Trial Outcomes & Findings for A Pilot Study of Oxaloacetate in Subjects With Treated PD (NCT NCT01741701)

NCT ID: NCT01741701

Last Updated: 2016-03-17

Results Overview

The UPDRS has 3 subscales including Mentation (4 questions based on patient report with answers on a scale of 0-4, with a total of 16 points), Activities of Daily Living (13 questions based on patient report with answers on a scale of 0-4, with a total of 52 points) and Motor (27 questions based on clinician assessment on a scale of 0-4, with a total of 108 points). The total scores represents the sum of each of these sections for a total of 176 points with a higher score representing greater dysfunction.

Recruitment status

COMPLETED

Study phase

PHASE2/PHASE3

Target enrollment

33 participants

Primary outcome timeframe

4 months

Results posted on

2016-03-17

Participant Flow

Participant milestones

Participant milestones
Measure
Oxaloacetate (OAA)
active capsule containing 100 mg OAA and 100 mg ascorbate, taken daily Oxaloacetate (OAA)
Placebo
placebo capsules that contain only 100 mg ascorbate, taken daily Placebo
Overall Study
STARTED
18
15
Overall Study
COMPLETED
13
13
Overall Study
NOT COMPLETED
5
2

Reasons for withdrawal

Reasons for withdrawal
Measure
Oxaloacetate (OAA)
active capsule containing 100 mg OAA and 100 mg ascorbate, taken daily Oxaloacetate (OAA)
Placebo
placebo capsules that contain only 100 mg ascorbate, taken daily Placebo
Overall Study
Adverse Event
5
1
Overall Study
Withdrawal by Subject
0
1

Baseline Characteristics

A Pilot Study of Oxaloacetate in Subjects With Treated PD

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Oxaloacetate (OAA)
n=18 Participants
active capsule containing 100 mg OAA and 100 mg ascorbate, taken daily Oxaloacetate (OAA)
Placebo
n=15 Participants
placebo capsules that contain only 100 mg ascorbate, taken daily Placebo
Total
n=33 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
11 Participants
n=5 Participants
10 Participants
n=7 Participants
21 Participants
n=5 Participants
Age, Categorical
>=65 years
7 Participants
n=5 Participants
5 Participants
n=7 Participants
12 Participants
n=5 Participants
Age, Continuous
62.4 years
STANDARD_DEVIATION 11.4 • n=5 Participants
64.6 years
STANDARD_DEVIATION 9.4 • n=7 Participants
63.4 years
STANDARD_DEVIATION 10.5 • n=5 Participants
Sex: Female, Male
Female
3 Participants
n=5 Participants
1 Participants
n=7 Participants
4 Participants
n=5 Participants
Sex: Female, Male
Male
15 Participants
n=5 Participants
14 Participants
n=7 Participants
29 Participants
n=5 Participants
Region of Enrollment
United States
18 participants
n=5 Participants
15 participants
n=7 Participants
33 participants
n=5 Participants
PD Duration
2.9 years
STANDARD_DEVIATION 1.5 • n=5 Participants
3.1 years
STANDARD_DEVIATION 1.7 • n=7 Participants
3.0 years
STANDARD_DEVIATION 1.6 • n=5 Participants

PRIMARY outcome

Timeframe: 4 months

Population: Only subjects that completed the study were included in the efficacy analyses, those that withdrew were not included in the efficacy analysis.

The UPDRS has 3 subscales including Mentation (4 questions based on patient report with answers on a scale of 0-4, with a total of 16 points), Activities of Daily Living (13 questions based on patient report with answers on a scale of 0-4, with a total of 52 points) and Motor (27 questions based on clinician assessment on a scale of 0-4, with a total of 108 points). The total scores represents the sum of each of these sections for a total of 176 points with a higher score representing greater dysfunction.

Outcome measures

Outcome measures
Measure
Oxaloacetate (OAA)
n=13 Participants
active capsule containing 100 mg OAA and 100 mg ascorbate, taken daily Oxaloacetate (OAA)
Placebo
n=13 Participants
placebo capsules that contain only 100 mg ascorbate, taken daily Placebo
Unified Parkinson's Disease Rating Scale (UPDRS) Total Score
UPDRS Total Score at Baseline
41.1 units on a scale
Standard Deviation 6.7
42.9 units on a scale
Standard Deviation 10.8
Unified Parkinson's Disease Rating Scale (UPDRS) Total Score
UPDRS Total Score at 4 months
34.9 units on a scale
Standard Deviation 10.4
34.4 units on a scale
Standard Deviation 12.4

SECONDARY outcome

Timeframe: 4 months

Population: Only subjects that completed the study were included in the efficacy analyses, those that withdrew were not included in the efficacy analysis.

The UPDRS has 3 subscales including Mentation (4 questions based on patient report with answers on a scale of 0-4, with a total of 16 points), Activities of Daily Living (13 questions based on patient report with answers on a scale of 0-4, with a total of 52 points) and Motor (27 questions based on clinician assessment on a scale of 0-4, with a total of 108 points). This measure examined the ADL + Motor subscales which have a total of 160 points with a higher score representing greater dysfunction.

Outcome measures

Outcome measures
Measure
Oxaloacetate (OAA)
n=13 Participants
active capsule containing 100 mg OAA and 100 mg ascorbate, taken daily Oxaloacetate (OAA)
Placebo
n=13 Participants
placebo capsules that contain only 100 mg ascorbate, taken daily Placebo
Unified Parkinson's Disease Rating Scale (UPDRS) ADL + Motor Score
UPDRS ADL + Motor at Baseline
39.4 units on a scale
Standard Deviation 6.9
42.2 units on a scale
Standard Deviation 10.7
Unified Parkinson's Disease Rating Scale (UPDRS) ADL + Motor Score
UPDRS ADL + Motor at 4 months
33.4 units on a scale
Standard Deviation 10.2
33.9 units on a scale
Standard Deviation 11.7

SECONDARY outcome

Timeframe: 4 months

Population: Only subjects that completed the study were included in the efficacy analyses, those that withdrew were not included in the efficacy analysis.

The PDQ-39 is a measure of quality of life in Parkinson's disease patients. It has 39 questions each with a response from 0-4 for a total of 156 points. The total score is calculated as a percentage so the scores of the 39 items are added and divided by 156 and multiplied by 100. The higher the score the worse quality of life.

Outcome measures

Outcome measures
Measure
Oxaloacetate (OAA)
n=13 Participants
active capsule containing 100 mg OAA and 100 mg ascorbate, taken daily Oxaloacetate (OAA)
Placebo
n=13 Participants
placebo capsules that contain only 100 mg ascorbate, taken daily Placebo
Parkinson's Disease Questionnaire - 39 (PDQ-39)
PDQ-39 Total Score at Baseline
13.0 percentage of total possible score
Standard Deviation 8.9
10.1 percentage of total possible score
Standard Deviation 7.8
Parkinson's Disease Questionnaire - 39 (PDQ-39)
PDQ-39 total score at 4 months
14.4 percentage of total possible score
Standard Deviation 11.2
11.3 percentage of total possible score
Standard Deviation 7.4

SECONDARY outcome

Timeframe: 4 months

Population: Only subjects that completed the study were included in the efficacy analyses, those that withdrew were not included in the efficacy analysis.

The MoCA is an assessment of cognitive function. The total possible score ranges from 0 to 30 points with a lower score representing greater cognitive impairment.

Outcome measures

Outcome measures
Measure
Oxaloacetate (OAA)
n=13 Participants
active capsule containing 100 mg OAA and 100 mg ascorbate, taken daily Oxaloacetate (OAA)
Placebo
n=13 Participants
placebo capsules that contain only 100 mg ascorbate, taken daily Placebo
Montreal Cognitive Assessment (MoCA)
MoCA at Baseline
27.4 units on a scale
Standard Deviation 2.0
27.2 units on a scale
Standard Deviation 2.4
Montreal Cognitive Assessment (MoCA)
MoCA at 4 months
28.1 units on a scale
Standard Deviation 1.6
27.6 units on a scale
Standard Deviation 2.4

SECONDARY outcome

Timeframe: 4 months

Population: Only subjects that completed the study were included in the efficacy analyses, those that withdrew were not included in the efficacy analysis.

The GDS is a measure of depression. The scale has 30 yes/no questions. Each question has a maximum score of 1 and a total possible score ranging from 0 to 30. The higher the score the greater the depression.

Outcome measures

Outcome measures
Measure
Oxaloacetate (OAA)
n=13 Participants
active capsule containing 100 mg OAA and 100 mg ascorbate, taken daily Oxaloacetate (OAA)
Placebo
n=13 Participants
placebo capsules that contain only 100 mg ascorbate, taken daily Placebo
Geriatric Depression Scale (GDS)
GDS at Baseline
5.3 units on a scale
Standard Deviation 4.4
3.3 units on a scale
Standard Deviation 3.1
Geriatric Depression Scale (GDS)
GDS at 4 months
5.8 units on a scale
Standard Deviation 5.4
3.8 units on a scale
Standard Deviation 3.7

SECONDARY outcome

Timeframe: 4 months

Population: Only subjects that completed the study were included in the efficacy analyses, those that withdrew were not included in the efficacy analysis.

The NMSQuest is a 30 item questionnaire with 30 yes/no questions. There is a total of 30 points with each "yes" score representing 1 point and therefore the higher the score the greater number of nonmotor symptoms present. The score can range from 0 to 30.

Outcome measures

Outcome measures
Measure
Oxaloacetate (OAA)
n=13 Participants
active capsule containing 100 mg OAA and 100 mg ascorbate, taken daily Oxaloacetate (OAA)
Placebo
n=13 Participants
placebo capsules that contain only 100 mg ascorbate, taken daily Placebo
NonMotor Symptom Questionnaire (NMSQuest)
NMSQuest at 4 months
7.9 units on a scale
Standard Deviation 6.3
6.9 units on a scale
Standard Deviation 4.6
NonMotor Symptom Questionnaire (NMSQuest)
NMSQuest at Baseline
7.1 units on a scale
Standard Deviation 4.4
6.2 units on a scale
Standard Deviation 4.4

Adverse Events

Oxaloacetate (OAA)

Serious events: 0 serious events
Other events: 11 other events
Deaths: 0 deaths

Placebo

Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Oxaloacetate (OAA)
n=18 participants at risk
active capsule containing 100 mg OAA and 100 mg ascorbate, taken daily Oxaloacetate (OAA)
Placebo
n=15 participants at risk
placebo capsules that contain only 100 mg ascorbate, taken daily Placebo
General disorders
insomnia/stimulant
16.7%
3/18 • Number of events 3 • Adverse effects for each subject were collected throughout the 4 month study period
6.7%
1/15 • Number of events 1 • Adverse effects for each subject were collected throughout the 4 month study period
Nervous system disorders
Worsening of Parkinson's disease
38.9%
7/18 • Number of events 7 • Adverse effects for each subject were collected throughout the 4 month study period
6.7%
1/15 • Number of events 1 • Adverse effects for each subject were collected throughout the 4 month study period
Gastrointestinal disorders
GI upset
22.2%
4/18 • Number of events 4 • Adverse effects for each subject were collected throughout the 4 month study period
0.00%
0/15 • Adverse effects for each subject were collected throughout the 4 month study period
Nervous system disorders
Fatigue
11.1%
2/18 • Number of events 2 • Adverse effects for each subject were collected throughout the 4 month study period
0.00%
0/15 • Adverse effects for each subject were collected throughout the 4 month study period
General disorders
dizziness
5.6%
1/18 • Number of events 1 • Adverse effects for each subject were collected throughout the 4 month study period
0.00%
0/15 • Adverse effects for each subject were collected throughout the 4 month study period
Psychiatric disorders
Cognitive worsening
5.6%
1/18 • Number of events 1 • Adverse effects for each subject were collected throughout the 4 month study period
6.7%
1/15 • Number of events 1 • Adverse effects for each subject were collected throughout the 4 month study period
Gastrointestinal disorders
Increased urination
0.00%
0/18 • Adverse effects for each subject were collected throughout the 4 month study period
6.7%
1/15 • Number of events 1 • Adverse effects for each subject were collected throughout the 4 month study period

Additional Information

Dr. Rajesh Pahwa

University of Kansas Medical Center

Phone: 913-588-6782

Results disclosure agreements

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