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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT01339884




Registration number
NCT01339884
Ethics application status
Date submitted
14/04/2011
Date registered
21/04/2011
Date last updated
22/01/2014

Titles & IDs
Public title
A Study of Resveratrol as Treatment for Friedreich Ataxia
Scientific title
An Open Label Clinical Pilot Study of Resveratrol as Treatment for Friedreich Ataxia
Secondary ID [1] 0 0
10358B
Universal Trial Number (UTN)
Trial acronym
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
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Resveratrol

Active Comparator: Resveratrol, 1g daily - 15 participants will receive resveratrol 1g daily

Active Comparator: Resveratrol, 5g daily - 15 participants will receive resveratrol, 5g daily


Treatment: Drugs: Resveratrol
Resveratrol 1g daily (500mg twice daily) for 12 weeks Resveratrol 5 daily (2.5g twice daily) for 12 weeks
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Lymphocyte frataxin level
Timepoint [1] 0 0
12 weeks
Secondary outcome [1] 0 0
Oxidative stress markers
Timepoint [1] 0 0
12 weeks
Secondary outcome [2] 0 0
Clinical rating scales of ataxia
Timepoint [2] 0 0
12 weeks
Secondary outcome [3] 0 0
Echocardiogram measures
Timepoint [3] 0 0
12 weeks
Secondary outcome [4] 0 0
Pharmacokinetic studies of resveratrol
Timepoint [4] 0 0
First 2 hours post dose

Eligibility
Key inclusion criteria
- Adults with Friedreich ataxia due to homozygosity for the GAA repeat expansion in
intron 1 of the FXN gene

- Functional stage on the Ataxia subscale of the FARS of 1 or higher
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Women who are pregnant or lactating

- Active arrythmias or significant cardiac insufficiency

- Use of idebenone, Coenzyme Q or vitamin E within 30 days prior to enrolment

- Use of amiodarone or other medications which may have clinically significant drug
interactions that cannot be safely monitored

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 1/Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/04/2011
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/12/2012
Sample size
Target
Accrual to date
Final
27
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Monash Medical Centre, Southern Health - Clayton, Melbourne
Recruitment postcode(s) [1] 0 0
3168 - Clayton, Melbourne

Funding & Sponsors
Primary sponsor type
Other
Name
Murdoch Childrens Research Institute
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Friedreich's Ataxia Research Alliance
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to determine the effect of two doses of resveratrol taken for a
12 week period, on frataxin levels in individuals with Friedreich ataxia. This study will
also measure the effect of resveratrol on markers of oxidative stress, clinical measures of
ataxia, and cardiac parameters.
Trial website
https://clinicaltrials.gov/ct2/show/NCT01339884
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Martin Delatycki, MBBS PhD
Address 0 0
Murdoch Childrens Research Institute
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT01339884