A Study of Resveratrol as Treatment for Friedreich Ataxia

This study has been completed.
Sponsor:
Collaborator:
Friedreich's Ataxia Research Alliance
Information provided by (Responsible Party):
Martin Delatycki, Murdoch Childrens Research Institute
ClinicalTrials.gov Identifier:
NCT01339884
First received: April 14, 2011
Last updated: January 19, 2014
Last verified: January 2014
  Purpose

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.


Condition Intervention Phase
Friedreich Ataxia
Drug: Resveratrol
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open Label Clinical Pilot Study of Resveratrol as Treatment for Friedreich Ataxia

Resource links provided by NLM:


Further study details as provided by Murdoch Childrens Research Institute:

Primary Outcome Measures:
  • Lymphocyte frataxin level [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
    Change in lymphocyte frataxin levels at 12 weeks compared to baseline


Secondary Outcome Measures:
  • Oxidative stress markers [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
    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 [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
    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 [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
    Changes in structural and functional 3D echocardiogram measures from baseline to 12 weeks will be reported

  • Pharmacokinetic studies of resveratrol [ Time Frame: First 2 hours post dose ] [ Designated as safety issue: No ]
    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.


Enrollment: 27
Study Start Date: April 2011
Study Completion Date: December 2012
Primary Completion Date: August 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Resveratrol, 1g daily
15 participants will receive resveratrol 1g daily
Drug: Resveratrol
Resveratrol 1g daily (500mg twice daily) for 12 weeks Resveratrol 5 daily (2.5g twice daily) for 12 weeks
Active Comparator: Resveratrol, 5g daily
15 participants will receive resveratrol, 5g daily
Drug: Resveratrol
Resveratrol 1g daily (500mg twice daily) for 12 weeks Resveratrol 5 daily (2.5g twice daily) for 12 weeks

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).

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01339884

Locations
Australia, Victoria
Monash Medical Centre, Southern Health
Clayton, Melbourne, Victoria, Australia, 3168
Sponsors and Collaborators
Murdoch Childrens Research Institute
Friedreich's Ataxia Research Alliance
Investigators
Principal Investigator: Martin Delatycki, MBBS PhD Murdoch Childrens Research Institute
  More Information

No publications provided

Responsible Party: Martin Delatycki, Prof Martin Delatycki, Murdoch Childrens Research Institute
ClinicalTrials.gov Identifier: NCT01339884     History of Changes
Other Study ID Numbers: 10358B
Study First Received: April 14, 2011
Last Updated: January 19, 2014
Health Authority: Australia: Therapeutic Goods Administration

Keywords provided by Murdoch Childrens Research Institute:
Friedreich ataxia
Resveratrol
Frataxin
Cardiomyopathy
Oxidative stress

Additional relevant MeSH terms:
Friedreich Ataxia
Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinocerebellar Degenerations
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Resveratrol
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Analgesics
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Pharmacologic Actions
Anti-Inflammatory Agents
Therapeutic Uses
Antirheumatic Agents
Antineoplastic Agents, Phytogenic
Antineoplastic Agents
Antioxidants

ClinicalTrials.gov processed this record on September 22, 2014