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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
Sponsor:
Collaborator:
Friedreich's Ataxia Research Alliance
Information provided by (Responsible Party):
Martin Delatycki, Murdoch Childrens Research Institute

Study Description
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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.

Condition or disease Intervention/treatment Phase
Friedreich Ataxia Drug: Resveratrol Phase 1 Phase 2

Detailed Description:
Resveratrol shows promise as an agent for the treatment of Friedreich ataxia due to its antioxidant properties, neuroprotective effects, and ability to increase frataxin levels in vitro and in vivo. This clinical pilot study aims to determine the effect of two doses of resveratrol (1g/day and 5g/day) taken for 12 weeks, on frataxin levels in individuals with Friedreich ataxia. Additional outcome measures include the effect of resveratrol on markers of oxidative stress, clinical measures of ataxia , and cardiac parameters (including relative wall thickness and left ventricular mass index).
Study Design
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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

Resource links provided by the National Library of Medicine

Drug Information available for: Resveratrol

Arms and Interventions
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Arm Intervention/treatment
Active Comparator: Resveratrol, 1g daily
15 participants will receive resveratrol 1g daily
 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


Outcome Measures
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Primary Outcome Measures :
  1. Lymphocyte frataxin level [ Time Frame: 12 weeks ]
    Change in lymphocyte frataxin levels at 12 weeks compared to baseline


Secondary Outcome Measures :
  1. 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

  2. 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

  3. Echocardiogram measures [ Time Frame: 12 weeks ]
    Changes in structural and functional 3D echocardiogram measures from baseline to 12 weeks will be reported

  4. 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.


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

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
Contacts and Locations
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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): NCT01339884


Locations
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Australia, Victoria
Monash Medical Centre, Southern Health
Clayton, Melbourne, Victoria, Australia, 3168
Sponsors and Collaborators
Murdoch Childrens Research Institute
Friedreich's Ataxia Research Alliance
Investigators
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Principal Investigator: Martin Delatycki, MBBS PhD Murdoch Childrens Research Institute
More Information
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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
Keywords provided by Martin Delatycki, Murdoch Childrens Research Institute:
Friedreich ataxia
Resveratrol
Frataxin
Cardiomyopathy
Oxidative stress
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
Resveratrol
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Enzyme Inhibitors
Platelet Aggregation Inhibitors