Effects of Coenzyme Q10 in Progressive Supranuclear Palsy (PSP)

Study Overview
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Brief Summary
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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
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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

Official Title
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Effects of Coenzyme Q10 in Progressive Supranuclear Palsy (PSP): A Multicenter, Randomized, Placebo-controlled, Double Blind Study

Conditions
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Progressive Supranuclear Palsy

Intervention / Treatment
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* Dietary Supplement: Coenzyme Q10
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 30 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Multicenter, Double-blind, Randomized, Placebo-Controlled Clinical Trial to examine tolerability, safety, and efficacy of CoenzymeQ10
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: CoQ10<br>Half of the enrolled patients will be randomized into the the CoQ10 arm and will receive a dosage of 2400mg/day of Coenzyme Q10 | Dietary Supplement: Coenzyme Q10<br>* active drug<br>* Other names: CoQ10;|
| Placebo Comparator: Placebo<br>Half of the enrolled patients will be randomized into the the Placebo arm and will receive a matching dose of placebo that resembles the 2400mg/day dose of the CoQ10 arm. | Other: Placebo<br>* Placebo<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 12 months | 
| 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 | 12 months | 
| 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. | 12 months | 
| Tolerability of Coenzyme Q10 | The tolerability of Coenzyme Q10 as determined by the number of subjects who complete the study on their original treatment assignment, and The tolerability of Coenzyme Q10 as determined by the number of subjects completing the study. | 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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CoQ10, Progressive Supranuclear Palsy, Coenzyme Q10