TRACK-FA is a natural history study to investigate brain and spinal cord changes in individuals with Friedreich ataxia. If imaging differences in the central nervous system are correlated with progression of FA, these differences could be used in future clinical trials to monitor treatment outcome. There will be three assessment periods (baseline, 12 and 24 months).
Anyone considering participating in a clinical trial should discuss the matter with their physician. FARA does not endorse or recommend any particular studies.
Study Details:
The aim of TRACK-FA is to develop an FA neuroimaging dataset from brain and spinal cord that is suitable for assessing the potential value of neuroimaging biomarkers and providing a basis for instituting them in clinical trials. The dataset will comprise a range of neuroimaging measures to assess changes in spinal cord and brain regions that have previously shown to be compromised in individuals with FA. In addition to neuroimaging measures, TRACK-FA will also include clinical, cognitive data and biospecimen data. The TRACK-FA dataset will provide a unique opportunity for academic researchers in collaboration with industry partners to access the images, subsidiary data, and associated clinical data for community research.
This multi-centre study is a collaborative effort across seven academic institutions, together with industry partners and the Friedreich’s Ataxia Research Alliance (FARA).
Key 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
Key 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 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.
Additional inclusion and exclusion criteria may apply and will be evaluated by a study doctor.
Length of Study Commitment:
- Three years