A Study of Resveratrol as Treatment for Friedreich Ataxia

Phase 1

Completed

• Conditions: Friedreich Ataxia
• Interventions: Drug: Resveratrol

• Registration Number: NCT01339884
• Lead Sponsor: Murdoch Childrens Research Institute

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 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 Timeline

• Study Start: 2011-04
• Study First Submitted: 2011-04-14
• Study First Submitted QC: 2011-04-20
• Study First Posted: 2011-04-21
• Primary Completion: 2012-08
• Study Completion: 2012-12
• Status Verified: 2014-01
• Last Update Submitted: 2014-01-19
• Last Update Posted: 2014-01-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 27

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Resveratrol, 1g daily	Resveratrol	15 participants will receive resveratrol 1g daily
Resveratrol, 5g daily	Resveratrol	15 participants will receive resveratrol, 5g daily

Primary Outcome Measures

Name	Time	Method
Lymphocyte frataxin level	12 weeks	Change in lymphocyte frataxin levels at 12 weeks compared to baseline

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetic studies of resveratrol	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.
Oxidative stress markers	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	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	12 weeks	Changes in structural and functional 3D echocardiogram measures from baseline to 12 weeks will be reported

Trial Locations

Locations (1)

Monash Medical Centre, Southern Health; 🇦🇺 Clayton, Melbourne, Victoria, Australia