A Study of Resveratrol as Treatment for Friedreich Ataxia

Murdoch Childrens Research Institute27 enrolled

Overview

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.

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 Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Friedreich Ataxia

Interventions

ResveratrolDRUG

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

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (1)

Monash Medical Centre, Southern Health

Clayton, Melbourne, Victoria, Australia

Outcomes

Primary Outcomes

Lymphocyte frataxin level

Change in lymphocyte frataxin levels at 12 weeks compared to baseline

Time frame: 12 weeks

Secondary Outcomes

Oxidative stress markers

Oxidative stress, as measured by a) plasma F2-isoprostanes and b) urinary 8-hydroxyl-2-deoxyguanosine levels at 12 weeks compared to baseline

Time frame: 12 weeks

Clinical rating scales of ataxia

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

Time frame: 12 weeks

Echocardiogram measures

Changes in structural and functional 3D echocardiogram measures from baseline to 12 weeks will be reported

Time frame: 12 weeks

Pharmacokinetic studies of resveratrol

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.

Time frame: First 2 hours post dose

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.