Status:
ACTIVE_NOT_RECRUITING
A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA)
Lead Sponsor:
Monash University
Collaborating Sponsors:
University of Minnesota
RWTH Aachen University
Conditions:
Friedreich Ataxia
Eligibility:
All Genders
5+ years
Brief 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 (base...
Detailed Description
Friedreich ataxia (FA) is a multi-system progressive disorder with the most prevalent and prominent symptoms relating to dysfunction in the central and peripheral nervous system, including, loss of ba...
Eligibility Criteria
Inclusion
- 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
- 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 neurological condition
- Controls must not have acute or ongoing medical or other conditions that would interfere with the conduct and assessments of the study
- Controls must not be siblings of individuals with FA whose carrier status (i.e., confirmed carrier, confirmed non-carrier, or obligate carrier) is unknown.
Key Trial Info
Start Date :
February 10 2021
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
October 1 2025
Estimated Enrollment :
300 Patients enrolled
Trial Details
Trial ID
NCT04349514
Start Date
February 10 2021
End Date
October 1 2025
Last Update
November 27 2024
Active Locations (7)
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1
University of Florida
Gainesville, Florida, United States, 32611
2
Center for Magnetic Resonance Research, University of Minnesota
Minneapolis, Minnesota, United States, 55455
3
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
4
Monash Biomedical Imaging, Monash University
Clayton, Victoria, Australia, 3168