A Study of Resveratrol as Treatment for Friedreich Ataxia

• Lymphocyte frataxin level [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  Change in lymphocyte frataxin levels at 12 weeks compared to baseline
  
  

• Oxidative stress markers [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  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 ] [ Designated as safety issue: No ]
  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 ] [ Designated as safety issue: No ]
  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 ] [ Designated as safety issue: No ]
  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.
  
  

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).