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A Study of Resveratrol as Treatment for Friedreich Ataxia

Primary Purpose

Friedreich Ataxia

Status
Completed
Phase
Phase 1
Locations
Australia
Study Type
Interventional
Intervention
Resveratrol
Sponsored by
Murdoch Childrens Research Institute
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Friedreich Ataxia focused on measuring Friedreich ataxia, Resveratrol, Frataxin, Cardiomyopathy, Oxidative stress

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • Monash Medical Centre, Southern Health

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Active Comparator

Arm Label

Resveratrol, 1g daily

Resveratrol, 5g daily

Arm Description

15 participants will receive resveratrol 1g daily

15 participants will receive resveratrol, 5g daily

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

Full Information

First Posted
April 14, 2011
Last Updated
January 19, 2014
Sponsor
Murdoch Childrens Research Institute
Collaborators
Friedreich's Ataxia Research Alliance
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1. Study Identification

Unique Protocol Identification Number
NCT01339884
Brief Title
A Study of Resveratrol as Treatment for Friedreich Ataxia
Official Title
An Open Label Clinical Pilot Study of Resveratrol as Treatment for Friedreich Ataxia
Study Type
Interventional

2. Study Status

Record Verification Date
January 2014
Overall Recruitment Status
Completed
Study Start Date
April 2011 (undefined)
Primary Completion Date
August 2012 (Actual)
Study Completion Date
December 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Murdoch Childrens Research Institute
Collaborators
Friedreich's Ataxia Research Alliance

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Friedreich Ataxia
Keywords
Friedreich ataxia, Resveratrol, Frataxin, Cardiomyopathy, Oxidative stress

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
27 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Resveratrol, 1g daily
Arm Type
Active Comparator
Arm Description
15 participants will receive resveratrol 1g daily
Arm Title
Resveratrol, 5g daily
Arm Type
Active Comparator
Arm Description
15 participants will receive resveratrol, 5g daily
Intervention Type
Drug
Intervention Name(s)
Resveratrol
Intervention Description
Resveratrol 1g daily (500mg twice daily) for 12 weeks Resveratrol 5 daily (2.5g twice daily) for 12 weeks
Primary Outcome Measure Information:
Title
Lymphocyte frataxin level
Description
Change in lymphocyte frataxin levels at 12 weeks compared to baseline
Time Frame
12 weeks
Secondary Outcome Measure Information:
Title
Oxidative stress markers
Description
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
Title
Clinical rating scales of ataxia
Description
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
Title
Echocardiogram measures
Description
Changes in structural and functional 3D echocardiogram measures from baseline to 12 weeks will be reported
Time Frame
12 weeks
Title
Pharmacokinetic studies of resveratrol
Description
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Martin Delatycki, MBBS PhD
Organizational Affiliation
Murdoch Childrens Research Institute
Official's Role
Principal Investigator
Facility Information:
Facility Name
Monash Medical Centre, Southern Health
City
Clayton, Melbourne
State/Province
Victoria
ZIP/Postal Code
3168
Country
Australia

12. IPD Sharing Statement

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A Study of Resveratrol as Treatment for Friedreich Ataxia

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