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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04349514




Registration number
NCT04349514
Ethics application status
Date submitted
1/04/2020
Date registered
16/04/2020

Titles & IDs
Public title
A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA)
Scientific title
A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA)
Secondary ID [1] 0 0
TRACK-FA
Universal Trial Number (UTN)
Trial acronym
(TRACK-FA)
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Friedreich Ataxia 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Neurodegenerative diseases

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Other interventions - Natural history

Friedreich ataxia - Individuals with a diagnosis of Friedreich ataxia.

Control - Individuals without a diagnosis of Friedreich ataxia.


Other interventions: Natural history
Longitudinal observation of neuroimaging, clinical, and blood markers.
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Baseline dentate nuclei magnetic susceptibility
Timepoint [1] 0 0
Baseline
Primary outcome [2] 0 0
Slope of change in dentate nuclei magnetic susceptibility
Timepoint [2] 0 0
Baseline to 24 months
Primary outcome [3] 0 0
Baseline dentate volume
Timepoint [3] 0 0
Baseline
Primary outcome [4] 0 0
Slope of change in dentate volume
Timepoint [4] 0 0
Baseline to 24 months
Primary outcome [5] 0 0
Baseline total cerebellar volume
Timepoint [5] 0 0
Baseline
Primary outcome [6] 0 0
Slope of change in total cerebellar volume
Timepoint [6] 0 0
Baseline to 24 months
Primary outcome [7] 0 0
Baseline superior cerebellar peduncle volume
Timepoint [7] 0 0
Baseline
Primary outcome [8] 0 0
Slope of change in superior cerebellar peduncle volume
Timepoint [8] 0 0
Baseline to 24 months
Primary outcome [9] 0 0
Baseline superior cerebellar peduncle fractional anisotropy
Timepoint [9] 0 0
Baseline
Primary outcome [10] 0 0
Slope of change in superior cerebellar peduncle fractional anisotropy
Timepoint [10] 0 0
Baseline to 24 months
Primary outcome [11] 0 0
Baseline superior cerebellar peduncle mean diffusivity
Timepoint [11] 0 0
Baseline
Primary outcome [12] 0 0
Slope of change in superior cerebellar peduncle mean diffusivity
Timepoint [12] 0 0
Baseline to 24 months
Primary outcome [13] 0 0
Baseline superior cerebellar peduncle radial diffusivity
Timepoint [13] 0 0
Baseline
Primary outcome [14] 0 0
Slope of change in superior cerebellar peduncle radial diffusivity
Timepoint [14] 0 0
Baseline to 24 months
Primary outcome [15] 0 0
Baseline superior cerebellar peduncle axial diffusivity
Timepoint [15] 0 0
Baseline
Primary outcome [16] 0 0
Slope of change in superior cerebellar peduncle axial diffusivity
Timepoint [16] 0 0
Baseline to 24 months
Primary outcome [17] 0 0
Baseline cervical spinal cord cross-sectional area
Timepoint [17] 0 0
Baseline
Primary outcome [18] 0 0
Slope of change in cervical spinal cord cross-sectional area
Timepoint [18] 0 0
Baseline to 24 months
Primary outcome [19] 0 0
Baseline cervical spinal cord fractional anisotropy
Timepoint [19] 0 0
Baseline
Primary outcome [20] 0 0
Slope of change in cervical spinal cord fractional anisotropy
Timepoint [20] 0 0
Baseline to 24 months
Primary outcome [21] 0 0
Baseline cervical spinal cord mean diffusivity
Timepoint [21] 0 0
Baseline
Primary outcome [22] 0 0
Slope of change in cervical spinal cord mean diffusivity
Timepoint [22] 0 0
Baseline to 24 months
Primary outcome [23] 0 0
Baseline cervical spinal cord radial diffusivity
Timepoint [23] 0 0
Baseline
Primary outcome [24] 0 0
Slope of change in cervical spinal cord radial diffusivity
Timepoint [24] 0 0
Baseline to 24 months
Primary outcome [25] 0 0
Baseline cervical spinal cord axial diffusivity
Timepoint [25] 0 0
Baseline
Primary outcome [26] 0 0
Slope of change in cervical spinal cord axial diffusivity
Timepoint [26] 0 0
Baseline to 24 months
Primary outcome [27] 0 0
Baseline cervical spine tNAA/mIns ratio
Timepoint [27] 0 0
Baseline
Primary outcome [28] 0 0
Slope of the cervical spine tNAA/mIns ratio
Timepoint [28] 0 0
Baseline to 24 months
Secondary outcome [1] 0 0
Modified Friedreich Ataxia Rating Scale (mFARS) score
Timepoint [1] 0 0
Baseline to 24 months
Secondary outcome [2] 0 0
Upright Stability (US) score
Timepoint [2] 0 0
Baseline to 24 months
Secondary outcome [3] 0 0
Activities of Daily Living (ADL) score
Timepoint [3] 0 0
Baseline to 24 months
Secondary outcome [4] 0 0
Scale for the Assessment and Rating of Ataxia (SARA) score
Timepoint [4] 0 0
Baseline to 24 months
Secondary outcome [5] 0 0
9 Hole Peg Test times
Timepoint [5] 0 0
Baseline to 24 months
Secondary outcome [6] 0 0
Speech analysis scores
Timepoint [6] 0 0
Baseline to 24 months
Secondary outcome [7] 0 0
Low-Contrast Sloan Letter Chart (LCSLC) test score
Timepoint [7] 0 0
Baseline to 24 months
Secondary outcome [8] 0 0
Cerebellar Cognitive Affective/Schmahmann Syndrome (CCAS) scale score
Timepoint [8] 0 0
Baseline to 24 months
Secondary outcome [9] 0 0
Hayling Sentence Completion Test (HSCT) scores
Timepoint [9] 0 0
Baseline to 24 months
Secondary outcome [10] 0 0
Hospital Anxiety and Depression Scale (HADS) scores
Timepoint [10] 0 0
Baseline to 24 months
Secondary outcome [11] 0 0
Junior Hayling Sentence Completion Test (Junior HSCT) scores
Timepoint [11] 0 0
Baseline to 24 months
Secondary outcome [12] 0 0
Paediatric Cerebellar Cognitive Affective/Schmahmann Syndrome (CCAS) scale score
Timepoint [12] 0 0
Baseline to 24 months
Secondary outcome [13] 0 0
Revised Children's Anxiety and Depression scale (RCADS) scores
Timepoint [13] 0 0
Baseline to 24 months

Eligibility
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
Minimum age
5 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
Yes
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.

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
10/02/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/10/2025
Actual
Sample size
Target
300
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Monash Biomedical Imaging, Monash University - Clayton
Recruitment postcode(s) [1] 0 0
3168 - Clayton
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida
Country [2] 0 0
United States of America
State/province [2] 0 0
Minnesota
Country [3] 0 0
United States of America
State/province [3] 0 0
Pennsylvania
Country [4] 0 0
Brazil
State/province [4] 0 0
São Paulo
Country [5] 0 0
Canada
State/province [5] 0 0
Quebec
Country [6] 0 0
Germany
State/province [6] 0 0
Aachen

Funding & Sponsors
Primary sponsor type
Other
Name
Monash University
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
University of Minnesota
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
RWTH Aachen University
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
University of Campinas, Brazil
Address [3] 0 0
Country [3] 0 0
Other collaborator category [4] 0 0
Other
Name [4] 0 0
Children's Hospital of Philadelphia
Address [4] 0 0
Country [4] 0 0
Other collaborator category [5] 0 0
Other
Name [5] 0 0
University of Florida
Address [5] 0 0
Country [5] 0 0
Other collaborator category [6] 0 0
Other
Name [6] 0 0
Friedreich's Ataxia Research Alliance
Address [6] 0 0
Country [6] 0 0
Other collaborator category [7] 0 0
Other
Name [7] 0 0
McGill University
Address [7] 0 0
Country [7] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Nellie Georgiou-Karistianis, PhD
Address 0 0
Monash University
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
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.
When will data be available (start and end dates)?
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.
Available to whom?
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.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.monash.edu/medicine/trackfa/news


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04349514