia Rating Scale (FARS) Functional staging score of ≤ 5 and total modified FARS (mFARS) score of ≤ 65 on enrolment Exclusion Criteria: * Age \< 5 years * Unable to provide written informed consent * Magnetic resonance contraindications (e.g. pacemaker or other metallic surgical implants) * Presence of metallic dental braces * Pregnancy (ascertained via a question or test as mandated at particular sites) * Individuals with FA must not have acute or ongoing medical or other conditions that, after discussion between the Site Investigator and steering committee, is deemed to interfere with the conduct and assessments of the study * Individuals with FA must not have another neurological condition apart from FA * Individuals with FA must not have other neurologic conditions that, in the opinion of the Site Investigator, would interfere with the conduct and assessments of the study * Controls must not have a diagnosed psychiatric or neurol…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Baseline dentate nuclei magnetic susceptibility

Timeframe: Baseline

Slope of change in dentate nuclei magnetic susceptibility

Timeframe: Baseline to 24 months

Baseline dentate volume

Timeframe: Baseline

Slope of change in dentate volume

Timeframe: Baseline to 24 months

Baseline total cerebellar volume

Timeframe: Baseline

Slope of change in total cerebellar volume

Timeframe: Baseline to 24 months

Baseline superior cerebellar peduncle volume

Timeframe: Baseline

Slope of change in superior cerebellar peduncle volume

Trial details

NCT IDNCT04349514

SponsorMonash University

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2025-10

View on ClinicalTrials.gov More Friedreich Ataxia trials

Plain-language summary

Who can participate

Age range

5 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Baseline dentate nuclei magnetic susceptibility

Timeframe: Baseline

Slope of change in dentate nuclei magnetic susceptibility

Timeframe: Baseline to 24 months

Baseline dentate volume

Timeframe: Baseline

Slope of change in dentate volume

Timeframe: Baseline to 24 months

Baseline total cerebellar volume

Timeframe: Baseline

Slope of change in total cerebellar volume

Timeframe: Baseline to 24 months

Baseline superior cerebellar peduncle volume

Timeframe: Baseline

Slope of change in superior cerebellar peduncle volume