Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12619001482156
Ethics application status
Approved
Date submitted
17/10/2019
Date registered
28/10/2019
Date last updated
28/10/2019
Date data sharing statement initially provided
28/10/2019
Type of registration
Prospectively registered

Titles & IDs
Public title
Positron Emission Tomography of Oxidative Stress in Friedreich Ataxia
Scientific title
Non-invasive, in vivo neuroimaging of oxidative stress in Friedreich ataxia
Secondary ID [1] 299574 0
Nil Known
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Friedreich ataxia 314849 0
Condition category
Condition code
Neurological 313188 313188 0 0
Neurodegenerative diseases
Human Genetics and Inherited Disorders 313266 313266 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Observational
Patient registry
False
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
- Intravenous bolus injection of 125MBq of 64Cu-ATSM, followed by PET scanning of the brain for up to 90mins.
- Single timepoint
Intervention code [1] 315830 0
Early Detection / Screening
Comparator / control treatment
This observational study will use a cross-sectional, between-group design. A comparator group of individuals who are not affected by Friedreich ataxia, and matched for demographic characteristics, will undergo the same procedures, allowing for between-group statistical comparison with the clinical cohort.
Control group
Active

Outcomes
Primary outcome [1] 321701 0
Standardised Uptake Value Ratio (SUVR) of 64Cu-ATSM binding in the dentate nuclei relative to the cerebral cortex estimated from the reconstructed PET scan after SUV equilibrium.
Timepoint [1] 321701 0
Single time-point
Primary outcome [2] 321702 0
Standardised Uptake Value Ratio (SUVR) of 64Cu-ATSM binding in the brainstem relative to the cerebral cortex estimated from the reconstructed PET scan after SUV equilibrium.
Timepoint [2] 321702 0
Single timepoint
Secondary outcome [1] 375946 0
Scale for the Assessment and Rating of Ataxias (SARA)
Timepoint [1] 375946 0
Single timepoint

Eligibility
Key inclusion criteria
- >18 years old
- Friedreich ataxia cohort: Genetically-confirmed diagnosis
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
- Neurological illnesses (other than Friedreich ataxia in the clinical cohort)
- Psychiatric illnesses requiring current pharmacotherapy
- Concussion in the past 12 months, or any history of major traumatic brain injury
- Pregnancy

Study design
Purpose
Screening
Duration
Cross-sectional
Selection
Case control
Timing
Prospective
Statistical methods / analysis
- Standardised uptake value ratios (SUVR) of the regions-of-interest (dentate nuclei and brainstem) will be calculated relative to the cerebral cortex.
- Between-group statistical inference will be undertaken using independent samples t-tests (or equivalent non-parametric Mann-Whitney U in the case of non-normality in the data).
- Linear correlations between SUVR values and clinical severity (SARA) will be undertaken using Pearson correlations (or equivalent non-parametric Spearman correlations in the event of non-normality in the data).

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
1/11/2019
Actual
Date of last participant enrolment
Anticipated
1/06/2020
Actual
Date of last data collection
Anticipated
1/06/2020
Actual
Sample size
Target
20
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC

Funding & Sponsors
Funding source category [1] 304058 0
University
Name [1] 304058 0
Monash University
Country [1] 304058 0
Australia
Primary sponsor type
University
Name
Monash University
Address
Monash Biomedical Imaging
770 Blackburn Road
Monash University
Victoria, 3800
Country
Australia
Secondary sponsor category [1] 304253 0
None
Name [1] 304253 0
None
Address [1] 304253 0
None
Country [1] 304253 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 304549 0
Monash University Human Research and Ethics Committee
Ethics committee address [1] 304549 0
Executive Officer
Monash University Human Research Ethics Committee (MUHREC)
Room 111, Chancellery Building E,
24 Sports Walk, Clayton Campus
Research Office
Monash University VIC 3800
Ethics committee country [1] 304549 0
Australia
Date submitted for ethics approval [1] 304549 0
Approval date [1] 304549 0
30/09/2019
Ethics approval number [1] 304549 0
18894

Summary
Brief summary
Friedreich ataxia (FA) is a chronic, progressive, and terminal illness. Currently, there are no effective treatments and no sensitive biometrics of illness progression for FA. Oxidative stress has been proposed as a candidate molecular process that contributes to the underlying neuropathology, and/or reflects the cellular dysfunction underlying this disease.

Measuring and tracking oxidative stress in the human brain using Positron Emission Tomography (PET) provides an opportunity for in vivo mechanistic characterisations disease in the human brain, and represents a novel and potentially sensitive approach to addressing the urgent need for pharmacodynamic and treatment monitoring biomarkers for use in clinical trials. The current study aims to investigate 64Cu-ATSM PET as a tool for imaging oxidative stress in the brains of individuals with FA.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 97346 0
Dr Ian Harding
Address 97346 0
Monash Biomedical Imaging
770 Blackburn Road
Monash University, VIC, 3800
Country 97346 0
Australia
Phone 97346 0
+61 3 9905 9283
Fax 97346 0
Email 97346 0
[email protected]
Contact person for public queries
Name 97347 0
Dr Ian Harding
Address 97347 0
Monash Biomedical Imaging
770 Blackburn Road
Monash University, VIC, 3800
Country 97347 0
Australia
Phone 97347 0
+61 3 9905 9283
Fax 97347 0
Email 97347 0
[email protected]
Contact person for scientific queries
Name 97348 0
Dr Ian Harding
Address 97348 0
Monash Biomedical Imaging
770 Blackburn Road
Monash University, VIC, 3800
Country 97348 0
Australia
Phone 97348 0
+61 3 9905 9283
Fax 97348 0
Email 97348 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.