A Study of Resveratrol as Treatment for Friedreich Ataxia
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ClinicalTrials.gov Identifier: NCT01339884 |
Recruitment Status :
Completed
First Posted : April 21, 2011
Last Update Posted : January 22, 2014
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Friedreich Ataxia | Drug: Resveratrol | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 27 participants |
Allocation: | Non-Randomized |
Intervention Model: | Parallel Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open Label Clinical Pilot Study of Resveratrol as Treatment for Friedreich Ataxia |
Study Start Date : | April 2011 |
Actual Primary Completion Date : | August 2012 |
Actual Study Completion Date : | December 2012 |

Arm | Intervention/treatment |
---|---|
Active Comparator: Resveratrol, 1g daily
15 participants will receive resveratrol 1g daily
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Drug: Resveratrol
Resveratrol 1g daily (500mg twice daily) for 12 weeks Resveratrol 5 daily (2.5g twice daily) for 12 weeks |
Active Comparator: Resveratrol, 5g daily
15 participants will receive resveratrol, 5g daily
|
Drug: Resveratrol
Resveratrol 1g daily (500mg twice daily) for 12 weeks Resveratrol 5 daily (2.5g twice daily) for 12 weeks |
- Lymphocyte frataxin level [ Time Frame: 12 weeks ]Change in lymphocyte frataxin levels at 12 weeks compared to baseline
- Oxidative stress markers [ Time Frame: 12 weeks ]Oxidative stress, as measured by a) plasma F2-isoprostanes and b) urinary 8-hydroxyl-2-deoxyguanosine levels at 12 weeks compared to baseline
- Clinical rating scales of ataxia [ Time Frame: 12 weeks ]Clinical rating scales of ataxia at 12 weeks will be compared to baseline. This will include: a) Friedreich Ataxia Rating Scale (FARS) b) International Cooperative Ataxia Rating Scale (ICARS) c) Scale for the Assessment and Rating of Ataxia (SARA) d) Friedreich Ataxia Functional Composite
- Echocardiogram measures [ Time Frame: 12 weeks ]Changes in structural and functional 3D echocardiogram measures from baseline to 12 weeks will be reported
- Pharmacokinetic studies of resveratrol [ Time Frame: First 2 hours post dose ]Pharmacokinetic data will be collected 45, 90 and 120 minutes after the first dose of resveratrol. Plasma concentration of resveratrol and its sulfate and glucuronide metabolites will be measured in ng/mL.

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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
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

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): NCT01339884
Australia, Victoria | |
Monash Medical Centre, Southern Health | |
Clayton, Melbourne, Victoria, Australia, 3168 |
Principal Investigator: | Martin Delatycki, MBBS PhD | Murdoch Childrens Research Institute |
Responsible Party: | Martin Delatycki, Prof Martin Delatycki, Murdoch Childrens Research Institute |
ClinicalTrials.gov Identifier: | NCT01339884 |
Other Study ID Numbers: |
10358B |
First Posted: | April 21, 2011 Key Record Dates |
Last Update Posted: | January 22, 2014 |
Last Verified: | January 2014 |
Friedreich ataxia Resveratrol Frataxin Cardiomyopathy Oxidative stress |
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 Resveratrol Antioxidants Molecular Mechanisms of Pharmacological Action Protective Agents Physiological Effects of Drugs Enzyme Inhibitors Platelet Aggregation Inhibitors |