A Study of Resveratrol as Treatment for Friedreich Ataxia - Full Text View

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:

Genetics Home Reference related topics: ataxia neuropathy spectrum childhood myocerebrohepatopathy spectrum deoxyguanosine kinase deficiency Friedreich ataxia mitochondrial neurogastrointestinal encephalopathy disease myoclonic epilepsy myopathy sensory ataxia

MedlinePlus related topics: Friedreich's Ataxia

Drug Information available for: Resveratrol

Genetic and Rare Diseases Information Center resources: Friedreich Ataxia Spinocerebellar Ataxia

U.S. FDA Resources

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:


Ataxia Friedreich Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Diseases Dyskinesias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Neurologic Manifestations Signs and Symptoms Spinal Cord Diseases	Spinocerebellar Degenerations Resveratrol Analgesics Analgesics, Non-Narcotic Anti-Inflammatory Agents Anti-Inflammatory Agents, Non-Steroidal Anticarcinogenic Agents Antimutagenic Agents Antineoplastic Agents Antineoplastic Agents, Phytogenic Antioxidants Antirheumatic Agents Central Nervous System Agents Enzyme Inhibitors Hematologic Agents

ClinicalTrials.gov processed this record on March 03, 2015