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NCT Number: NCT04693520

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

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.

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Key information

Conditions

Early Alzheimer's Disease Alzheimer Disease Brain Diseases Central Nervous System Diseases Dementia Mental Disorders Nervous System Diseases Neurocognitive Disorders Neurodegenerative Diseases Tauopathies

Age range

50 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

St. Anne's University Hospital, Brno, Czechia

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About this study

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)

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

ALZ-801

Drug

ALZ-801 265 mg twice daily (BID)

Primary outcomes

  1. Plasma Biomarker of Core AD Pathology

    Time frame: Week 104

    Percent change from baseline in p-tau181

  2. 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.

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

    Time frame: Weeks 104

    Change from baseline in hippocampal volume measured in mm3

Secondary outcomes

  1. 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

  2. vMRI Biomarker - Ventricular volume and Cortical Thickness

    Time frame: Weeks 104, 156 and 208

    Change from baseline in cortical thickness measured in mm3

  3. 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

  4. 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.

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

    Time frame: Week 156 and week 208

    Change from baseline in hippocampal volume measured in mm3

Other outcomes

  1. Cognitive assessment - Rey Auditory Verbal Learning Test (RAVLT)

    Time frame: Weeks 104, week 156 and week 208

    Change from baseline in RAVLT score

  2. Cognitive Assessment - Digit Symbol Substitution Test (DSST)

    Time frame: Weeks 104, Week 156 and Week 208

    Change from baseline in DSST score

  3. 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

  4. Cognitive Assessment - Mini Mental State Examination (MMSE)

    Time frame: Weeks 104, Week 156 and Week 208

    Change from baseline in MMSE score

  5. 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

Sponsors and collaborators

Lead sponsor

Alzheon Inc.

Industry

Registry information

Official study 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)

Important dates

Study start
2020
Primary completion
2023
Study completion
2025
First posted
Jan 5, 2021
Registry last updated
Dec 18, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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