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NCT Number: NCT01339884

A Study of Resveratrol as Treatment for Friedreich Ataxia

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.

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Key information

Conditions

Friedreich Ataxia Brain Diseases Cardiomyopathies Cardiovascular Diseases Central Nervous System Diseases Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heart Diseases Heredodegenerative Disorders, Nervous System Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Spinal Cord Diseases Spinocerebellar Degenerations

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Monash Medical Centre, Southern Health

Clayton, Melbourne, Victoria, 3168, Australia

About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Resveratrol

Drug

Resveratrol 1g daily (500mg twice daily) for 12 weeks Resveratrol 5 daily (2.5g twice daily) for 12 weeks

Primary outcomes

  1. Lymphocyte frataxin level

    Time frame: 12 weeks

    Change in lymphocyte frataxin levels at 12 weeks compared to baseline

Secondary outcomes

  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.

Sponsors and collaborators

Lead sponsor

Murdoch Childrens Research Institute

Other

Collaborators

  • Friedreich's Ataxia Research Alliance

Registry information

Official study title

An Open Label Clinical Pilot Study of Resveratrol as Treatment for Friedreich Ataxia

Important dates

Study start
2011
Primary completion
2012
Study completion
2012
First posted
Apr 21, 2011
Registry last updated
Jan 22, 2014

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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