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The purpose of this study is to find out whether ubiquinol is well tolerated, can affect the symptoms of Parkinson's Disease and change the energy levels in the brain. Subjects will be randomized to taking ubiquinol or placebo and will have a neurological evaluation, magnetic resonance spectroscopy (MRS) and blood test for biological markers taken during the study.
Multiple lines of evidence have implicated abnormal energy metabolism and deficient mitochondrial function in Parkinson's disease, presenting a unique target for therapy. A pilot study of ubiquinol in PD was therefore undertaken to determine its effects upon physiologic measures of mitochondrial metabolic function. The incorporation of a neuroimaging biomarker is particularly important, since changes would demonstrate our ability to achieve Central Nervous System (CNS) access from this an formulation, accompanied by a meaningful neurophysiologic effect. Hydrogen Proton Magnetic Resonance Spectroscopy Imaging (1H MRSI) is a technique that provides insight into the metabolism of several endogenous brain compounds, most notably N-acetyl-L-aspartate (NAA), choline-containing compounds (Cho), and creatine and phosphocreatine (Cr). A number of studies of mitochondrial function have now firmly established the utility of 1H MRSI in probing potential mitochondrial energy metabolism dysfunction, in primary mitochondrial disorders, but also in PD. This pilot study is therefore designed to test whether oral ubiquinol affects cerebral indices of mitochondrial dysfunction, as measured by 1H MRSI in patients with Parkinson's disease, and to gather preliminary information on the safety and tolerability of ubiquinol in individuals with PD.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ubiquinol | Active Comparator | 600mg ubiquinol daily for 24 weeks |
|
| Placebo | Placebo Comparator | Placebo daily for 24 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ubiquinol | Drug | Ubiquinol caplets 600mg/day |
| |
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Adverse Events | The incidence and severity of adverse events in Parkinson disease patients taking 600mg ubiquinol or placebo daily over a 6 month period. | at 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Cerebral Redox Markers | Change from baseline in lactate levels at 8 weeks as determined by Magnetic Resonance Spectroscopy | at baseline and 8 weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Claire Henchcliffe, MD DPhil | Weill Medical College of Cornell University | Principal Investigator |
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One screening visit consisting of: neurological examination, interview on health topics, and questionnaires on health topics.
Recruited from the clinical practice at Weill Cornell Medicine's (WCM) Parkinson's Disease Department as well as Weill Cornell Medicine, and utilized flyers.
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| ID | Title | Description |
|---|---|---|
| FG000 | Ubiquinol | 600mg ubiquinol daily for 24 weeks Ubiquinol: Ubiquinol caplets 600mg/day |
| FG001 | Placebo | Placebo daily for 24 weeks Placebo: placebo |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Ubiquinol | 600mg ubiquinol daily for 24 weeks Ubiquinol: Ubiquinol caplets 600mg/day |
| BG001 | Placebo | Placebo daily for 24 weeks Placebo: placebo |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Adverse Events | The incidence and severity of adverse events in Parkinson disease patients taking 600mg ubiquinol or placebo daily over a 6 month period. | ITT | Posted | Number | Number of Adverse Events | at 24 weeks |
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Ubiquinol | 600mg ubiquinol daily for 24 weeks Ubiquinol: Ubiquinol caplets 600mg/day |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Fever | General disorders | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Claire Henchcliffe | Weill Cornell Medical College | 2127462584 | clh2007@med.cornell.edu |
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| ID | Term |
|---|---|
| D010300 | Parkinson Disease |
| ID | Term |
|---|---|
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| ID | Term |
|---|---|
| C003741 | ubiquinol |
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| Dietary Supplement |
placebo |
|
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
| Units |
|---|
| Counts |
|---|
| Participants |
|
|
| Secondary | Cerebral Redox Markers | Change from baseline in lactate levels at 8 weeks as determined by Magnetic Resonance Spectroscopy | Posted | Mean | Standard Deviation | Ratio | at baseline and 8 weeks |
|
|
|
| 0 |
| 6 |
| 5 |
| 6 |
| EG001 | Placebo | Placebo daily for 24 weeks Placebo: placebo | 0 | 5 | 3 | 5 |
| Motor Vehicle Accident | General disorders | Non-systematic Assessment |
|
| Common Cold Syndrome | Infections and infestations | Systematic Assessment |
|
| Dizziness | General disorders | Systematic Assessment |
|
| Sleepiness | General disorders | Systematic Assessment |
|
| Skin rash, itching | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Nausea | General disorders | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | Systematic Assessment |
|
| Upset stomach | Gastrointestinal disorders | Systematic Assessment |
|
| Cognitive decline | Nervous system disorders | Systematic Assessment |
|
| Depression | Psychiatric disorders | Systematic Assessment |
|
| Moodiness | General disorders | Systematic Assessment |
|
| Stomatitis | General disorders | Systematic Assessment |
|
| Lumbar pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Sudden falls | General disorders | Systematic Assessment |
|
| Peripheral edema | Vascular disorders | Systematic Assessment |
|
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| D009422 | Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |