Friedreich AtaxiaPhase 1/2NCT01339884
A Study of Resveratrol as Treatment for Friedreich Ataxia
A Phase 1/2 study testing Resveratrol in people with Friedreich Ataxia, run by Murdoch Childrens Research Institute at 1 site in Australia.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Friedreich Ataxia
- What is being tested?
- Resveratrol
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 1 siteAustralia
- Study dates
- Apr 2011to Dec 2012
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
Australia1 site
Monash Medical Centre, Southern Health
Clayton, Melbourne, Victoria, 3168
About this study
Official title: An Open Label Clinical Pilot Study of Resveratrol as Treatment for Friedreich Ataxia
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.
Read the 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).
- Conditions
- Friedreich Ataxia
- Being tested
- Resveratrol
- Keywords
- Friedreich ataxia, Resveratrol, Frataxin, Cardiomyopathy, Oxidative stress
- Medical subject headings
- Friedreich Ataxia, Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ResveratrolDrug
Resveratrol 1g daily (500mg twice daily) for 12 weeks Resveratrol 5 daily (2.5g twice daily) for 12 weeks
| Study group | Role | What they receive |
|---|---|---|
| Resveratrol, 1g daily | Active comparator | 15 participants will receive resveratrol 1g daily Receives: Drug: Resveratrol |
| Resveratrol, 5g daily | Active comparator | 15 participants will receive resveratrol, 5g daily Receives: Drug: Resveratrol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Lymphocyte frataxin level
Change in lymphocyte frataxin levels at 12 weeks compared to baseline
Measured: 12 weeks
Secondary outcomes (4)
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
Measured: 12 weeks
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
Measured: 12 weeks
Echocardiogram measures
Changes in structural and functional 3D echocardiogram measures from baseline to 12 weeks will be reported
Measured: 12 weeks
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.
Measured: First 2 hours post dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Apr 2011 (estimated)
- Study startApr 2011
- Primary completionAug 2012
- Study completionDec 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich AtaxiaStudies of the same condition, open and under way.
- More trials run by Murdoch Childrens Research InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Murdoch Childrens Research Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Friedreich's Ataxia Research Alliance
- Responsible party
- Principal investigator, Martin Delatycki, Murdoch Childrens Research Institute
- Investigators
- Martin Delatycki, MBBS PhD, Principal investigator, Murdoch Childrens Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01339884
- Sponsor's protocol ID
- 10358B
- Status last verified
- Jan 2014
- First submitted
- 14 Apr 2011
- Last updated
- 22 Jan 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 22 Jan 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Clinical Pilot Study of Resveratrol as Treatment for Friedreich Ataxia
- ClinicalTrials.gov ID
- NCT01339884
- Acronym / sponsor ID
- 10358B
- Conditions
- Friedreich Ataxia
- Keywords
- Friedreich ataxia, Resveratrol, Frataxin, Cardiomyopathy, Oxidative stress
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Murdoch Childrens Research InstituteUniversity, hospital or other
- Collaborators
- Friedreich's Ataxia Research Alliance
- Study start
- Apr 2011
- Primary completion
- Aug 2012 (actual)
- Study completion
- Dec 2012 (actual)
- First posted
- 21 Apr 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Resveratrol, 1g daily | Active comparator | Drug: Resveratrol15 participants will receive resveratrol 1g daily |
| Resveratrol, 5g daily | Active comparator | Drug: Resveratrol15 participants will receive resveratrol, 5g daily |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ResveratrolResveratrol 1g daily (500mg twice daily) for 12 weeks Resveratrol 5 daily (2.5g twice daily) for 12 weeks | Drug | Resveratrol, 1g daily, Resveratrol, 5g daily |
Primary outcome
Lymphocyte frataxin level
Time frame: 12 weeks
Change in lymphocyte frataxin levels at 12 weeks compared to baseline
4 secondary outcomes
Oxidative stress markers
Time frame: 12 weeks
Clinical rating scales of ataxia
Time frame: 12 weeks
Echocardiogram measures
Time frame: 12 weeks
Pharmacokinetic studies of resveratrol
Time frame: First 2 hours post dose
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jan 2014.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Martin Delatycki, MBBS PhD principal investigator
Murdoch Childrens Research Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Resveratrol in people with Friedreich Ataxia, run by Murdoch Childrens Research Institute at 1 site in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Jan 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Lymphocyte frataxin level (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jan 2014.