Progressive Supranuclear Palsy
Conditions
Keywords
Progressive Supranuclear Palsy, CoQ10, Coenzyme Q10
Brief summary
The clinical syndrome of PSP responds poorly to all available forms of therapy used in Parkinson's Disease (PD). Currently, no effective treatment exists. Coenzyme Q10 in high doses has been shown to be a beneficial therapy in PD and might possibly be a beneficial therapy for PSP. This study will compare the efficacy, safety and tolerability of Coenzyme Q10 versus placebo in patients with atypical parkinsonian syndrome, progressive supranuclear palsy (PSP).
Detailed description
The study is designed as a multicenter randomized, placebo-controlled, double blind trial. Up to 60 patients with PSP will be enrolled at several centers in the United States
Interventions
active drug
Placebo
Sponsors
Study design
Intervention model description
Multicenter, Double-blind, Randomized, Placebo-Controlled Clinical Trial to examine tolerability, safety, and efficacy of CoenzymeQ10
Eligibility
Inclusion criteria
* Patients fulfilling the diagnostic criteria of PSP/diagnosed within the past 5 years * Either vertical supranuclear palsy or both slowing of vertical saccades * Prominent postural instability with falls in the first year of disease onset * No evidence of other diseases that could explain the foregoing features
Exclusion criteria
* Current or previous therapeutic use of CoQ10 * Parkinsonism due to drugs * History of pallidotomy, thalamotomy, active deep brain stimulator or fetal tissue transplant * History of active epilepsy, stroke, structural brain disease * Use of methylphenidate hydrochloride, cinnarizine, reserpine, amphetamines, or monoamine oxidase-A inhibitors within 3 months before the baseline visit. * Active cancer or cancer undergoing treatment * Participation in other drug studies or the use of other investigational drugs within 30 days before screening. * Known hypersensitivity to Coenzyme Q10. * Pregnant, planning a pregnancy or nursing woman
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy of Coenzyme Q10 (Unified Parkinson's Disease Rating Scale [UPDRS] AND Progressive Supranuclear Palsy Rating Scale [PSPRS]) | 12 months | Unified Parkinson's Disease Rating Scale \[UPDRS\]: Higher scores indicate a higher degree of impairment The total score is calculated by summing the subscores. Total scores range from 0 (normal) to 166 (severely impaired) Part I \[Mentation, Behavior, & Mood\] Scale: 0-16 Part II \[Activities of Daily Living, Both ON & OFF\] Scale:0-52 Part III \[Motor Examination\] Scale: 0-56 Part IV \[Complications of Therapy\] Scale: 0-34 Part V \[Modified Hoehn & Yahr Staging\] Scale: 0-8 Part VI \[Schwab & England Activities of Daily Living Scale\] Scale: 0 - 100% Progressive Supranuclear Palsy Rating Scale \[PSPRS\] - Total Scale ranges from 0 (normal) to 128 (severely impaired) Section 1 \[History\] Scale: 0-31 Section 2 \[Mentation\] Scale: 0-20 Section 3 \[Bulbar\] Scale: 0-10 Section 4 \[Ocular Motor\] Scale: 0-20 Section 5 \[Limb Motor\] Scale: 0-22 Section 6 \[Gait and midline\] Scale: 0-25 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy of Coenzyme Q10 (Mini-Mental State Examination [MMSE] and Activities of Daily Living [ADL] Scales) | 12 months | Efficacy of Coenzyme Q10 measured by change in intellectual function (the Mini-Mental State Examination \[MMSE\]) and Activities of Daily Living (ADL) scores. MMSE: 30 point scale with 5 subsections (Total Score Range: minimum score = 0, maximum = 30): Orientation (max=10 pts, min=0 pts), Registration (max=3 pts, min=0 pts), Attention & Calculation (max=5pts, min=0 pts), Recall (max=3 pts, min= 0 pts), Language & Praxis (max=9 pts, min=0 pts). Lower scores indicate greater impairment. Scores \<24 are considered abnormal. Activities of Daily Living scores: The UPDRS part II is used to assess subject's degree in which they can perform their Activities of Daily Living. Part II of the UPDRS rates subjects on a 0-4 scale, with 0 being Normal and 4 being Severe Impairment, for each respective item. For each item, the reported score is divided by the maximum total score for that item and multiplied by 100 to give a 0-100 scale range. Higher scores indicate greater impairment. |
| Quality of Life Questionnaires: Parkinson's Disease Questionnaire - 39 [PDQ-39], Short Form-36 [SF-36] | 12 months | Parkinson's Disease Questionnaire - 39 (PDQ-39): 39 Items. 8 subscales: mobility, activities of daily living, emotional well-being, stigma, social support , cognitions, communication, bodily discomfort. Subjects indicate never (0 pts), occasionally (1 pts), sometimes (2pts), often (3pts), or always/cannot do at all (4pts) for each item in each section. The sum of scores of each item in the dimension divided by the maximum possible score of all the items in the dimension, multiplied by 100. Range of scores for each dimension: 0-100. The sum of dimension total scores are divided by 8 to calculate the Parkinson's Disease Severity Index. Range of total score for the Parkinsons' Disease Severity Index: 0-100. Lower scores indicate better quality of life. Short Form-36 (SF-36): 36 items 8 subscales: (2) General Health Sections, Limitations of Activities, Physical Health Problems, Emotional Health Problems, Pain, (2) Social Activities sections, and Energy and Emotions. Each qu |
| Safety Profile of Coenzyme Q10 | 12 months | The safety Profile of Coenzyme Q10 as determined by the analysis of the frequency and severity of the adverse event data, changes in the vital signs, electrocardiograms and clinical laboratory values, recorded over the course of the trial. |
| Tolerability of Coenzyme Q10 | 12 months | 1. The tolerability of Coenzyme Q10 as determined by the number of subjects who complete the study on their original treatment assignment, and 2. The tolerability of Coenzyme Q10 as determined by the number of subjects completing the study. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Coenzyme Q10 (2400mg/Day) Patients randomized into this arm received 2400mg of coenzyme Q10 per day (300mg wafers eight times per day). Each wafer also contained 300IU of vitamin E. Patients in this group were to take this dose for 12 months. | 32 |
| Placebo Patients randomized into this arm received placebo wafers. These wafers looked identical to the Coenzyme Q10 wafers. Patients took 8 placebo wafers per day. | 29 |
| Total | 61 |
Baseline characteristics
| Characteristic | Coenzyme Q10 (2400mg/Day) | Placebo | Total |
|---|---|---|---|
| Age, Customized Age | 66.3 years | 66.9 years | 66.6 years |
| Sex: Female, Male Female | 8 Participants | 16 Participants | 24 Participants |
| Sex: Female, Male Male | 24 Participants | 13 Participants | 37 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 11 / 32 | 8 / 29 |
| serious Total, serious adverse events | 5 / 32 | 8 / 29 |
Outcome results
Efficacy of Coenzyme Q10 (Unified Parkinson's Disease Rating Scale [UPDRS] AND Progressive Supranuclear Palsy Rating Scale [PSPRS])
Unified Parkinson's Disease Rating Scale \[UPDRS\]: Higher scores indicate a higher degree of impairment The total score is calculated by summing the subscores. Total scores range from 0 (normal) to 166 (severely impaired) Part I \[Mentation, Behavior, & Mood\] Scale: 0-16 Part II \[Activities of Daily Living, Both ON & OFF\] Scale:0-52 Part III \[Motor Examination\] Scale: 0-56 Part IV \[Complications of Therapy\] Scale: 0-34 Part V \[Modified Hoehn & Yahr Staging\] Scale: 0-8 Part VI \[Schwab & England Activities of Daily Living Scale\] Scale: 0 - 100% Progressive Supranuclear Palsy Rating Scale \[PSPRS\] - Total Scale ranges from 0 (normal) to 128 (severely impaired) Section 1 \[History\] Scale: 0-31 Section 2 \[Mentation\] Scale: 0-20 Section 3 \[Bulbar\] Scale: 0-10 Section 4 \[Ocular Motor\] Scale: 0-20 Section 5 \[Limb Motor\] Scale: 0-22 Section 6 \[Gait and midline\] Scale: 0-25
Time frame: 12 months
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Coenzyme Q10 (2400mg/Day) | Efficacy of Coenzyme Q10 (Unified Parkinson's Disease Rating Scale [UPDRS] AND Progressive Supranuclear Palsy Rating Scale [PSPRS]) | Change in UPDRS scores | 5.9 units on a scale | Standard Deviation 10 |
| Coenzyme Q10 (2400mg/Day) | Efficacy of Coenzyme Q10 (Unified Parkinson's Disease Rating Scale [UPDRS] AND Progressive Supranuclear Palsy Rating Scale [PSPRS]) | Change in PSPRS scores | 11.5 units on a scale | Standard Deviation 11.1 |
| Placebo | Efficacy of Coenzyme Q10 (Unified Parkinson's Disease Rating Scale [UPDRS] AND Progressive Supranuclear Palsy Rating Scale [PSPRS]) | Change in UPDRS scores | 11.8 units on a scale | Standard Deviation 8.6 |
| Placebo | Efficacy of Coenzyme Q10 (Unified Parkinson's Disease Rating Scale [UPDRS] AND Progressive Supranuclear Palsy Rating Scale [PSPRS]) | Change in PSPRS scores | 12.8 units on a scale | Standard Deviation 9.1 |
Efficacy of Coenzyme Q10 (Mini-Mental State Examination [MMSE] and Activities of Daily Living [ADL] Scales)
Efficacy of Coenzyme Q10 measured by change in intellectual function (the Mini-Mental State Examination \[MMSE\]) and Activities of Daily Living (ADL) scores. MMSE: 30 point scale with 5 subsections (Total Score Range: minimum score = 0, maximum = 30): Orientation (max=10 pts, min=0 pts), Registration (max=3 pts, min=0 pts), Attention & Calculation (max=5pts, min=0 pts), Recall (max=3 pts, min= 0 pts), Language & Praxis (max=9 pts, min=0 pts). Lower scores indicate greater impairment. Scores \<24 are considered abnormal. Activities of Daily Living scores: The UPDRS part II is used to assess subject's degree in which they can perform their Activities of Daily Living. Part II of the UPDRS rates subjects on a 0-4 scale, with 0 being Normal and 4 being Severe Impairment, for each respective item. For each item, the reported score is divided by the maximum total score for that item and multiplied by 100 to give a 0-100 scale range. Higher scores indicate greater impairment.
Time frame: 12 months
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Coenzyme Q10 (2400mg/Day) | Efficacy of Coenzyme Q10 (Mini-Mental State Examination [MMSE] and Activities of Daily Living [ADL] Scales) | Change in ADL scores | -13.5 units on a scale | Standard Deviation 20.6 |
| Coenzyme Q10 (2400mg/Day) | Efficacy of Coenzyme Q10 (Mini-Mental State Examination [MMSE] and Activities of Daily Living [ADL] Scales) | Change in MMSE scores | -1.3 units on a scale | Standard Deviation 3.4 |
| Placebo | Efficacy of Coenzyme Q10 (Mini-Mental State Examination [MMSE] and Activities of Daily Living [ADL] Scales) | Change in ADL scores | -18.4 units on a scale | Standard Deviation 15.9 |
| Placebo | Efficacy of Coenzyme Q10 (Mini-Mental State Examination [MMSE] and Activities of Daily Living [ADL] Scales) | Change in MMSE scores | -1.3 units on a scale | Standard Deviation 1.8 |
Quality of Life Questionnaires: Parkinson's Disease Questionnaire - 39 [PDQ-39], Short Form-36 [SF-36]
Parkinson's Disease Questionnaire - 39 (PDQ-39): 39 Items. 8 subscales: mobility, activities of daily living, emotional well-being, stigma, social support , cognitions, communication, bodily discomfort. Subjects indicate never (0 pts), occasionally (1 pts), sometimes (2pts), often (3pts), or always/cannot do at all (4pts) for each item in each section. The sum of scores of each item in the dimension divided by the maximum possible score of all the items in the dimension, multiplied by 100. Range of scores for each dimension: 0-100. The sum of dimension total scores are divided by 8 to calculate the Parkinson's Disease Severity Index. Range of total score for the Parkinsons' Disease Severity Index: 0-100. Lower scores indicate better quality of life. Short Form-36 (SF-36): 36 items 8 subscales: (2) General Health Sections, Limitations of Activities, Physical Health Problems, Emotional Health Problems, Pain, (2) Social Activities sections, and Energy and Emotions. Each qu
Time frame: 12 months
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Coenzyme Q10 (2400mg/Day) | Quality of Life Questionnaires: Parkinson's Disease Questionnaire - 39 [PDQ-39], Short Form-36 [SF-36] | Change in Parkinson's Disease Questionnaire scores | 3.4 units on a scale | Standard Deviation 13.9 |
| Coenzyme Q10 (2400mg/Day) | Quality of Life Questionnaires: Parkinson's Disease Questionnaire - 39 [PDQ-39], Short Form-36 [SF-36] | Change in Short Form-36 Scores | -2.1 units on a scale | Standard Deviation 19.1 |
| Placebo | Quality of Life Questionnaires: Parkinson's Disease Questionnaire - 39 [PDQ-39], Short Form-36 [SF-36] | Change in Parkinson's Disease Questionnaire scores | 7.5 units on a scale | Standard Deviation 12.6 |
| Placebo | Quality of Life Questionnaires: Parkinson's Disease Questionnaire - 39 [PDQ-39], Short Form-36 [SF-36] | Change in Short Form-36 Scores | -7.2 units on a scale | Standard Deviation 15.4 |
Safety Profile of Coenzyme Q10
The safety Profile of Coenzyme Q10 as determined by the analysis of the frequency and severity of the adverse event data, changes in the vital signs, electrocardiograms and clinical laboratory values, recorded over the course of the trial.
Time frame: 12 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Coenzyme Q10 (2400mg/Day) | Safety Profile of Coenzyme Q10 | Adverse Events | 27 Adverse Events |
| Coenzyme Q10 (2400mg/Day) | Safety Profile of Coenzyme Q10 | Serious Adverse Events | 5 Adverse Events |
| Placebo | Safety Profile of Coenzyme Q10 | Adverse Events | 38 Adverse Events |
| Placebo | Safety Profile of Coenzyme Q10 | Serious Adverse Events | 8 Adverse Events |
Tolerability of Coenzyme Q10
1. The tolerability of Coenzyme Q10 as determined by the number of subjects who complete the study on their original treatment assignment, and 2. The tolerability of Coenzyme Q10 as determined by the number of subjects completing the study.
Time frame: 12 months
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Coenzyme Q10 (2400mg/Day) | Tolerability of Coenzyme Q10 | 20 Participants |
| Placebo | Tolerability of Coenzyme Q10 | 16 Participants |