Clinical Trial: A Study of Resveratrol as Treatment for Friedreich Ataxia

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

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.

Detailed Summary: 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).
Sponsor: Murdoch Childrens Research Institute

Current Primary Outcome: Lymphocyte frataxin level [ Time Frame: 12 weeks ]

Change in lymphocyte frataxin levels at 12 weeks compared to baseline


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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.


Original Secondary Outcome:

  • 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
  • Cardiac parameters [ Time Frame: 12 weeks ]
    Changes in echocardiographic outcomes 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.


Information By: Murdoch Childrens Research Institute

Dates:
Date Received: April 14, 2011
Date Started: April 2011
Date Completion:
Last Updated: January 19, 2014
Last Verified: January 2014