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

December 17, 2025 updated by: Alzheon Inc.

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)

The study will investigate the effects of oral ALZ-801, in subjects with Early AD who have the APOE4/4 or APOE3/4 genotype, on the biomarkers of core AD pathology. The objectives of this study include determining the efficacy and safety/tolerability of ALZ-801. In addition, the study will evaluate the extended PK profile over 8 hours in 16 subjects after 65 weeks of treatment.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

The LTE year 1 & 2 study will investigate the effects of oral ALZ-801, in subjects with Early AD who have the APOE4/4 or APOE3/4 genotype, on the biomarkers of core AD pathology. The objectives of LTE study are to continue longitudinal assessment of the efficacy and safety/tolerability of ALZ-801 over a total period of 4 years (2-year Core study plus 2 years of LTE).

Core study: ALZ-801 265 mg twice daily (BID) in the Core Study, Weeks 0-104

LTE year 1: ALZ-801 265 mg BID in the Core Study and the Long-Term Extension (LTE, Weeks 104-208)

LTE year 2: ALZ-801 265mg BID in the Core Study and LTE Year 1 (Weeks 104-156), and LTE Year 2 (Weeks 156-208)

Study Type

Interventional

Enrollment (Actual)

84

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Czechia
      • Brno, Czechia
        • St. Anne's University Hospital
      • Prague, Czechia
        • Motol University Hospital
      • Rychnov nad Kněžnou, Czechia
        • Vestra Clinics
  • Netherlands
      • 's-Hertogenbosch, Netherlands
        • Brain Research Center
      • Amsterdam, Netherlands
        • Brain Research Center
      • Zwolle, Netherlands
        • Brain Research Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

50 years to 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Active treatment
ALZ-801 265 mg tablets once daily for two weeks and twice daily thereafter
Drug: ALZ-801
ALZ-801 265 mg twice daily (BID)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence, Nature, and Severity of Treatment Emergent Adverse events (TEAE)
Time Frame: Week 108
Safety and tolerability as measured by incidence, nature and severity of treatment emergent adverse events (TEAE), serious TEAE, and TEAE leading to withdrawal.
Week 108
Plasma Biomarker of Core AD Pathology
Time Frame: Week 104
Percent change from baseline in p-tau181
Week 104
Volumetric Magnetic Resonance Imaging (vMRI) Biomarker - Hippocampal Volume
Time Frame: Weeks 104
Change from baseline in hippocampal volume measured in mm3
Weeks 104

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Plasma Biomarkers of AD and Neurodegeneration
Time Frame: Weeks 104
Percent changes from baseline in: Aβ-40, Aβ-42,p-tau217 and plasma glial fibrillary acidic protein (GFAP),NfL
Weeks 104
vMRI Biomarker - Ventricular volume and Cortical Thickness
Time Frame: Weeks 104, 156 and 208
Change from baseline in cortical thickness measured in mm3
Weeks 104, 156 and 208
Additional CSF Biomarkers of AD Pathology and Neurodegeneration
Time Frame: Weeks 104
Percent changes from baseline for: p-tau217,Aβ-40, Aβ-42, NfL, t-tau, sTREM2, YKL-40 and neurogranin
Weeks 104
Incidence, Nature, and Severity of Treatment Emergent Adverse events (TEAE)
Time Frame: Week 160 and week 212
Safety and tolerability as measured by incidence, nature and severity of treatment emergent adverse events (TEAE), serious TEAE, and TEAE leading to withdrawal.
Week 160 and week 212
Volumetric Magnetic Resonance Imaging (vMRI) Biomarker - Hippocampal Volume
Time Frame: Week 156 and week 208
Change from baseline in hippocampal volume measured in mm3
Week 156 and week 208

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Cognitive assessment - Rey Auditory Verbal Learning Test (RAVLT)
Time Frame: Weeks 104, week 156 and week 208
Change from baseline in RAVLT score
Weeks 104, week 156 and week 208
Cognitive Assessment - Digit Symbol Substitution Test (DSST)
Time Frame: Weeks 104, Week 156 and Week 208
Change from baseline in DSST score
Weeks 104, Week 156 and Week 208
Functional Assessment - Amsterdam Instrumental Activities of Daily Living (A-IADL)
Time Frame: Weeks 104, Week 156 and Week 208
Change from baseline in A-IADL score
Weeks 104, Week 156 and Week 208
Cognitive Assessment - Mini Mental State Examination (MMSE)
Time Frame: Weeks 104, Week 156 and Week 208
Change from baseline in MMSE score
Weeks 104, Week 156 and Week 208
Global Assessment - Clinical Dementia Rating - Sum of Boxes (CDR-SB)
Time Frame: Weeks 104, Week 156 and Week 208
Change from baseline in CDR-SB score
Weeks 104, Week 156 and Week 208

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alzheon Inc.

Investigators

  • Study Director: John Hey, PhD, Alzheon Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 30, 2020

Primary Completion (Actual)

June 20, 2023

Study Completion (Actual)

July 16, 2025

Study Registration Dates

First Submitted

December 28, 2020

First Submitted That Met QC Criteria

December 31, 2020

First Posted (Actual)

January 5, 2021

Study Record Updates

Last Update Posted (Estimated)

December 18, 2025

Last Update Submitted That Met QC Criteria

December 17, 2025

Last Verified

December 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALZ-801-201ADBM

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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