Skip to main content
Delfa

← Trials/Trial dossier/NCT01729598

VPA

CompletedPhase EARLY_1Results posted

Valproic 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

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 ArmCerebrospinal 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

Org study ID12-0068-F6A
NCT IDNCT01729598

Timeline

Milestones

Study start2012-04 (month precision)
Study first posted2012-11-20estimated
Primary completion2014-10actual (month precision)
Study completion2014-10actual (month precision)
Last update posted2019-10-09actual
Results first posted2019-10-09actual

Assets

Drug assets

Study populations

Who this study enrolls

Alzheimer’s disease

Eligibility

Who can enroll

Minimum age65 Years
Maximum age90 Years
SexAll
Healthy volunteersAccepted

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:

-Platelets > 100,000
-Serum creatinine ≤ 1.6 mg/dL
-Liver function tests ≤ 1.5 upper limit of normal
-No clinically significant abnormalities of other laboratory studies (blood counts, chemistry panel, urinalysis) as determined by the study physician

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
Amyloid biomarkers
2
Tau biomarkers
2
Fluid / digital biomarkers
2
Safety / tolerability / PK
2

Memory

2 endpoints
Secondary/protocol endpoint

Change in Free & Cued Selective Reminding Test- Free Recall (Number of Items Correct)

Time frame:Baseline and day 28

change from baseline, improvement

Secondary/registry result

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

GroupValue (mean), number of items recalledStandard deviation
Valproic Acidn=10 Participants1.43.5
Placebon=4 Participants-4.53.4
p0.02t-test, 2 sided

Amyloid biomarkers

2 endpoints
Primary/protocol endpoint

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

Primary/registry result

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

GroupValue (mean), pg/mlStandard deviation
Valproic Acidn=10 Participants57.128.4
Placebon=4 Participants44.724.9
p0.55t-test, 2 sided

Tau biomarkers

2 endpoints
Secondary/protocol endpoint

Change in Cerebrospinal Fluid P-tau Levels (pg/ml)

Time frame:Baseline and day 28

change from baseline, improvement

Secondary/registry result

Change in Cerebrospinal Fluid P-tau Levels (pg/ml)

Time frame:Baseline and day 28

change from baseline, improvement

Posted result

GroupValue (mean), pg/mlStandard deviation
Valproic Acidn=10 Participants-5.711.9
Placebon=4 Participants11.025.7
p0.19t-test, 2 sided

Fluid / digital biomarkers

2 endpoints
Secondary/protocol endpoint

Change in Cerebrospinal Fluid Clusterin Levels (pg/ml)

Time frame:Baseline and day 28

change from baseline, improvement

Secondary/registry result

Change in Cerebrospinal Fluid Clusterin Levels (pg/ml)

Time frame:Baseline and day 28

change from baseline, improvement

Posted result

GroupValue (mean), pg/mlStandard deviation
Valproic Acidn=10 Participants26104373
Placebon=4 Participants201871
p0.31t-test, 2 sided

Safety / tolerability / PK

2 endpoints
Primary/protocol endpoint

Frequency of Adverse Events Over the Duration of the Study by Study Arm

Time frame:Day 35

event count, event

Primary/registry result

Frequency of Adverse Events Over the Duration of the Study by Study Arm

Time frame:Day 35

event count, event

Posted result

GroupValue (count_of_participants), ParticipantsReported bounds
Valproic Acidn=10 Participants8-
Placebon=4 Participants1-

Provenance

Sources

Trial identity, design, statusClinicalTrials.gov API v2
Snapshot dateJuly 21, 2026
Endpoint classificationDelfa ADRD endpoint taxonomy
Results tableClinicalTrials.gov results section

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.