Parkinson Disease
Conditions
Keywords
Parkinson Disease, ubiquinol, CoQ10, magnetic resonance spectroscopy, lactate, glutathione
Brief summary
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.
Detailed description
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.
Interventions
DRUGUbiquinol
Ubiquinol caplets 600mg/day
DIETARY_SUPPLEMENTPlacebo
placebo
Sponsors
Weill Medical College of Cornell University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
40 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* diagnosis of PD according to the Untied Kingdom (UK) Brain Bank Criteria, and confirmed by a Movement Disorders neurologist; * age 40-75 years; diagnosis within 5 years of study participation; * PD medications able to remain at stable doses in the opinion of the enrolling investigator; * able to undergo MRI; * absence of significant medical, psychiatric, and other neurological disease; * absence of dementia and Mini-Mental State Examination (MMSE) \> 26.
Exclusion criteria
* failure to meet diagnosis by above criteria; * time since diagnosis \> 5 years before study participation; * PD medications not predicted to remain at stable doses in the opinion of the enrolling investigator; * unable to undergo MRI; * unable to comply with informed consent process; * presence of significant medical, psychiatric (including major depressive disorder) or other neurological (including epilepsy, brain tumor, stroke) disease; * diagnosis of dementia and/or MMSE 26 or lower; * possibility of pregnancy (negative test required in women of childbearing age); * taking medications including antipsychotic agents and dopamine- blocking anti-emetic agents; * taking Coenzyme Q10; * participation in another clinical trial within the last 3 months.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Adverse Events | at 24 weeks | The incidence and severity of adverse events in Parkinson disease patients taking 600mg ubiquinol or placebo daily over a 6 month period. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Cerebral Redox Markers | at baseline and 8 weeks | Change from baseline in lactate levels at 8 weeks as determined by Magnetic Resonance Spectroscopy |
Participant flow
Recruitment details
Recruited from the clinical practice at Weill Cornell Medicine's (WCM) Parkinson's Disease Department as well as Weill Cornell Medicine, and utilized flyers.
Pre-assignment details
One screening visit consisting of: neurological examination, interview on health topics, and questionnaires on health topics.
Participants by arm
| Arm | Count |
|---|---|
| Ubiquinol 600mg ubiquinol daily for 24 weeks
Ubiquinol: Ubiquinol caplets 600mg/day | 6 |
| Placebo Placebo daily for 24 weeks
Placebo: placebo | 4 |
| Total | 10 |
Baseline characteristics
| Characteristic | Ubiquinol | Placebo | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 3 Participants | 4 Participants | 7 Participants |
| Age, Categorical Between 18 and 65 years | 3 Participants | 0 Participants | 3 Participants |
| Region of Enrollment United States | 6 Participants | 4 Participants | 10 Participants |
| Sex: Female, Male Female | 0 Participants | 2 Participants | 2 Participants |
| Sex: Female, Male Male | 6 Participants | 2 Participants | 8 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 5 / 6 | 3 / 5 |
| serious Total, serious adverse events | 0 / 6 | 0 / 5 |
Outcome results
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.
Time frame: at 24 weeks
Population: ITT
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ubiquinol | Number of Adverse Events | 27 Number of Adverse Events |
| Placebo | Number of Adverse Events | 12 Number of Adverse Events |
Secondary
Cerebral Redox Markers
Change from baseline in lactate levels at 8 weeks as determined by Magnetic Resonance Spectroscopy
Time frame: at baseline and 8 weeks
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ubiquinol | Cerebral Redox Markers | -11.98 Ratio | Standard Deviation 18.57 |
| Placebo | Cerebral Redox Markers | 6.23 Ratio | Standard Deviation 23.19 |