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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04349514
Recruitment Status : Not yet recruiting
First Posted : April 16, 2020
Last Update Posted : July 20, 2020
Sponsor:
Collaborators:
University of Minnesota
RWTH Aachen University
University of Campinas, Brazil
Children's Hospital of Philadelphia
University of Florida
Friedreich's Ataxia Research Alliance
Information provided by (Responsible Party):
Nellie Georgiou-Karistianis, Monash University

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 (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.

Condition or disease Intervention/treatment
Friedreich Ataxia Other: Natural history

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 balance and coordination, frequent falls, loss of ambulation, dysarthria, dysphagia and loss of vision and hearing. Other symptoms include cardiomyopathy, diabetes, scoliosis and fatigue. Age of onset can vary but most often presents during childhood, ages 5-15 years.

There is currently no cure and no disease-modifying treatment. Drug candidates to potentially treat FA are under development; however, there is a lack of well- characterized neuroimaging biomarkers for testing their efficacy in clinical trials, hampering this process. Establishing disease-specific neuroimaging biomarkers to track disease progression requires high-quality longitudinal data from large cohorts of patients, compared to controls. In rare diseases, such as FA, this can only be achieved through multi-site collaboration.

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 six academic institutions, together with industry partners and the Friedreich's Ataxia Research Alliance USA (FARA).

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Study Type : Observational
Estimated Enrollment : 300 participants
Observational Model: Case-Control
Time Perspective: Prospective
Official Title: A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals With Friedreich Ataxia (TRACK-FA)
Estimated Study Start Date : October 1, 2020
Estimated Primary Completion Date : October 2023
Estimated Study Completion Date : October 2023


Group/Cohort Intervention/treatment
Friedreich ataxia
Individuals with a diagnosis of Friedreich ataxia.
Other: Natural history
Longitudinal observation of neuroimaging, clinical, and blood markers.

Control
Individuals without a diagnosis of Friedreich ataxia.
Other: Natural history
Longitudinal observation of neuroimaging, clinical, and blood markers.




Primary Outcome Measures :
  1. Baseline dentate nuclei magnetic susceptibility [ Time Frame: Baseline ]
    Magnetic susceptibility of the dentate nuclei will be measured using T2*-weighted multiecho magnetic resonance imaging and quantitative susceptibility mapping processing. Baseline dentate nuclei susceptibility will be compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  2. Slope of change in dentate nuclei magnetic susceptibility [ Time Frame: Baseline to 24 months ]
    Magnetic susceptibility of the dentate nuclei will be measured using T2*-weighted multiecho magnetic resonance imaging and quantitative susceptibility mapping processing. The within-person slope of dentate nuclei susceptibility over the three study visits will be estimated and compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  3. Baseline dentate volume [ Time Frame: Baseline ]
    Volume of the dentate nuclei will be measured using T2*-weighted multiecho magnetic resonance imaging and quantitative susceptibility mapping processing. Baseline dentate nuclei volume will be compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  4. Slope of change in dentate volume [ Time Frame: Baseline to 24 months ]
    Volume of the dentate nuclei will be measured using T2*-weighted multiecho magnetic resonance imaging and quantitative susceptibility mapping processing. The within-person slope of dentate nuclei volume over the three study visits will be estimated and compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  5. Baseline total cerebellar volume [ Time Frame: Baseline ]
    Total volume of the cerebellum will be measured using T1- and T2-weighted magnetic resonance imaging. Baseline total cerebellar volume will be compared between the Friedreich ataxia and control groups.

  6. Slope of change in total cerebellar volume [ Time Frame: Baseline to 24 months ]
    Total volume of the cerebellum will be measured using T1- and T2-weighted magnetic resonance imaging. The within-person slope of total cerebellar volume over the three study visits will be estimated and compared between the Friedreich ataxia and control groups.

  7. Baseline superior cerebellar peduncle volume [ Time Frame: Baseline ]
    Volume of the superior cerebellar peduncles will be measured using T1- and T2-weighted magnetic resonance imaging. Baseline superior cerebellar peduncle volume will be compared between the Friedreich ataxia and control groups.

  8. Slope of change in superior cerebellar peduncle volume [ Time Frame: Baseline to 24 months ]
    Volume of the superior cerebellar peduncles will be measured using T1- and T2-weighted magnetic resonance imaging. The within-person slope of superior cerebellar peduncle volume over the three study visits will be estimated and compared between the Friedreich ataxia and control groups.

  9. Baseline superior cerebellar peduncle fractional anisotropy [ Time Frame: Baseline ]
    Fractional anisotropy of the superior cerebellar peduncles will be measured using diffusion tensor magnetic resonance imaging. Baseline superior cerebellar peduncle fractional anisotropy will be compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  10. Slope of change in superior cerebellar peduncle fractional anisotropy [ Time Frame: Baseline to 24 months ]
    Fractional anisotropy of the superior cerebellar peduncles will be measured using diffusion tensor magnetic resonance imaging. The within-person slope of superior cerebellar peduncle fractional anisotropy over the three study visits will be estimated and compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  11. Baseline superior cerebellar peduncle mean diffusivity [ Time Frame: Baseline ]
    Mean diffusivity of the superior cerebellar peduncles will be measured using diffusion tensor magnetic resonance imaging. Baseline superior cerebellar peduncle mean diffusivity will be compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  12. Slope of change in superior cerebellar peduncle mean diffusivity [ Time Frame: Baseline to 24 months ]
    Mean diffusivity of the superior cerebellar peduncles will be measured using diffusion tensor magnetic resonance imaging. The within-person slope of superior cerebellar peduncle mean diffusivity over the three study visits will be estimated and compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  13. Baseline superior cerebellar peduncle radial diffusivity [ Time Frame: Baseline ]
    Radial diffusivity of the superior cerebellar peduncles will be measured using diffusion tensor magnetic resonance imaging. Baseline superior cerebellar peduncle radial diffusivity will be compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  14. Slope of change in superior cerebellar peduncle radial diffusivity [ Time Frame: Baseline to 24 months ]
    Radial diffusivity of the superior cerebellar peduncles will be measured using diffusion tensor magnetic resonance imaging. The within-person slope of superior cerebellar peduncle radial diffusivity over the three study visits will be estimated and compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  15. Baseline superior cerebellar peduncle axial diffusivity [ Time Frame: Baseline ]
    Axial diffusivity of the superior cerebellar peduncles will be measured using diffusion tensor magnetic resonance imaging. Baseline superior cerebellar peduncle axial diffusivity will be compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  16. Slope of change in superior cerebellar peduncle axial diffusivity [ Time Frame: Baseline to 24 months ]
    Axial diffusivity of the superior cerebellar peduncles will be measured using diffusion tensor magnetic resonance imaging. The within-person slope of superior cerebellar peduncle axial diffusivity over the three study visits will be estimated and compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  17. Baseline cervical spinal cord cross-sectional area [ Time Frame: Baseline ]
    Cross-sectional area of the cervical portion of the spinal cord will be measured using T2-weighted magnetic resonance imaging. Baseline cervical spinal cord cross-sectional area will be compared between the Friedreich ataxia and control groups.

  18. Slope of change in cervical spinal cord cross-sectional area [ Time Frame: Baseline to 24 months ]
    Cross-sectional area of the cervical portion of the spinal cord will be measured using T2-weighted magnetic resonance imaging. The within-person slope of cervical spinal cord cross-sectional area over the three study visits will be estimated and compared between the Friedreich ataxia and control groups.

  19. Baseline cervical spinal cord fractional anisotropy [ Time Frame: Baseline ]
    Fractional anisotropy of the cervical portion of the spinal cord will be measured using diffusion tensor magnetic resonance imaging. Baseline cervical spinal cord fractional anisotropy will be compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  20. Slope of change in cervical spinal cord fractional anisotropy [ Time Frame: Baseline to 24 months ]
    Fractional anisotropy of the cervical portion of the spinal cord will be measured using diffusion tensor magnetic resonance imaging. The within-person slope of cervical spinal cord fractional anisotropy over the three study visits will be estimated and compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  21. Baseline cervical spinal cord mean diffusivity [ Time Frame: Baseline ]
    Mean diffusivity of the cervical portion of the spinal cord will be measured using diffusion tensor magnetic resonance imaging. Baseline cervical spinal cord mean diffusivity will be compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  22. Slope of change in cervical spinal cord mean diffusivity [ Time Frame: Baseline to 24 months ]
    Mean diffusivity of the cervical portion of the spinal cord will be measured using diffusion tensor magnetic resonance imaging. The within-person slope of cervical spinal cord mean diffusivity over the three study visits will be estimated and compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  23. Baseline cervical spinal cord radial diffusivity [ Time Frame: Baseline ]
    Radial diffusivity of the cervical portion of the spinal cord will be measured using diffusion tensor magnetic resonance imaging. Baseline cervical spinal cord radial diffusivity will be compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  24. Slope of change in cervical spinal cord radial diffusivity [ Time Frame: Baseline to 24 months ]
    Radial diffusivity of the cervical portion of the spinal cord will be measured using diffusion tensor magnetic resonance imaging. The within-person slope of cervical spinal cord radial diffusivity over the three study visits will be estimated and compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  25. Baseline cervical spinal cord axial diffusivity [ Time Frame: Baseline ]
    Axial diffusivity of the cervical portion of the spinal cord will be measured using diffusion tensor magnetic resonance imaging. Baseline cervical spinal cord axial diffusivity will be compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  26. Slope of change in cervical spinal cord axial diffusivity [ Time Frame: Baseline to 24 months ]
    Axial diffusivity of the cervical portion of the spinal cord will be measured using diffusion tensor magnetic resonance imaging. The within-person slope of cervical spinal cord axial diffusivity over the three study visits will be estimated and compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  27. Baseline cervical spine tNAA/mIns ratio [ Time Frame: Baseline ]
    The ratio of N-acetylaspartate (tNAA) and myo-inositol (mIns) within cervical spinal cord will be measured using sLASER magnetic resonance spectroscopy. The baseline tNAA/mIns ratio will be compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.

  28. Slope of the cervical spine tNAA/mIns ratio [ Time Frame: Baseline to 24 months ]
    The ratio of N-acetylaspartate (tNAA) and myo-inositol (mIns) within cervical spinal cord will be measured using sLASER magnetic resonance spectroscopy. The within-person slope of the tNAA/mIns ratio over the three study visits will be estimated and compared between the Friedreich ataxia and control groups. This measurement will be obtained for participants aged 11 years and over.


Secondary Outcome Measures :
  1. Modified Friedreich Ataxia Rating Scale (mFARS) score [ Time Frame: Baseline to 24 months ]
    The modified Friedreich Ataxia Rating Scale (mFARS) is a neurological rating scale comprising four subscales (bulbar, upper limb coordination, lower limb coordination, and upright stability). The total score ranges from 0 to 93, with a higher score reflecting greater neurological severity. This assessment will be administered to participants with FA only.

  2. Upright Stability (US) score [ Time Frame: Baseline to 24 months ]
    The Upright Stability (US) assessment is part of the neurological examination within the Modified Friedreich Ataxia Rating Scale (mFARS). This component comprises nine items: sitting position, stance with feet apart, stance with feet apart and eyes closed, stance with feet together, stance with feet together and eyes closed, tandem stance, stance with dominant foot, tandem walk, and gait. The score ranges from 0 to 9, with a higher score reflecting poorer upright stability (i.e., greater neurological severity). This assessment will be administered to participants with FA only.

  3. Activities of Daily Living (ADL) score [ Time Frame: Baseline to 24 months ]
    Activities of Daily Living (ADL) is a component of the Friedreich Ataxia Rating Scale (FARS), a clinical rating scale developed for FA. The ADL score aims to quantify essential and routine aspects of self-care, often reported on by a family member or caregiver of a person with FA. The ADL comprises 9 items: speech, swallowing, cutting food and handling utensils, dressing, personal hygiene, falling, walking, quality of sitting position, and bladder function. The score ranges from 0 to 36, with a higher score reflecting greater difficulty completing activities of daily living independently. This assessment will be administered to participants with FA only.

  4. Scale for the Assessment and Rating of Ataxia (SARA) score [ Time Frame: Baseline to 24 months ]
    The SARA is a semi-quantitative assessment of ataxia, measuring ataxia of upper limb, lower limb, gait, balance and speech. It has eight items: gait, stance, sitting, speech disturbance, finger chase, nose-finger test, fast alternating hand movement, and heel-shin slide. The total score ranges from 0 (no ataxia) to 40 (severe ataxia). This assessment will be administered to participants with FA only.

  5. 9 Hole Peg Test times [ Time Frame: Baseline to 24 months ]
    The 9 Hole Peg Test (9HPT) examines finger dexterity and involves placing and removing nine pegs in a pegboard in the quickest possible time. Two consecutive trials of the dominant hand, followed immediately by two consecutive trials of the non-dominant hand, are undertaken. The average time taken to complete the task, for each of the dominant and non-dominant hand, is calculated. The 9HPT has high intra- and inter-rater reliability and is the most commonly used measure of upper limb function in FA. This assessment will be administered to participants with FA only.

  6. Speech analysis scores [ Time Frame: Baseline to 24 months ]
    A battery of speech evaluations will be administered and recorded on a laptop for analysis, using Redenlab software. This will include: reading of a phonetically-balanced passage, sustained vowel sound, listing days of the week, repeating syllables, and a monologue task. This will form a measure of dysarthria. Redenlab is a US-Australian speech-testing company, https://redenlab.com. This assessment will be administered to participants with FA and controls.

  7. Low-Contrast Sloan Letter Chart (LCSLC) test score [ Time Frame: Baseline to 24 months ]
    Contrast letter acuity for vision will be assessed using back-lit Low-Contrast Sloan Letter Charts (LCSLCs). Participants will sit at an eye distance of 2 metres from the chart. Binocular vision will be assessed using participants' normal corrective lenses where relevant. Participants are required to read each letter on the chart. Three charts will be presented, with three different visual contrast levels: 100% (equivalent to high-contrast visual acuity), 2.5%, and 1.25%. The maximum total score across the three charts (number of letters read correctly) is 240. Scores for each individual chart will also be recorded. This assessment will be administered to participants with FA only.

  8. Cerebellar Cognitive Affective/Schmahmann Syndrome (CCAS) scale score [ Time Frame: Baseline to 24 months ]
    The Cerebellar Cognitive Affective/Schmahmann Syndrome (CCAS) Scale is a 10-item screening measure assessing attention and concentration, executive functioning, memory, language, visuospatial functioning, abstract thinking, and neuropsychiatric features. Each item has an associated raw score and pass/fail evaluation. The total raw score (maximum 120) and the total number of "failed" items (maximum 10) will be recorded. This scale is to be administered to participants who are aged 18 years and over. This assessment will be administered to participants with FA and controls.

  9. Hayling Sentence Completion Test (HSCT) scores [ Time Frame: Baseline to 24 months ]
    The Hayling Sentence Completion Test (HSCT) is an orally-administered test measuring response initiation and response suppression. In the first section, participants are asked to complete a series of incomplete sentences with a sensible word. In the second section, participants are asked to supply an unrelated word to complete each sentence. Scaled scores for total response latency in each section, a scaled score for errors in the second section, and an overall scale score are calculated. This test is to be administered to participants who are aged 18 years and over. This assessment will be administered to participants with FA and controls.

  10. Hospital Anxiety and Depression Scale (HADS) scores [ Time Frame: Baseline to 24 months ]
    The Hospital Anxiety and Depression Scale (HADS) is a 14-item self-assessment scale designed to screen for states of depression and anxiety and measure the severity of these states. It contains a 7-item subscale for each of Anxiety (HADS-A) and Depression (HADS-D). Possible scores for each of the HADS-Anxiety and HADS-Depression scales range from 0 to 21. Higher scores indicate more severe anxiety and depression. This scale is be administered to participants who are aged 18 year and over. This assessment will be administered to participants with FA and controls.

  11. Junior Hayling Sentence Completion Test (Junior HSCT) scores [ Time Frame: Baseline to 24 months ]
    The Junior Hayling Sentence Completion Test (Junior HSCT) is an orally-administered measure of response initiation and response suppression in children. To be administered to participants who are aged at least 8 years but less than 18 years. This assessment will be administered to participants with FA and controls.

  12. Paediatric Cerebellar Cognitive Affective/Schmahmann Syndrome (CCAS) scale score [ Time Frame: Baseline to 24 months ]
    The Paediatric Cerebellar Cognitive Affective/Schmahmann Syndrome (CCAS) scale is an assessment of the executive, visual-spatial and linguistic components of cognitive control and affect in children. Currently under development. To be incorporated as a secondary outcome measure if available at the time of study commencement. To be administered to participants who are aged at least 8 years but less than 18 years. This assessment will be administered to participants with FA and controls.

  13. Revised Children's Anxiety and Depression scale (RCADS) scores [ Time Frame: Baseline to 24 months ]
    The Revised Child Anxiety and Depression Scale (RCADS) is a 47-item, self-report questionnaire six subscales: separation anxiety disorder, social phobia, generalized anxiety disorder, panic disorder, obsessive compulsive disorder, and major depressive disorder. It also yields a Total Anxiety Scale and a Total Internalizing Scale. A higher score indicates a higher level of the given disorder/syndrome. This scale is to be administered to participants who are aged at least 8 years but less than 18 years. This assessment will be administered to participants with FA and controls.


Other Outcome Measures:
  1. Serum neurofilament light chain (NfL) level [ Time Frame: Baseline to 24 months ]
    Neurofilament light chain (NfL) is a peripherally-accessible blood biomarker of neuroaxonal destruction in various neurodegenerative diseases. It has been shown for several such diseases that blood NfL might serve as a progression biomarker as well as a treatment response biomarker. Blood samples will be collected at each of the three study visits, stored locally, and shipped to a central location for analysis of NfL levels.

  2. Frataxin protein level [ Time Frame: Baseline to 24 months ]
    Levels of frataxin protein in lymphocyte and serum will be measured. Frataxin is produced at reduced levels in FA as a consequence of the guanine-adenine-adenine (GAA) repeat expansion in the frataxin gene (FXN) on chromosome 9. Blood samples will be collected at each of the three study visits, stored locally, and shipped to a central location for analysis of frataxin levels.


Biospecimen Retention:   Samples With DNA
A blood sample (approximately 18-20ml) will be collected from each participant at the first assessment by a trained phlebotomist or clinician and processed immediately by research staff.


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
The study population will consist of individuals aged 5 and above who either have a diagnosis of Friedreich ataxia (FA) or who do not (Controls). Controls will be healthy volunteers who are age- and gender-matched to the FA cohort.
Criteria

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 neurological condition
  • Controls must not have acute or ongoing medical or other conditions that would interfere with the conduct and assessments of the study

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04349514


Contacts
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Contact: Nellie Georgiou-Karistianis, PhD + 61 3 9905 1575 nellie.georgiou-karistianis@monash.edu

Locations
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United States, Florida
University of Florida
Gainesville, Florida, United States, 32611
Contact: Samantha Norman    352-273-8218    samantha.norman@peds.ufl.edu   
Principal Investigator: Sub H Subramony, MD         
Principal Investigator: Thomas Mareci, PhD         
Principal Investigator: Manuela Corti, PhD         
United States, Minnesota
Center for Magnetic Resonance Research, University of Minnesota
Minneapolis, Minnesota, United States, 55455
Contact: Pierre-Gilles Henry, PhD       henry044@umn.edu   
Principal Investigator: Pierre-Gilles Henry, PhD         
Principal Investigator: Christophe Lenglet, PhD         
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Contact: William Gaetz, PhD       gaetzw@email.chop.edu   
Principal Investigator: William C Gaetz, PhD         
Principal Investigator: Timothy PL Roberts, PhD         
Principal Investigator: David Lynch, MD, PhD         
Australia, Victoria
Monash Biomedical Imaging, Monash University
Clayton, Victoria, Australia, 3168
Contact: Nellie Georgiou-Karistianis, PhD    + 61 3 9905 1575    nellie.georgiou-karistianis@monash.edu   
Principal Investigator: Nellie Georgiou-Karistianis, PhD         
Principal Investigator: Ian H Harding, PhD         
Principal Investigator: Louise A Corben, PhD         
Principal Investigator: Martin B Delatycki, PhD         
Brazil
Lab of Neuroimaging and Dept of Neurology, University of Campinas (UNICAMP)
São Paulo, Brazil
Contact: Marcondes França, MD, PhD    +55 19 3521 9217    mcfjr@unicamp.br   
Principal Investigator: Marcondes C França, MD, PhD         
Principal Investigator: Thiago JR Rezende, PhD         
Germany
Department of Neurology, RWTH Aachen University
Aachen, Germany
Contact: Kathrin Reetz, MD    +49 241 80 89600    kreetz@ukaachen.de   
Principal Investigator: Kathrin Reetz, MD         
Principal Investigator: Imis Dogan, PhD         
Principal Investigator: Sandro Romanzetti, PhD         
Principal Investigator: Jörg B Schulz, MD         
Sponsors and Collaborators
Monash University
University of Minnesota
RWTH Aachen University
University of Campinas, Brazil
Children's Hospital of Philadelphia
University of Florida
Friedreich's Ataxia Research Alliance
Investigators
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Principal Investigator: Nellie Georgiou-Karistianis, PhD Monash University
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Responsible Party: Nellie Georgiou-Karistianis, Professor Nellie Georgiou-Karistianis, Monash University
ClinicalTrials.gov Identifier: NCT04349514    
Other Study ID Numbers: TRACK-FA
First Posted: April 16, 2020    Key Record Dates
Last Update Posted: July 20, 2020
Last Verified: July 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: It is recognised that this project will generate data that is of interest to the FA academic and bio-pharmaceutical, drug development community. All such data (de-identified) will be made available to third parties at the completion of the study after request, with approval from the TRACK-FA Steering Committee. Each site will be required to ensure that participants are consented in such a way that allows the sharing of de-identified data with the community in this manner.
Time Frame: Data will become available after the conclusion of the TRACK-FA study. The study will be 5 years in duration and each academic site may have a slightly different start and end date.
Access Criteria: Data access will be granted on a case-by-case basis after the study has been completed. The requesting party will be required to submit a formal request to the TRACK-FA Steering Committee outlining how the data is to be used and for what purpose.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Nellie Georgiou-Karistianis, Monash University:
Neuroimaging
Longitudinal
Biomarkers
Natural history
Additional relevant MeSH terms:
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Ataxia
Cerebellar Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases