Condition or disease | Intervention/treatment | Phase |
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Friedreich Ataxia | Drug: Resveratrol | Phase 2 |
Friedreich ataxia (FRDA) is the most common hereditary ataxia, with an estimated prevalence in Caucasians of 1 in 30,000. Neurological features of FRDA are progressive gait and limb ataxia, absent lower limb reflexes, and loss of position and vibration sense. There are currently no treatments proven to alter the natural history of FRDA. Resveratrol is a naturally occurring compound found in red wine, berries, and nuts. It is postulated to have wide-ranging health benefits, including antioxidant, anticarcinogenic, antidiabetic and neuroprotective properties.
The study will be a double-blinded, placebo-controlled randomised 2-period crossover trial of 2g/day of micronised resveratrol in FRDA over 24 weeks. The study will enrol 40 patients with FRDA from 3 sites. The primary outcome measure is the change in modified Friedreich Ataxia Rating Scale (mFARS) score from baseline to 24 weeks.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 40 participants |
Allocation: | Randomized |
Intervention Model: | Crossover Assignment |
Intervention Model Description: | Double-blind, randomised, placebo-controlled 2-period crossover trial of 2g/day of micronised resveratrol versus placebo. Participants will be randomised in terms of the order in which they received micronised resveratrol and placebo. |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Masking Description: | Participants will be randomised between receiving resveratrol in period 1 and placebo in period 2, or placebo in period 1 and resveratrol in period 2. |
Primary Purpose: | Treatment |
Official Title: | A Randomised Placebo-controlled Crossover Trial of Micronised Resveratrol as a Treatment for Friedreich Ataxia |
Actual Study Start Date : | May 23, 2019 |
Estimated Primary Completion Date : | August 2022 |
Estimated Study Completion Date : | August 2022 |
Arm | Intervention/treatment |
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Resveratrol followed by placebo
1g micronised resveratrol twice daily for 24 weeks, a wash-out period of 4 weeks, followed by twice daily placebo for 24 weeks.
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Drug: Resveratrol
Drug name: Micronised resveratrol. Dosage form: 500mg capsules. Alternate name: 1,3-Benzenediol, 5-[2-(4-hydroxyphenyl)ethenyl]-, (E). Ingredients: 99.50% pure trans-resveratrol. Placebo capsules will be identical in terms of taste, smell, and appearance.
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Placebo followed by Resveratrol
Twice daily placebo for 24 weeks, a wash-out period of 4 weeks, followed by 1g micronised resveratrol twice daily for 24 weeks
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Drug: Resveratrol
Drug name: Micronised resveratrol. Dosage form: 500mg capsules. Alternate name: 1,3-Benzenediol, 5-[2-(4-hydroxyphenyl)ethenyl]-, (E). Ingredients: 99.50% pure trans-resveratrol. Placebo capsules will be identical in terms of taste, smell, and appearance.
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Ages Eligible for Study: | 16 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contact: Geneieve Tai | +61 3 8341 6374 | geneieve.tai@mcri.edu.au |
Australia, New South Wales | |
Royal North Shore Hospital | Recruiting |
St Leonards, New South Wales, Australia, 2065 | |
Contact: Melanie Burk (02) 9463 1856 Melanie.Burk@health.nsw.gov.au | |
Principal Investigator: Christina Liang | |
Australia, Queensland | |
University of Queensland Centre for Clinical Research | Recruiting |
Herston, Queensland, Australia, 4029 | |
Contact: Elizabeth Arnold +61 7 3365 5147 elizabeth.arnold@uq.edu.au | |
Contact: Lily Tang +61 7 3646 3111 Lily.Tang@health.qld.gov.au | |
Principal Investigator: John O'Sullivan | |
Australia, Victoria | |
Murdoch Children's Research Institute | Recruiting |
Parkville, Victoria, Australia, 3052 | |
Contact: Geneieve Tai +61 3 8341 6374 geneieve.tai@mcri.edu.au | |
Principal Investigator: Martin Delatycki | |
Australia, Western Australia | |
Royal Perth Hospital | Recruiting |
Perth, Western Australia, Australia, 6000 | |
Contact: Lee Fyfe +61 8 9224 3353 Beverly.Fyfe@health.wa.gov.au | |
Principal Investigator: Phillipa Lamont |
Principal Investigator: | Martin B Delatycki, PhD, MBBS | Murdoch Children's Research Institute |
Tracking Information | |||||||||||||||||||
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First Submitted Date ICMJE | April 16, 2019 | ||||||||||||||||||
First Posted Date ICMJE | May 1, 2019 | ||||||||||||||||||
Last Update Posted Date | February 23, 2021 | ||||||||||||||||||
Actual Study Start Date ICMJE | May 23, 2019 | ||||||||||||||||||
Estimated Primary Completion Date | August 2022 (Final data collection date for primary outcome measure) | ||||||||||||||||||
Current Primary Outcome Measures ICMJE |
Modified Friedreich Ataxia Rating Scale [ Time Frame: 24 weeks ] Change in the Modified Friedreich Ataxia Rating Scale score (score range 0-99) at 24 weeks compared with baseline. Higher scores are indicative of more severe disease.
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Original Primary Outcome Measures ICMJE | Same as current | ||||||||||||||||||
Change History | |||||||||||||||||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE | Same as current | ||||||||||||||||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||||||||||||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||||||||||||||
Descriptive Information | |||||||||||||||||||
Brief Title ICMJE | Micronised Resveratrol as a Treatment for Friedreich Ataxia | ||||||||||||||||||
Official Title ICMJE | A Randomised Placebo-controlled Crossover Trial of Micronised Resveratrol as a Treatment for Friedreich Ataxia | ||||||||||||||||||
Brief Summary | The aim of this study is to assess the efficacy of micronised resveratrol as a treatment for FRDA, in terms of reducing the severity of ataxia symptoms at 24 weeks, through a randomised blinded, placebo controlled crossover trial. | ||||||||||||||||||
Detailed Description |
Friedreich ataxia (FRDA) is the most common hereditary ataxia, with an estimated prevalence in Caucasians of 1 in 30,000. Neurological features of FRDA are progressive gait and limb ataxia, absent lower limb reflexes, and loss of position and vibration sense. There are currently no treatments proven to alter the natural history of FRDA. Resveratrol is a naturally occurring compound found in red wine, berries, and nuts. It is postulated to have wide-ranging health benefits, including antioxidant, anticarcinogenic, antidiabetic and neuroprotective properties. The study will be a double-blinded, placebo-controlled randomised 2-period crossover trial of 2g/day of micronised resveratrol in FRDA over 24 weeks. The study will enrol 40 patients with FRDA from 3 sites. The primary outcome measure is the change in modified Friedreich Ataxia Rating Scale (mFARS) score from baseline to 24 weeks. |
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Study Type ICMJE | Interventional | ||||||||||||||||||
Study Phase ICMJE | Phase 2 | ||||||||||||||||||
Study Design ICMJE | Allocation: Randomized Intervention Model: Crossover Assignment Intervention Model Description: Double-blind, randomised, placebo-controlled 2-period crossover trial of 2g/day of micronised resveratrol versus placebo. Participants will be randomised in terms of the order in which they received micronised resveratrol and placebo. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)Masking Description: Participants will be randomised between receiving resveratrol in period 1 and placebo in period 2, or placebo in period 1 and resveratrol in period 2. Primary Purpose: Treatment
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Condition ICMJE | Friedreich Ataxia | ||||||||||||||||||
Intervention ICMJE | Drug: Resveratrol
Drug name: Micronised resveratrol. Dosage form: 500mg capsules. Alternate name: 1,3-Benzenediol, 5-[2-(4-hydroxyphenyl)ethenyl]-, (E). Ingredients: 99.50% pure trans-resveratrol. Placebo capsules will be identical in terms of taste, smell, and appearance.
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Study Arms ICMJE |
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Publications * | Not Provided | ||||||||||||||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||||||||||||||
Recruitment Status ICMJE | Recruiting | ||||||||||||||||||
Estimated Enrollment ICMJE |
40 | ||||||||||||||||||
Original Estimated Enrollment ICMJE | Same as current | ||||||||||||||||||
Estimated Study Completion Date ICMJE | August 2022 | ||||||||||||||||||
Estimated Primary Completion Date | August 2022 (Final data collection date for primary outcome measure) | ||||||||||||||||||
Eligibility Criteria ICMJE |
Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 16 Years and older (Child, Adult, Older Adult) | ||||||||||||||||||
Accepts Healthy Volunteers ICMJE | No | ||||||||||||||||||
Contacts ICMJE |
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Listed Location Countries ICMJE | Australia | ||||||||||||||||||
Removed Location Countries | |||||||||||||||||||
Administrative Information | |||||||||||||||||||
NCT Number ICMJE | NCT03933163 | ||||||||||||||||||
Other Study ID Numbers ICMJE | 36007 | ||||||||||||||||||
Has Data Monitoring Committee | Yes | ||||||||||||||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Responsible Party | Martin Delatycki, Murdoch Childrens Research Institute | ||||||||||||||||||
Study Sponsor ICMJE | Murdoch Childrens Research Institute | ||||||||||||||||||
Collaborators ICMJE | Not Provided | ||||||||||||||||||
Investigators ICMJE |
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PRS Account | Murdoch Childrens Research Institute | ||||||||||||||||||
Verification Date | February 2021 | ||||||||||||||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |