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Early Alzheimer's DiseasePhase 2NCT04693520

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

A Phase 2 study testing ALZ-801 in people with Early Alzheimer's Disease, run by Alzheon Inc. at 6 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 50 to 80 years
Conditions the study is for
Early Alzheimer's Disease
What is being tested?
ALZ-801
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
84 participants
Where
6 sitesin 2 countries
Study dates
30 Sep 2020to 16 Jul 2025
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 50 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age between 50 and 80 years, inclusive.
  • 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].
  • One of the following apolipoprotein E (APOE) genotypes - either APOE4/4 (homozygous) or APOE3/4 (heterozygous).
  • 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.
  • 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.
  • Stable doses of acetylcholinesterase for the duration of the study are allowed.

Who cannot join

  • Brain MRI at screening indicative of significant abnormality
  • Diagnosis of neurodegenerative disorder other than AD
  • Current diagnosis of Major Depressive Disorder (MDD)
  • Concomitant treatment with memantine.

Trial locations and contacts

6 sites in 2 countries.

6 sites in 2 countries

Czechia3 sites

  • St. Anne's University Hospital

    Brno

  • Motol University Hospital

    Prague

  • Vestra Clinics

    Rychnov nad Kněžnou

Other trials in Czechia

Netherlands3 sites

  • Brain Research Center

    's-Hertogenbosch

  • Brain Research Center

    Amsterdam

  • Brain Research Center

    Zwolle

Other trials in Netherlands

About this study

Official title: 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.

Read the 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)

Being tested
ALZ-801
Medical subject headings
Alzheimer Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ALZ-801Drug

    ALZ-801 265 mg twice daily (BID)

Study groups in NCT04693520
Study groupRoleWhat they receive
Active treatmentExperimentalALZ-801 265 mg tablets once daily for two weeks and twice daily thereafter
Receives: Drug: ALZ-801

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Plasma Biomarker of Core AD Pathology

    Percent change from baseline in p-tau181

    Measured: Week 104

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

    Safety and tolerability as measured by incidence, nature and severity of treatment emergent adverse events (TEAE), serious TEAE, and TEAE leading to withdrawal.

    Measured: Week 108

  3. Volumetric Magnetic Resonance Imaging (vMRI) Biomarker - Hippocampal Volume

    Change from baseline in hippocampal volume measured in mm3

    Measured: Weeks 104

Secondary outcomes (5)

  1. Plasma Biomarkers of AD and Neurodegeneration

    Percent changes from baseline in: Aβ-40, Aβ-42,p-tau217 and plasma glial fibrillary acidic protein (GFAP),NfL

    Measured: Weeks 104

  2. vMRI Biomarker - Ventricular volume and Cortical Thickness

    Change from baseline in cortical thickness measured in mm3

    Measured: Weeks 104, 156 and 208

  3. Additional CSF Biomarkers of AD Pathology and Neurodegeneration

    Percent changes from baseline for: p-tau217,Aβ-40, Aβ-42, NfL, t-tau, sTREM2, YKL-40 and neurogranin

    Measured: Weeks 104

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

    Safety and tolerability as measured by incidence, nature and severity of treatment emergent adverse events (TEAE), serious TEAE, and TEAE leading to withdrawal.

    Measured: Week 160 and week 212

  5. Volumetric Magnetic Resonance Imaging (vMRI) Biomarker - Hippocampal Volume

    Change from baseline in hippocampal volume measured in mm3

    Measured: Week 156 and week 208

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Jan 2021
  2. Study start30 Sep 2020
  3. Primary completion20 Jun 2023
  4. Study completion16 Jul 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
84 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04693520
Sponsor's protocol ID
ALZ-801-201ADBM
Status last verified
Dec 2025
First submitted
28 Dec 2020
Last updated
18 Dec 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Hey JA, Abushakra S, Power A, Watson D, Porsteinsson A, Sabbagh M, MacSweeney E, Cohen S, Boada M, Doraiswamy PM, Elahi FM, Rissman RA, Liang E, Flint S, Hristova E, Kesslak P, McLaine R, Albayrak A, Schaefer JF, Yu JY, Tolar L, Tolar M. Plasma Biomarker Effects of Oral Valiltramiprosate/ALZ-801 in Early Alzheimer's Disease from Phase 3 and Phase 2 Trials: Analysis of Core Biomarkers and Correlations with Clinical and Neuroimaging Outcomes. Drugs. 2026 Sep 1. doi: 10.1007/s40265-026-02381-3. Online ahead of print.

    PubMed 42678603

  • Hey JA, Yu JY, Abushakra S, Schaefer JF, Power A, Kesslak P, Paul J, Tolar M. Clinical Pharmacokinetics of Oral ALZ-801/Valiltramiprosate in a 2-Year Phase 2 Trial of APOE4 Carriers with Early Alzheimer's Disease. Clin Pharmacokinet. 2025 Mar;64(3):407-424. doi: 10.1007/s40262-025-01482-8. Epub 2025 Feb 5.

    PubMed 39907966

  • Hey JA, Yu JY, Abushakra S, Schaefer JF, Power A, Kesslak P, Tolar M. Analysis of Cerebrospinal Fluid, Plasma beta-Amyloid Biomarkers, and Cognition from a 2-Year Phase 2 Trial Evaluating Oral ALZ-801/Valiltramiprosate in APOE4 Carriers with Early Alzheimer's Disease Using Quantitative Systems Pharmacology Model. Drugs. 2024 Jul;84(7):825-839. doi: 10.1007/s40265-024-02068-7. Epub 2024 Jun 20.

    PubMed 38902572

  • Hey JA, Abushakra S, Blennow K, Reiman EM, Hort J, Prins ND, Sheardova K, Kesslak P, Shen L, Zhu X, Albayrak A, Paul J, Schaefer JF, Power A, Tolar M. Effects of Oral ALZ-801/Valiltramiprosate on Plasma Biomarkers, Brain Hippocampal Volume, and Cognition: Results of 2-Year Single-Arm, Open-Label, Phase 2 Trial in APOE4 Carriers with Early Alzheimer's Disease. Drugs. 2024 Jul;84(7):811-823. doi: 10.1007/s40265-024-02067-8. Epub 2024 Jun 20.

    PubMed 38902571

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ALZ-801 in people with Early Alzheimer's Disease, run by Alzheon Inc. at 6 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 18 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in Czechia, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Plasma Biomarker of Core AD Pathology (time frame: Week 104). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04693520, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Dec 2025.

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