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A Study of Resveratrol as Treatment for Friedreich Ataxia

This study has been completed.

Sponsor:

ClinicalTrials.gov Identifier:

NCT01339884

First Posted: April 21, 2011

Last Update Posted: January 22, 2014

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.

Collaborator:

Friedreich's Ataxia Research Alliance

Information provided by (Responsible Party):

Martin Delatycki, Murdoch Childrens Research Institute

Purpose

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	Intervention	Phase
Friedreich Ataxia	Drug: Resveratrol	Phase 1 Phase 2


Study Type:	Interventional
Study Design:	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

Resource links provided by NLM:

Genetics Home Reference related topics: Friedreich ataxia VLDLR-associated cerebellar hypoplasia autosomal recessive cerebellar ataxia type 1

MedlinePlus related topics: Friedreich's Ataxia

Drug Information available for: Resveratrol

Genetic and Rare Diseases Information Center resources: Friedreich Ataxia Spinocerebellar Ataxia

U.S. FDA Resources

Further study details as provided by Martin Delatycki, Murdoch Childrens Research Institute:

Primary Outcome Measures:

Lymphocyte frataxin level [ Time Frame: 12 weeks ]
Change in lymphocyte frataxin levels at 12 weeks compared to baseline

Secondary Outcome Measures:

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.


Enrollment:	27
Study Start Date:	April 2011
Study Completion Date:	December 2012
Primary Completion Date:	August 2012 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
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

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).

Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:	18 Years and older (Adult, Senior)
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

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


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


Principal Investigator:	Martin Delatycki, MBBS PhD	Murdoch Childrens Research Institute

More Information


Responsible Party:	Martin Delatycki, Prof Martin Delatycki, Murdoch Childrens Research Institute
ClinicalTrials.gov Identifier:	NCT01339884 History of Changes
Other Study ID Numbers:	10358B
First Submitted:	April 14, 2011
First Posted:	April 21, 2011
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:


Ataxia Cerebellar Ataxia Friedreich Ataxia Dyskinesias Neurologic Manifestations Nervous System Diseases Signs and Symptoms 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 Anti-Inflammatory Agents, Non-Steroidal Analgesics, Non-Narcotic Analgesics Sensory System Agents Peripheral Nervous System Agents Physiological Effects of Drugs Anti-Inflammatory Agents Antirheumatic Agents Antineoplastic Agents, Phytogenic Antineoplastic Agents Antioxidants Molecular Mechanisms of Pharmacological Action

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