Trial Outcomes & Findings for Effects of Coenzyme Q10 in Progressive Supranuclear Palsy (PSP) (NCT NCT00382824)

NCT ID: NCT00382824

Last Updated: 2019-08-01

Results Overview

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

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 61 participants
• Primary outcome timeframe: 12 months
• Results posted on: 2019-08-01

Participant Flow

Participant milestones

Measure	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 200IU of vitamin E for a total of 1200IU of vitamin E/day. Patients in this group were to take this dose for 12 months.	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. Each wafer also contained 200IU of vitamin E. Patients received 1200IU of viatmin E/day
Overall Study STARTED	32	29
Overall Study COMPLETED	20	16
Overall Study NOT COMPLETED	12	13

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Effects of Coenzyme Q10 in Progressive Supranuclear Palsy (PSP)

Baseline characteristics by cohort

Measure	Coenzyme Q10 (2400mg/Day) n=32 Participants 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.	Placebo n=29 Participants Patients randomized into this arm received placebo wafers. These wafers looked identical to the Coenzyme Q10 wafers. Patients took 8 placebo wafers per day.	Total n=61 Participants Total of all reporting groups
Age, Customized Age	66.3 years n=5 Participants	66.9 years n=7 Participants	66.6 years n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants	16 Participants n=7 Participants	24 Participants n=5 Participants
Sex: Female, Male Male	24 Participants n=5 Participants	13 Participants n=7 Participants	37 Participants n=5 Participants

PRIMARY outcome

Timeframe: 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

Outcome measures

Measure	Coenzyme Q10 (2400mg/Day) n=20 Participants Patients randomized into this arm received 2400mg of coenzyme Q10 per day (300mg wafers eight times per day). Each wafer also contained 200IU of vitamin E for a total of 1200IU of vitamin E/day. Patients in this group were to take this dose for 12 months.	Placebo n=16 Participants Patients randomized into this arm received placebo wafers. These wafers looked identical to the Coenzyme Q10 wafers. Patients took 8 placebo wafers per day. Each wafer also contained 200IU of vitamin E. Patients received 1200IU of viatmin E/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 10.0	11.8 units on a scale Standard Deviation 8.6
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	12.8 units on a scale Standard Deviation 9.1

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Coenzyme Q10 (2400mg/Day) n=20 Participants Patients randomized into this arm received 2400mg of coenzyme Q10 per day (300mg wafers eight times per day). Each wafer also contained 200IU of vitamin E for a total of 1200IU of vitamin E/day. Patients in this group were to take this dose for 12 months.	Placebo n=16 Participants Patients randomized into this arm received placebo wafers. These wafers looked identical to the Coenzyme Q10 wafers. Patients took 8 placebo wafers per day. Each wafer also contained 200IU of vitamin E. Patients received 1200IU of viatmin E/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	-18.4 units on a scale Standard Deviation 15.9
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	-1.3 units on a scale Standard Deviation 1.8

SECONDARY outcome

Timeframe: 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

Outcome measures

Measure	Coenzyme Q10 (2400mg/Day) n=20 Participants Patients randomized into this arm received 2400mg of coenzyme Q10 per day (300mg wafers eight times per day). Each wafer also contained 200IU of vitamin E for a total of 1200IU of vitamin E/day. Patients in this group were to take this dose for 12 months.	Placebo n=16 Participants Patients randomized into this arm received placebo wafers. These wafers looked identical to the Coenzyme Q10 wafers. Patients took 8 placebo wafers per day. Each wafer also contained 200IU of vitamin E. Patients received 1200IU of viatmin E/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	7.5 units on a scale Standard Deviation 12.6
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	-7.2 units on a scale Standard Deviation 15.4

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Coenzyme Q10 (2400mg/Day) n=32 Participants Patients randomized into this arm received 2400mg of coenzyme Q10 per day (300mg wafers eight times per day). Each wafer also contained 200IU of vitamin E for a total of 1200IU of vitamin E/day. Patients in this group were to take this dose for 12 months.	Placebo n=29 Participants Patients randomized into this arm received placebo wafers. These wafers looked identical to the Coenzyme Q10 wafers. Patients took 8 placebo wafers per day. Each wafer also contained 200IU of vitamin E. Patients received 1200IU of viatmin E/day
Safety Profile of Coenzyme Q10 Adverse Events	27 Adverse Events	38 Adverse Events
Safety Profile of Coenzyme Q10 Serious Adverse Events	5 Adverse Events	8 Adverse Events

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Coenzyme Q10 (2400mg/Day) n=32 Participants Patients randomized into this arm received 2400mg of coenzyme Q10 per day (300mg wafers eight times per day). Each wafer also contained 200IU of vitamin E for a total of 1200IU of vitamin E/day. Patients in this group were to take this dose for 12 months.	Placebo n=29 Participants Patients randomized into this arm received placebo wafers. These wafers looked identical to the Coenzyme Q10 wafers. Patients took 8 placebo wafers per day. Each wafer also contained 200IU of vitamin E. Patients received 1200IU of viatmin E/day
Tolerability of Coenzyme Q10	20 Participants	16 Participants

Adverse Events

Coenzyme Q10 (2400mg/Day)

Serious events: 5 serious events

Other events: 11 other events

Deaths: 0 deaths

Placebo

Serious events: 8 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Measure	Coenzyme Q10 (2400mg/Day) n=32 participants at risk Patients randomized into this arm received 2400mg of coenzyme Q10 per day (300mg wafers eight times per day). Each wafer also contained 200IU of vitamin E for a total of 1200IU of vitamin E/day. Patients in this group were to take this dose for 12 months.	Placebo n=29 participants at risk Patients randomized into this arm received placebo wafers. These wafers looked identical to the Coenzyme Q10 wafers. Patients took 8 placebo wafers per day. Each wafer also contained 200IU of vitamin E. Patients received 1200IU of viatmin E/day
Gastrointestinal disorders Diverticulitis	3.1% 1/32 • Number of events 1 • 12 months	0.00% 0/29 • 12 months
Musculoskeletal and connective tissue disorders Bone fracture	3.1% 1/32 • Number of events 1 • 12 months	13.8% 4/29 • Number of events 4 • 12 months
Infections and infestations Aspiration pneumonia	0.00% 0/32 • 12 months	3.4% 1/29 • Number of events 1 • 12 months
Renal and urinary disorders Benign postatic hyperplasia with obstruction	0.00% 0/32 • 12 months	3.4% 1/29 • Number of events 1 • 12 months
Surgical and medical procedures Stent placement	0.00% 0/32 • 12 months	3.4% 1/29 • Number of events 1 • 12 months
Infections and infestations C. difficile infection	3.1% 1/32 • Number of events 1 • 12 months	0.00% 0/29 • 12 months
Gastrointestinal disorders Bleeding ulcer	3.1% 1/32 • Number of events 1 • 12 months	0.00% 0/29 • 12 months
Renal and urinary disorders Ureteral calculus	0.00% 0/32 • 12 months	3.4% 1/29 • Number of events 1 • 12 months
General disorders Laceration to head	3.1% 1/32 • Number of events 1 • 12 months	0.00% 0/29 • 12 months

Other adverse events

Measure	Coenzyme Q10 (2400mg/Day) n=32 participants at risk Patients randomized into this arm received 2400mg of coenzyme Q10 per day (300mg wafers eight times per day). Each wafer also contained 200IU of vitamin E for a total of 1200IU of vitamin E/day. Patients in this group were to take this dose for 12 months.	Placebo n=29 participants at risk Patients randomized into this arm received placebo wafers. These wafers looked identical to the Coenzyme Q10 wafers. Patients took 8 placebo wafers per day. Each wafer also contained 200IU of vitamin E. Patients received 1200IU of viatmin E/day
Renal and urinary disorders Urinary tract infection	12.5% 4/32 • Number of events 4 • 12 months	3.4% 1/29 • Number of events 1 • 12 months
Gastrointestinal disorders Constipation	3.1% 1/32 • Number of events 1 • 12 months	10.3% 3/29 • Number of events 3 • 12 months
Eye disorders Cataract	6.2% 2/32 • Number of events 2 • 12 months	3.4% 1/29 • Number of events 1 • 12 months
General disorders Nausea	6.2% 2/32 • Number of events 2 • 12 months	3.4% 1/29 • Number of events 1 • 12 months
General disorders Diarrhea	6.2% 2/32 • Number of events 2 • 12 months	0.00% 0/29 • 12 months
Ear and labyrinth disorders Dizziness	0.00% 0/32 • 12 months	6.9% 2/29 • Number of events 2 • 12 months

Additional Information

Jordan Jara

Lahey Hospital & Medical Center

Phone: 7817442958

Email: jordan.jara@lahey.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place