A Study of Resveratrol as Treatment for Friedreich Ataxia

Friedreich Ataxia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01339884
• Other study ID #: 10358B
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: April 14, 2011
• Last updated: January 19, 2014
• Start date: April 2011
• Est. completion date: December 2012

Study information

• Verified date: January 2014
• Source: Murdoch Childrens Research Institute
• Contact: n/a
• Is FDA regulated: No
• Health authority: Australia: Therapeutic Goods Administration
• Study type: Interventional

Clinical Trial 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.

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 27
• Est. completion date: December 2012
• Est. primary completion date: August 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Friedreich Ataxia

Intervention

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

Locations

Country	Name	City	State
Australia	Monash Medical Centre, Southern Health	Clayton, Melbourne	Victoria

Sponsors (2)

Lead Sponsor	Collaborator
Murdoch Childrens Research Institute	Friedreich's Ataxia Research Alliance

Country where clinical trial is conducted

Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Lymphocyte frataxin level	Change in lymphocyte frataxin levels at 12 weeks compared to baseline	12 weeks	No
Secondary	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	12 weeks	No
Secondary	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	12 weeks	No
Secondary	Echocardiogram measures	Changes in structural and functional 3D echocardiogram measures from baseline to 12 weeks will be reported	12 weeks	No
Secondary	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.	First 2 hours post dose	No