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VPA
CompletedPhase EARLY_1Results postedValproic Acid in Subjects With Intact Cognition - Proof of Concept Study
Safety And Target Engagement Of Clu1 By Valproic Acid In Subjects With Intact Cognition: Proof Of Concept For The Development Of A Prevention Trial For Alzheimer's Disease
Lead sponsor
Asset
Valproic Acid
Listed sites
1
Recruiting sites
-
Enrollment
14
actual
Study population
Alzheimer’s disease
Key I/E criterion
•Age 65-90
Primary endpoints
•Adverse Events Over the Duration of the Study by Study Arm•Cerebrospinal Fluid Amyloid Levels (pg/ml)
Footprint
Where this trial recruits
Site locations as reported to ClinicalTrials.gov. Site count is not enrollment count; per-site enrollment is not available from source.
Identifiers
Registered as
Timeline
Milestones
Assets
Drug assets
Study populations
Who this study enrolls
Eligibility
Who can enroll
Inclusion criteria
1. Men or women aged 65-90, inclusive.
2. English-speaking, to ensure compliance with cognitive testing and study visit procedures.
3. Female participants must not be pregnant or of childbearing potential, i.e. either surgically sterile or postmenopausal for > 1 year.
4. Stable medical condition for three months prior to screening visit, with no clinically significant abnormalities of hepatic, renal, and hematologic function defined as follows:
5. Stable medications for 4 weeks prior to screening visit.
6. Able to ingest oral medications.
7. No history of adverse drug reactions to VPA or similar agents.
8. Physically acceptable for this study as confirmed by medical history, physical exam, neurological exam and clinical tests in the opinion of the study physician.
9. Not demented by Hachinski Ischemic Index (< 4)
Exclusion criteria
1. Significant neurologic disease such as Parkinson's disease, stroke, brain tumor, multiple sclerosis or seizure disorder.
2. Major depression in past 12 months (DSM-IV criteria), major mental illness such as schizophrenia, or recent (in past 12 months) alcohol or substance abuse by history.
3. History of invasive cancer within the past two years (excluding non-melanoma skin cancer).
4. Contra-indications to lumbar puncture (bleeding disorder, platelet count < 100,000, anticoagulant treatment, major structural abnormality or sepsis in the area of the lumbosacral spine that would make spinal fluid collection technically difficult).
5. Clinically significant MRI abnormalities that contraindicate lumber or suggest central nervous system disease processes that could influence study outcomes in the opinion of the PI.
6. Use of any investigational agents within 30 days prior to screening.
7. Major surgery within eight weeks prior to the Baseline Visit.
8. Severe unstable medical illnesses, including uncontrolled cardiac conditions or heart failure (New York Heart Association Class III or IV) .
9. Antiretroviral therapy for human immunodeficiency virus (HIV).
10. Residence in a skilled nursing facility.
11. Blindness, deafness, language difficulties or any other disability which may prevent the participant from participating or cooperating in the protocol.
Excluded Medications
1. Experimental drugs
2. Lamictal
3. Tricyclic antidepressants (amitriptyline/nortryptiline)
4. Carbamazepine/ oxcarbazepine
5. Benzodiazepines
6. Phenobarbital
7. Phenytoin
8. Tolbutamide
9. Topiramate
10. Warfarin
11. Zidovudine
Endpoints (10)
What's being measured
Protocol endpoints and posted registry outcome measures, grouped into outcome categories. Composite endpoints show their component event types. Standard codes (LOINC, SNOMED CT) are shown where available.
Coverage by outcome category
Memory
2 endpointsChange in Free & Cued Selective Reminding Test- Free Recall (Number of Items Correct)
Time frame:Baseline and day 28
change from baseline, improvement
Change in Free & Cued Selective Reminding Test- Free Recall (Number of Items Correct)
Time frame:Baseline and day 28
change from baseline, improvement
Posted result
| Group | Value (mean), number of items recalled | Standard deviation |
|---|---|---|
| Valproic Acidn=10 Participants | 1.4 | 3.5 |
| Placebon=4 Participants | -4.5 | 3.4 |
Amyloid biomarkers
2 endpointsChange in Cerebrospinal Fluid Amyloid Levels (pg/ml) Over 28 Day Intervention Period by Study Arm
Time frame:Baseline and day 28
change from baseline, improvement
Change in Cerebrospinal Fluid Amyloid Levels (pg/ml) Over 28 Day Intervention Period by Study Arm
Time frame:Baseline and day 28
change from baseline, improvement
Posted result
| Group | Value (mean), pg/ml | Standard deviation |
|---|---|---|
| Valproic Acidn=10 Participants | 57.1 | 28.4 |
| Placebon=4 Participants | 44.7 | 24.9 |
Tau biomarkers
2 endpointsChange in Cerebrospinal Fluid P-tau Levels (pg/ml)
Time frame:Baseline and day 28
change from baseline, improvement
Change in Cerebrospinal Fluid P-tau Levels (pg/ml)
Time frame:Baseline and day 28
change from baseline, improvement
Posted result
| Group | Value (mean), pg/ml | Standard deviation |
|---|---|---|
| Valproic Acidn=10 Participants | -5.7 | 11.9 |
| Placebon=4 Participants | 11.0 | 25.7 |
Fluid / digital biomarkers
2 endpointsChange in Cerebrospinal Fluid Clusterin Levels (pg/ml)
Time frame:Baseline and day 28
change from baseline, improvement
Change in Cerebrospinal Fluid Clusterin Levels (pg/ml)
Time frame:Baseline and day 28
change from baseline, improvement
Posted result
| Group | Value (mean), pg/ml | Standard deviation |
|---|---|---|
| Valproic Acidn=10 Participants | 2610 | 4373 |
| Placebon=4 Participants | 201 | 871 |
Safety / tolerability / PK
2 endpointsFrequency of Adverse Events Over the Duration of the Study by Study Arm
Time frame:Day 35
event count, event
Frequency of Adverse Events Over the Duration of the Study by Study Arm
Time frame:Day 35
event count, event
Posted result
| Group | Value (count_of_participants), Participants | Reported bounds |
|---|---|---|
| Valproic Acidn=10 Participants | 8 | - |
| Placebon=4 Participants | 1 | - |
Provenance
Sources
Trial facts come from public ClinicalTrials.gov records. Endpoint categories are Delfa's classification of those records, not a ClinicalTrials.gov field. All figures reflect the July 21, 2026 snapshot.