CompletedPhase 2

Biomarker Effects of ALZ-801 in APOE4 Carriers With Early Alzheimer's Disease

A Phase 2, Single-arm Study of the Biomarker Effects of ALZ-801 in Subjects With Early Alzheimer's Disease Who Are Carriers of the ε4 Variant of the Apolipoprotein E Gene (APOE4/4 or APOE3/4)

Lead sponsor

Alzheon Inc.

Asset

ALZ-801

Listed sites

Recruiting sites

Enrollment

actual

Study population

Alzheimer’s disease

Key I/E criteria

•Alzheimer's disease•Amyloid biomarker required (PET/CSF)•MMSE 22-30

Primary endpoints

•Phosphorylated tau 181 (p-tau181)•Incidence, Nature, and Severity of Treatment Emergent Adverse events (TEAE)•Volumetric Magnetic Resonance Imaging (vMRI) Biomarker - Hippocampal Volume

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 IDALZ-801-201ADBM

NCT IDNCT04693520

Timeline

Milestones

Study start2020-09-30actual

Study first posted2021-01-05actual

Primary completion2023-06-20actual

Study completion2025-07-16actual

Last update posted2025-12-18actual

Assets

Drug assets

ALZ-801

Study populations

Who this study enrolls

Alzheimer’s disease

Eligibility

Who can enroll

Minimum age50 Years

Maximum age80 Years

SexAll

Healthy volunteersNot accepted

Inclusion criteria

1. Age between 50 and 80 years, inclusive.

2. Early Alzheimer's Disease (AD): a diagnosis of Probable AD Dementia or Mild Cognitive Impairment (MCI) due to AD in accordance with the National Institute on Aging-Alzheimer's Association (NIA-AA) Working Group Criteria [Albert et al, 2011; McKhann et al, 2011].

3. One of the following apolipoprotein E (APOE) genotypes - either APOE4/4 (homozygous) or APOE3/4 (heterozygous).

4. MMSE score 22 to 30 inclusive; Clinical Dementia Rating (CDR)-Global Score of 0.5 or 1.0, and CDR Memory Box Score of ≥ 0.5.

5. Documented confirmation of AD diagnosis by either positive amyloid positron emission tomography (PET) or positive CSF AD signature. Subjects without documented positive AD biomarker status must have a positive CSF biomarker result from a sample provided at screening.

6. Stable doses of acetylcholinesterase for the duration of the study are allowed

Exclusion criteria

1. Brain MRI at screening indicative of significant abnormality

2. Diagnosis of neurodegenerative disorder other than AD

3. Current diagnosis of Major Depressive Disorder (MDD)

4. Concomitant treatment with memantine.

Endpoints (13)

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

Global cognition

Neuroimaging

Safety / tolerability / PK

Function / daily living

Tau biomarkers

Neurodegeneration biomarkers

Fluid / digital biomarkers

Global cognition

4 endpoints

Other/protocol endpoint

Cognitive assessment - Rey Auditory Verbal Learning Test (RAVLT)

Time frame:Weeks 104, week 156 and week 208

change from baseline, improvement

Other/protocol endpoint

Cognitive Assessment - Digit Symbol Substitution Test (DSST)

Time frame:Weeks 104, Week 156 and Week 208

change from baseline, improvement

Other/protocol endpoint

Cognitive Assessment - Mini Mental State Examination (MMSE)

Time frame:Weeks 104, Week 156 and Week 208

Mini-Mental State Examination (MMSE)

change from baseline, improvement

Other/protocol endpoint

Global Assessment - Clinical Dementia Rating - Sum of Boxes (CDR-SB)

Time frame:Weeks 104, Week 156 and Week 208

Clinical Dementia Rating-Sum of Boxes (CDR-SB)

change from baseline, improvement

Function / daily living

1 endpoint

Other/protocol endpoint

Functional Assessment - Amsterdam Instrumental Activities of Daily Living (A-IADL)

Time frame:Weeks 104, Week 156 and Week 208

change from baseline, improvement

Tau biomarkers

1 endpoint

Secondary/protocol endpoint

Additional CSF Biomarkers of AD Pathology and Neurodegeneration

Time frame:Weeks 104

Phosphorylated tau 217 (p-tau217)

percent change from baseline, improvement

Neurodegeneration biomarkers

1 endpoint

Secondary/protocol endpoint

Plasma Biomarkers of AD and Neurodegeneration

Time frame:Weeks 104

Phosphorylated tau 217 (p-tau217)

percent change from baseline, improvement

Neuroimaging

3 endpoints

Primary/protocol endpoint

Volumetric Magnetic Resonance Imaging (vMRI) Biomarker - Hippocampal Volume

Time frame:Weeks 104

change from baseline, improvement

Secondary/protocol endpoint

vMRI Biomarker - Ventricular volume and Cortical Thickness

Time frame:Weeks 104, 156 and 208

change from baseline, improvement

Secondary/protocol endpoint

Volumetric Magnetic Resonance Imaging (vMRI) Biomarker - Hippocampal Volume

Time frame:Week 156 and week 208

change from baseline, improvement

Fluid / digital biomarkers

1 endpoint

Primary/protocol endpoint

Plasma Biomarker of Core AD Pathology

Time frame:Week 104

Phosphorylated tau 181 (p-tau181)

percent change from baseline, improvement

Safety / tolerability / PK

2 endpoints

Primary/protocol endpoint

Incidence, Nature, and Severity of Treatment Emergent Adverse events (TEAE)

Time frame:Week 108

event count, event

Secondary/protocol endpoint

Incidence, Nature, and Severity of Treatment Emergent Adverse events (TEAE)

Time frame:Week 160 and week 212

event count, event

Publications (3)

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.