A Study of Resveratrol as Treatment for Friedreich Ataxia

NCT01339884 | Completed Phase 1 DMC

• 1 other identifier: 10358B
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 1

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.

Conditions

Friedreich Ataxia

Keywords

Friedreich ataxia; Resveratrol; Frataxin; Cardiomyopathy; Oxidative stress

Outcome Measures

Primary Outcomes (1)

• Lymphocyte frataxin level

  Change in lymphocyte frataxin levels at 12 weeks compared to baseline

  12 weeks

Secondary Outcomes (4)

• Oxidative stress markers

  12 weeks

• Clinical rating scales of ataxia

  12 weeks

• Echocardiogram measures

  12 weeks

• Pharmacokinetic studies of resveratrol

  First 2 hours post dose

Study Arms (2)

Resveratrol, 1g daily

ACTIVE COMPARATOR

15 participants will receive resveratrol 1g daily

Drug: Resveratrol

Resveratrol, 5g daily

ACTIVE COMPARATOR

15 participants will receive resveratrol, 5g daily

Drug: Resveratrol

Interventions

Resveratrol DRUG

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

Resveratrol, 1g daily; Resveratrol, 5g daily

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Murdoch Childrens Research Institute: lead
• Friedreich's Ataxia Research Alliance: collaborator

Study Sites (1)

Monash Medical Centre, Southern Health

Clayton, Melbourne, Victoria, 3168, Australia

Location

MeSH Terms

Conditions

Friedreich Ataxia; Cardiomyopathies

Interventions

Resveratrol

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Heart Diseases; Cardiovascular Diseases

Intervention Hierarchy (Ancestors)

Stilbestrols; Stilbenes; Benzylidene Compounds; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polyphenols; Phenols

Study Officials

• Martin Delatycki, MBBS PhD

  Murdoch Childrens Research Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof Martin Delatycki

Study Record Dates

• First Submitted: April 14, 2011
• First Posted: April 21, 2011
• Study Start: April 1, 2011
• Primary Completion: August 1, 2012
• Study Completion: December 1, 2012
• Last Updated: January 22, 2014

Record last verified: 2014-01

Locations

AU (1)