Trial Outcomes & Findings for Valproic Acid in Subjects With Intact Cognition - Proof of Concept Study (NCT NCT01729598)
NCT ID: NCT01729598
Last Updated: 2019-10-09
Results Overview
Safety assessments will be based on medical review of adverse event reports and the results of vital sign measurements, physical examinations, and clinical laboratory tests throughout the study. The incidence of observed toxicities and adverse events will be tabulated, the frequencies compared in participants who receive active medication and those who receive placebo, and reviewed for potential significance and clinical importance.
Recruitment status
COMPLETED
Study phase
EARLY_PHASE1
Target enrollment
14 participants
Primary outcome timeframe
Day 35
Results posted on
2019-10-09
Participant Flow
Participant milestones
| Measure |
Valproic Acid
Valproic acid 250 mg or 500mg by mouth twice daily.
Valproic Acid: generic valproic acid tablets packaged in placebo-matched capsules.
|
Placebo
Placebo capsule by mouth twice daily.
Placebo: Placebo capsule without active study medication in identical capsules as experimental medicine.
|
|---|---|---|
|
Overall Study
STARTED
|
10
|
4
|
|
Overall Study
COMPLETED
|
10
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Valproic Acid in Subjects With Intact Cognition - Proof of Concept Study
Baseline characteristics by cohort
| Measure |
Valproic Acid
n=10 Participants
Valproic acid 250 mg or 500mg by mouth twice daily.
Valproic Acid: generic valproic acid tablets packaged in placebo-matched capsules.
|
Placebo
n=4 Participants
Placebo capsule by mouth twice daily.
Placebo: Placebo capsule without active study medication in identical capsules as experimental medicine.
|
Total
n=14 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
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
10 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
|
Age, Continuous
|
76 years
STANDARD_DEVIATION 7.3 • n=5 Participants
|
81 years
STANDARD_DEVIATION 12.2 • n=7 Participants
|
78 years
STANDARD_DEVIATION 8.6 • n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
10 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 35Safety assessments will be based on medical review of adverse event reports and the results of vital sign measurements, physical examinations, and clinical laboratory tests throughout the study. The incidence of observed toxicities and adverse events will be tabulated, the frequencies compared in participants who receive active medication and those who receive placebo, and reviewed for potential significance and clinical importance.
Outcome measures
| Measure |
Valproic Acid
n=10 Participants
Valproic acid 250 mg or 500mg by mouth twice daily.
Valproic Acid: generic valproic acid tablets packaged in placebo-matched capsules.
|
Placebo
n=4 Participants
Placebo capsule by mouth twice daily.
Placebo: Placebo capsule without active study medication in identical capsules as experimental medicine.
|
|---|---|---|
|
Frequency of Adverse Events Over the Duration of the Study by Study Arm
|
8 Participants
|
1 Participants
|
PRIMARY outcome
Timeframe: Baseline and day 28Change in cerebrospinal fluid amyloid-beta 1-42 levels in pg/ml from baseline to end of treatment (day 28)
Outcome measures
| Measure |
Valproic Acid
n=10 Participants
Valproic acid 250 mg or 500mg by mouth twice daily.
Valproic Acid: generic valproic acid tablets packaged in placebo-matched capsules.
|
Placebo
n=4 Participants
Placebo capsule by mouth twice daily.
Placebo: Placebo capsule without active study medication in identical capsules as experimental medicine.
|
|---|---|---|
|
Change in Cerebrospinal Fluid Amyloid Levels (pg/ml) Over 28 Day Intervention Period by Study Arm
|
57.1 pg/ml
Standard Deviation 28.4
|
44.7 pg/ml
Standard Deviation 24.9
|
SECONDARY outcome
Timeframe: Baseline and day 28Change in cerebrospinal fluid p181-tau levels (pg/ml) from baseline to end of treatment (Day 28)
Outcome measures
| Measure |
Valproic Acid
n=10 Participants
Valproic acid 250 mg or 500mg by mouth twice daily.
Valproic Acid: generic valproic acid tablets packaged in placebo-matched capsules.
|
Placebo
n=4 Participants
Placebo capsule by mouth twice daily.
Placebo: Placebo capsule without active study medication in identical capsules as experimental medicine.
|
|---|---|---|
|
Change in Cerebrospinal Fluid P-tau Levels (pg/ml)
|
-5.7 pg/ml
Standard Deviation 11.9
|
11.0 pg/ml
Standard Deviation 25.7
|
SECONDARY outcome
Timeframe: Baseline and day 28Change in Free \& Cued Selective Reminding Test- delayed free recall from baseline to end of treatment (Day 28)
Outcome measures
| Measure |
Valproic Acid
n=10 Participants
Valproic acid 250 mg or 500mg by mouth twice daily.
Valproic Acid: generic valproic acid tablets packaged in placebo-matched capsules.
|
Placebo
n=4 Participants
Placebo capsule by mouth twice daily.
Placebo: Placebo capsule without active study medication in identical capsules as experimental medicine.
|
|---|---|---|
|
Change in Free & Cued Selective Reminding Test- Free Recall (Number of Items Correct)
|
1.4 number of items recalled
Standard Deviation 3.5
|
-4.5 number of items recalled
Standard Deviation 3.4
|
SECONDARY outcome
Timeframe: Baseline and day 28Change in cerebrospinal fluid clusterin levels (pg/ml) from baseline to end of treatment (Day 28)
Outcome measures
| Measure |
Valproic Acid
n=10 Participants
Valproic acid 250 mg or 500mg by mouth twice daily.
Valproic Acid: generic valproic acid tablets packaged in placebo-matched capsules.
|
Placebo
n=4 Participants
Placebo capsule by mouth twice daily.
Placebo: Placebo capsule without active study medication in identical capsules as experimental medicine.
|
|---|---|---|
|
Change in Cerebrospinal Fluid Clusterin Levels (pg/ml)
|
2610 pg/ml
Standard Deviation 4373
|
201 pg/ml
Standard Deviation 871
|
Adverse Events
Valproic Acid
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Valproic Acid
n=10 participants at risk
Valproic acid 250 mg or 500mg by mouth twice daily.
Valproic Acid: generic valproic acid tablets packaged in placebo-matched capsules.
|
Placebo
n=4 participants at risk
Placebo capsule by mouth twice daily.
Placebo: Placebo capsule without active study medication in identical capsules as experimental medicine.
|
|---|---|---|
|
Endocrine disorders
hypoglycemia
|
10.0%
1/10 • Number of events 1 • 35 days
|
0.00%
0/4 • 35 days
|
|
Nervous system disorders
fatigue
|
30.0%
3/10 • Number of events 4 • 35 days
|
25.0%
1/4 • Number of events 1 • 35 days
|
|
Nervous system disorders
Headache
|
40.0%
4/10 • Number of events 4 • 35 days
|
0.00%
0/4 • 35 days
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place