Sporadic AtaxiaPhase 1CoQATAXNCT00957216
Coenzyme Q10 in Adult-Onset Ataxia
A Phase 1 study testing Coenzyme Q10 in people with Sporadic Ataxia, run by The University of Texas Medical Branch, Galveston at 1 site in United States.
Trial summary
This is a Physician-sponsored pilot study, whose purpose it is determine if high-dose oral Coenzyme Q10 (CoQ10) is safe and tolerated in patients with sporadic forms of adult-onset spinocerebellar ataxias (SAOA), a group of degenerative neurological disorders affecting the cerebellum and pathways to and from the… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Sporadic Ataxia
- What is being tested?
- Coenzyme Q10
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2008to Aug 2009
- Phase
- Phase 1
- 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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of SAOA
- Age 18 or older
- Adult onset of ataxia
- Ambulatory capability (with or without an assisting device)
- Women with 2 years post menopause or surgical sterility or practicing adequate birth control
- Stable doses of psychotropic drugs
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- Stable doses of drugs for movement disorders
- Ability to give informed consent
- Ability to comply with trial procedures
- Able to take oral medication
- No active and significant systemic disease (cardiac, pulmonary, hepatic, renal disease or cancer) that is not under adequate medical control
- Women with child-bearing potential who have a negative urine pregnancy test and practice adequate contraception during the study
Who cannot join
- A history or known sensitivity of intolerability to Coenzyme Q10
- Diagnosis of secondary (non-degenerative) ataxia
- Family history of degenerative ataxia
- Diagnosis of childhood-onset ataxia
- DNA diagnosis of inherited ataxia in the absence of family history
- Other investigational agent within 30 days of screening
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- Ingestion of Coenzyme Q10 within 120 days of the baseline visit
- Diagnosis of ongoing malignancy
- Women who are pregnant or lactating or who have child bearing potential and not using effective birth control
- Uncontrolled hypertension
- Symptomatic orthostatic hypotension
- Uncontrolled diabetes mellitus
- Untreated thyroid disease
- Major psychiatric disease within 12 months of screening
- History of non-compliance with other therapies
- Drug or alcohol abuse within 12 months of screening
- Other condition or therapy that may prevent participation in the opinion of the investigator
Trial locations and contacts
1 site in 1 country.
United States1 site
The Universtity of Texas, Galveston
Galveston, Texas, 77555-0539
About this study
Official title: Safety and Tolerability of Coenzyme Q10 in Adult-Onset Sporadic Spinocerebellar Ataxia
This is a Physician-sponsored pilot study, whose purpose it is determine if high-dose oral Coenzyme Q10 (CoQ10) is safe and tolerated in patients with sporadic forms of adult-onset spinocerebellar ataxias (SAOA), a group of degenerative neurological disorders affecting the cerebellum and pathways to and from the cerebellum, with or without additional central nervous system (CNS) manifestations, in the absence of family history of degenerative ataxias.
Read the detailed description
This is a Physician-sponsored pilot study, whose purpose it is determine if high-dose oral Coenzyme Q10 (CoQ10) is safe and tolerated in patients with sporadic forms of adult-onset spinocerebellar ataxias (SAOA), a group of degenerative neurological disorders affecting the cerebellum and pathways to and from the cerebellum, with or without additional central nervous system (CNS) manifestations, in the absence of family history of degenerative ataxias.
CoQ10 is an essential cofactor of the electron transport chain and is a potent free radical scavenger in lipid and mitochondrial membranes. CoQ10 has shown efficacy in treatment of Parkinson's disease patients, and a Huntington's disease trial gave a trend of slowing down disease progression (CARE-HD). A small trial of CoQ10 in patients with Friedreich's ataxia suggested potential beneficial effects on ventricular thickness. CoQ10 is being tested on ALS patients and is considered to be potentially useful for treatment of Alzheimer's disease. Thus, CoQ10 is considered to be a promising therapeutic agent that might slow down the disease progression in a wide variety of neurodegenerative disorders. To date, very high doses of CoQ10 have not been used in patients with ataxia and the safety and tolerability in this group of patients should be established before efficacy trials are launched.
Twenty patients with SAOA will be recruited for a double-blind, randomized, placebo-controlled, multicenter study. Fifteen patients will receive a total of 2400 mg of oral CoQ10 daily, and five patients will receive placebo, for a period or 4 weeks. Cerebellar functions will be measured using a validated rating scale (SARA), an oculomotor examination, and functional measurement of motor function using a 9 hole peg test and timed walk. Safety labs will be collected and a digital movie will be recorded at the beginning (prior to treatment with CoQ10) and at the end of the study.
- Conditions
- Sporadic Ataxia
- Being tested
- Placebo (sugar pill), Coenzyme Q10
- Keywords
- adult onset ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Placebo (sugar pill)Drug
placebo: 2 wafers 4 times a day
Coenzyme Q10Drug
Also called CoQ10
300mg wafers; 2 wafers 4 times a day
| Study group | Role | What they receive |
|---|---|---|
| sugar pill | Placebo comparator | Not described Receives: Drug: Placebo (sugar pill) |
| Coenzyme Q10 | Active comparator | The CoQ10 arm will be compared with the placebo arm to determine if high-dose CoQ10 is safe and well tolerated in subjects with sporadic adult-onset spinocerebellar ataxias Receives: Drug: Coenzyme Q10 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Measure cerebellar functions by a validated scale (SARA)
Measured: 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Aug 2009 (estimated)
- Study startApr 2008
- Primary completionAug 2009
- Study completionAug 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sporadic AtaxiaStudies of the same condition, open and under way.
- More trials run by The University of Texas Medical Branch, GalvestonThe 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
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- University of Florida
- Responsible party
- Sponsor
- Investigators
- Sankarasubramoney H Subramony, MD, Principal investigator, University of Texas Medical Branch, Galveston
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00957216
- Sponsor's protocol ID
- 08-129
- Other IDs
- 08-SAOA-001
- Acronym
- CoQATAX
- Status last verified
- Apr 2010
- First submitted
- 22 Jun 2009
- Last updated
- 11 Jul 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jul 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Tolerability of Coenzyme Q10 in Adult-Onset Sporadic Spinocerebellar Ataxia
- ClinicalTrials.gov ID
- NCT00957216
- Acronym / sponsor ID
- CoQATAX / 08-129
- Conditions
- Sporadic Ataxia
- Keywords
- adult onset ataxia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 1 participants (actual)
- Lead sponsor
- The University of Texas Medical Branch, GalvestonUniversity, hospital or other
- Collaborators
- University of Florida
- Study start
- Apr 2008
- Primary completion
- Aug 2009 (actual)
- Study completion
- Aug 2009 (actual)
- First posted
- 12 Aug 2009 (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.
This is a Physician-sponsored pilot study, whose purpose it is determine if high-dose oral Coenzyme Q10 (CoQ10) is safe and tolerated in patients with sporadic forms of adult-onset spinocerebellar ataxias (SAOA), a group of degenerative neurological disorders affecting the cerebellum and pathways to and from the cerebellum, with or without additional central nervous system (CNS) manifestations, in the absence of family history of degenerative ataxias.
Detailed description
This is a Physician-sponsored pilot study, whose purpose it is determine if high-dose oral Coenzyme Q10 (CoQ10) is safe and tolerated in patients with sporadic forms of adult-onset spinocerebellar ataxias (SAOA), a group of degenerative neurological disorders affecting the cerebellum and pathways to and from the cerebellum, with or without additional central nervous system (CNS) manifestations, in the absence of family history of degenerative ataxias.
CoQ10 is an essential cofactor of the electron transport chain and is a potent free radical scavenger in lipid and mitochondrial membranes. CoQ10 has shown efficacy in treatment of Parkinson's disease patients, and a Huntington's disease trial gave a trend of slowing down disease progression (CARE-HD). A small trial of CoQ10 in patients with Friedreich's ataxia suggested potential beneficial effects on ventricular thickness. CoQ10 is being tested on ALS patients and is considered to be potentially useful for treatment of Alzheimer's disease. Thus, CoQ10 is considered to be a promising therapeutic agent that might slow down the disease progression in a wide variety of neurodegenerative disorders. To date, very high doses of CoQ10 have not been used in patients with ataxia and the safety and tolerability in this group of patients should be established before efficacy trials are launched.
Twenty patients with SAOA will be recruited for a double-blind, randomized, placebo-controlled, multicenter study. Fifteen patients will receive a total of 2400 mg of oral CoQ10 daily, and five patients will receive placebo, for a period or 4 weeks. Cerebellar functions will be measured using a validated rating scale (SARA), an oculomotor examination, and functional measurement of motor function using a 9 hole peg test and timed walk. Safety labs will be collected and a digital movie will be recorded at the beginning (prior to treatment with CoQ10) and at the end of the study.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| sugar pill | Placebo comparator | Drug: Placebo (sugar pill) |
| Coenzyme Q10 | Active comparator | Drug: Coenzyme Q10The CoQ10 arm will be compared with the placebo arm to determine if high-dose CoQ10 is safe and well tolerated in subjects with sporadic adult-onset spinocerebellar ataxias |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Diagnosis of SAOA
- Age 18 or older
- Adult onset of ataxia
- Ambulatory capability (with or without an assisting device)
- Women with 2 years post menopause or surgical sterility or practicing adequate birth control
Key exclusion criteria (17 listed)
- A history or known sensitivity of intolerability to Coenzyme Q10
- Diagnosis of secondary (non-degenerative) ataxia
- Family history of degenerative ataxia
- Diagnosis of childhood-onset ataxia
- DNA diagnosis of inherited ataxia in the absence of family history
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 |
|---|---|---|
| Placebo (sugar pill)placebo: 2 wafers 4 times a day | Drug | sugar pill |
| Coenzyme Q10Also: CoQ10300mg wafers; 2 wafers 4 times a day | Drug | Coenzyme Q10 |
Primary outcome
Measure cerebellar functions by a validated scale (SARA)
Time frame: 6 weeks
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 11 Jul 2018.
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Sporadic Degenerative Ataxia With Adult Onset: Natural History Study
Late Onset Sporadic Cerebellar Ataxia
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.
Sankarasubramoney H Subramony, MD principal investigator
University of Texas Medical Branch, Galveston
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Coenzyme Q10 in people with Sporadic Ataxia, run by The University of Texas Medical Branch, Galveston at 1 site in United States.
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 12 inclusion and 17 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 11 Jul 2018. 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 United States. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
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?
Measure cerebellar functions by a validated scale (SARA) (time frame: 6 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 11 Jul 2018.