A Study of Resveratrol as Treatment for Friedreich Ataxia

Murdoch Childrens Research Institute

Status and phase

Completed

Phase 2

Phase 1

Conditions

Friedreich Ataxia

Treatments

Drug: Resveratrol

Study type

Interventional

Funder types

Other

Identifiers

NCT01339884

10358B

Details and patient eligibility

About

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.

Full 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).

Enrollment

27 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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