← Trials/Trial dossier/NCT03706885

EPAD

CompletedPhase 1

Efavirenz for Patients With Alzheimer's Disease

A Proof-of-Concept Clinical Research Study of Efavirenz in Patients With Alzheimer's Disease

Lead sponsor

Case Western Reserve University

Asset

Efavirenz

Listed sites

Recruiting sites

Enrollment

actual

Study population

Alzheimer’s disease

Key I/E criteria

•Alzheimer's disease•MMSE 16-30

Primary endpoint

•Plasma levels of 24-hydroxycholesterol

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 IDADDF 20160601

NCT IDNCT03706885

Timeline

Milestones

Study start2018-05-05actual

Study first posted2018-10-16actual

Primary completion2022-01-28actual

Study completion2022-01-28actual

Last update posted2023-02-15actual

Assets

Drug assets

Efavirenz

Study populations

Who this study enrolls

Alzheimer’s disease

Eligibility

Who can enroll

Minimum age55 Years

Maximum age85 Years

SexAll

Healthy volunteersNot accepted

Inclusion criteria

•Between the ages of 55-85

•Either male or female

•Diagnosis of (a) as per below:

a) Mild Cognitive Impairment (MCI) or early dementia due to AD as defined by (1) complaint of cognitive decline, (2) MMSE Total=16-30, (3) CDR=0.5-1

•Fluent in English

•Education >8 years, literate, and/or good working history that precludes consideration of mental retardation

•Visual and auditory acuity sufficient for neuropsychological testing

•Modified Hachinski Ischemic Score<4

•No major health issues or diseases expected to interfere with the study

•Willing to complete all assessments and study procedures

•Not pregnant, lactating or of child-bearing potential (women must be >2 years post- menopausal or surgically sterile)

•If cognitively impaired, study partner with frequent contact with patient willing to accompany patient to visits and complete partner study forms

•No contraindication or hypersensitivity to EFV

•Screening laboratory testing must be within normal limits or, if abnormal, must be judged to be clinically insignificant by the investigators

•Stable use of cholinesterase inhibitor is permitted if doses are stable for 3 months prior to enrollment

Exclusion criteria

•Any CNS disease other than suspected prodromal or early AD, such as clinical stroke, brain tumor, normal pressure hydrocephalus, brain tumor, multiple sclerosis, significant head trauma with persistent neurological or cognitive deficits or complaints, Parkinson's Disease, frontotemporal dementia, or other neurodegenerative diseases

•Ongoing major and active psychiatric disorder and/or other concurrent medical condition that, in the opinion of the investigator, might compromise safety and/or compliance with study requirements.

•History of alcohol or other substance abuse or dependence within the past two years

•Any significant systemic illness or unstable medical condition that could affect study compliance, including a history of prolonged QTc

•Laboratory abnormalities in B12, TSH, or other common laboratory parameters that might contribute to cognitive dysfunction

•Current use of medications with psychoactive properties that may deleteriously affect cognition (e.g., anticholinergics, antihistamines, antipsychotics, sedative hypnotics, anxiolytics) that, in the opinion of the investigator, may deleteriously affect cognition. Use of other investigational agents one month prior to entry and for the duration of the study

•Treatment with any of the following agents/classes within the past 3 months: simvastatin, antiepileptic agents, clopidogrel, voriconazole, systemic ketoconazole, cyclosporine, St. John's Wort.

Endpoints (3)

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.

Other (unclassified)

3 endpoints

Primary/protocol endpoint/low confidence

Plasma levels of 24-hydroxycholesterol

Time frame:1 year

descriptive

Secondary/protocol endpoint/low confidence

Plasma levels of deuterated 24-hydroxycholesterol

Time frame:1 year

descriptive

Secondary/protocol endpoint/low confidence

APOE isoform status (E2, E3, or E4) and presence of the SNPs rs754203 and rs3745274 in CYP46A1 and CYP2B6, respectively.

Time frame:1 year

categorical status, descriptive

Publications (40)

Bibliography

Records linked to this trial through ClinicalTrials.gov references, PubMed NCT search, and curated study seeds. 'Canonical' marks design/result papers; others are registry references or candidates.

Registry references + supporting bibliography

Provenance

Sources

Trial identity, design, statusClinicalTrials.gov API v2

Snapshot dateJuly 21, 2026

Endpoint classificationDelfa ADRD endpoint taxonomy

Results tableno registry results posted yet

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.