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CompletedNCT04770220APOLLOE4Updated Sep 29, 2026Results posted

An Efficacy and Safety Study of ALZ-801 in APOE4/4 Early AD Subjects

A Phase 3 interventional study of Experimental: ALZ-801 and Placebo Comparator: Placebo in Early Alzheimer's Disease, sponsored by Alzheon Inc.. Completed at 95 sites in 9 countries. Open to participants aged 50 Years to 80 Years. Per ClinicalTrials.gov, last updated 2026-09-29.

Sponsored by Alzheon Inc. · Phase 3, Interventional, and Treatment

Updated Sep 29, 2026Posted results revisedGo to Updates ↓
Phase
Phase 3
Study type
Interventional
Enrollment
325
Allocation
Randomized
Ages
50 Years to 80 Years
Sex
All
01

Study summary

This study is being conducted to evaluate the safety and efficacy of ALZ-801 in Early Alzheimer's disease (AD) subjects with the APOE4/4 genotype. This is a double-blind, randomized trial with one dose of ALZ-801 compared to placebo.

Read the detailed description

This is a multi-center, double-blind study that will evaluate 265 mg twice daily (BID) of ALZ-801, an oral tablet, over 78 weeks as a treatment for subjects (50-80 years old) with Early AD who are homozygous for the ε4 allele of the apolipoprotein gene (APOE4 homozygous or APOE4/4). The primary efficacy outcome assessment is a measure of cognition (ADAS-cog 13). Additional measures of global and functional impairments will also be assessed. Imaging and soluble biomarkers of AD and neurodegeneration will be measured and a sub-study to evaluate cerebrospinal fluid (CSF) biomarkers is also included.

02

Conditions studied

  • Early Alzheimer's Disease

Browse trials for

03

In context

Alzheimer Disease

3,965 studies on the registry are indexed under Alzheimer Disease; 926 are open to participants now.

This study's enrollment of 325 is above the median of 70 across 3,019 interventional studies indexed under Alzheimer Disease.

Browse Alzheimer Disease studies →

Lead sponsor

Alzheon Inc. is the lead sponsor of 6 studies on the registry; 1 is open to participants now.

Of its 5 completed or terminated interventional studies of FDA-regulated products, 1 (20%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
50 Years to 80 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • Clinical diagnosis of MCI or Mild Dementia due to AD consistent with the National Institute on Aging-Alzheimer's Association (NIA-AA) Working Group Criteria.
  • Homozygous for the ε4 allele of the apolipoprotein E gene (APOE4/4).
  • score at Screening of 22 to 30 (inclusive).
  • CDR - Global score of 0.5 or 1 and CDR Memory Box Score of ≥ 0.5.
  • RBANS delayed memory index score ≤ 85.
  • Evidence of progressive memory loss over the last 12 months per investigator assessment

Exclusion criteria

Exclusion Criteria:

  • Brain magnetic resonance imaging (MRI) indicative of significant abnormality per central reader, other than AD related atrophy. Computed tomography (CT) scan acceptable for subjects who cannot undergo MRI.
  • Diagnosis of neurodegenerative disorder other than AD.
  • Diagnosis of major depressive disorder (MDD) within one year prior to screening.
  • Currently taking memantine or has taken memantine within 12 weeks prior to the Baseline Visit.
  • History of suicidal behavior within one year prior to screening or has ongoing suicidal ideation.
  • History of seizures, excluding febrile seizures of childhood or a single distant seizure (> 5 years).
  • Medically confirmed history of recent cerebral infarct or within one year prior to screening.
Terms in these criteria, in plain language(2)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

MMSE and MoCA

Short tests of memory and thinking, each scored out of 30. Lower scores suggest more difficulty; criteria give a range, either to include people with memory problems or to leave them out.

TIA (transient ischaemic attack)

A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.

Stroke symptoms need emergency care at once, even if they go away.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
325 participants (actual)

Study arms

  • Experimental
    ALZ-801

    ALZ-801 265 mg BID tablet orally. Subjects will receive placebo in the morning and one tablet of ALZ-801 265 mg tablet in the evening during the first two weeks of the study; thereafter, they will receive a 265 mg tablet twice daily (BID).

    Drug: Experimental: ALZ-801 · Drug: Placebo Comparator: Placebo

  • Placebo comparator
    Placebo

    Subjects in the placebo treatment arm will receive placebo tablets BID throughout the study

    Drug: Placebo Comparator: Placebo

Interventions

  • DrugExperimental: ALZ-801

    ALZ-801 tablet 265 mg once daily in the evening for the first 2 weeks, then ALZ-801 tablet 265 mg BID

    Also known as: valiltramiprosate, tramiprosate prodrug

  • DrugPlacebo Comparator: Placebo

    Placebo tablet BID

06

What researchers measure

Primary outcomes

  1. Primary Cognitive Efficacy Endpoint (ADAS-Cog13)

    Change from baseline in Alzheimer's Disease Assessment Scale - Cognitive Subscale 13 (ADAS-Cog13) scores. The ADAS-Cog13 is a rater administered instrument that was designed to assess the severity of the dysfunction in the cognitive and noncognitive behaviors characteristic of persons with AD. The cognitive subscale of the ADAS-Cog13 consists of 13 items assessing areas of cognitive function most typically impaired in AD: orientation, verbal memory, language, praxis, delayed free recall, digit cancellation. The ADAS-Cog13 scale ranges from 0 to 85. Higher scores indicate greater disease severity.

    Time frame: Baseline to Week 78

  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 TEAEs, and TEAEs leading to withdrawal.

    Time frame: Entire study: approximately 82 weeks. (first dose of study drug until end of Safety Follow-up Visit at 28 +/- 7 days after the last dose (ie, 78-week treatment period plus 4-weeks follow-up after last dose up to total of 82 weeks)

Secondary outcomes

  1. Key Secondary Endpoint (A-IADL-W)

    Change from baseline in Amsterdam - Instrumental Activities of Daily Living scores calculated using the weighted average method (A-IADL-W score). The A-IADL Questionnaire is a 70-item informant-based computerized questionnaire aimed at detecting deficits in complex functions at the early stages of AD. Instrumental ADL can be described as the activities necessary to function independently in society. These activities include, but are not limited to, cooking, doing finances, and shopping. They are complex everyday tasks, determined by multiple cognitive processes and controlled processing. They can be distinguished from basic ADL, which include basic self-care skills. The A-IADL-W has a score range of 0-100 and is calculated as follows: (sum of all scores / number of questions scored) × 25. For A-IADL-W, higher scores indicates worse functioning or more impairment.

    Time frame: Baseline to Week 78

  2. Key Secondary Endpoint (CDR-SB)

    Change from baseline in Clinical Dementia Rating - Sum of Boxes (CDR-SB) scores. CDR-SB is a semi-structured interview of participants and their caregivers. Participant's cognitive status is rated across 6 domains of functioning, including memory, orientation, judgment/problem solving, community affairs, home/hobbies, and personal care. Severity score assigned for each of 6 domains; Total score (SB) ranges from 0 to 18. Higher scores indicate greater disease severity.

    Time frame: Baseline to Week 78

  3. Functional Assessment (DAD)

    Change from baseline in Disability Assessment for Dementia (DAD)scores. The DAD consists of 40 items with a score range of 0-100 to evaluate the basic and instrumental activities of daily living of subjects with dementia. It is administered through an interview with the caregiver. Higher DAD scores indicate less disability or better function.

    Time frame: Baseline to Week 78

  4. Global Cognition Assessment (MMSE)

    Change from baseline in Mini-Mental State Examination (MMSE) score. The MMSE is a measure of global cognition that is widely used for clinical staging of Alzheimer's Disease. It consists of 11 domains items for a score range of 0-30 to assess general cognitive function. Higher score on MMSE means better cognitive skills.

    Time frame: Baseline to Week 78

Other outcomes

  1. Imaging Biomarker Endpoint (Hippocampal Volume)

    Change from baseline in total (bilateral) hippocampal volume (HV) (uL) as measured by Magnetic Resonance Imaging (MRI). HV atrophy is an early event in AD patients, especially in APOE4/4 homozygotes who show accelerated atrophy compared to APOE3/3 patients with Early AD. HV may be a marker of synaptic loss and neurodegeneration.

    Time frame: Baseline to Week 78

  2. Imaging Biomarker Endpoint (Cortical Thickness [Whole Cortex])

    Change from baseline in total (bilateral) cortical thickness (mm) as measured by MRI. Brain MRI studies in Alzheimer's Disease patients show progressive cortical atrophy, reflecting progressive neurodegeneration.

    Time frame: Baseline to Week 78

  3. Imaging Biomarker Endpoint (Cortical Thickness [Mayo Index])

    Change from baseline in total (bilateral) cortical thickness (mm) as measured by MRI. Brain MRI studies in Alzheimer's Disease patients show progressive cortical atrophy, reflecting progressive neurodegeneration. The Mayo Index refers specifically to the measurement of cortical thickness in the medial temporal lobe.

    Time frame: Baseline to Week 78

  4. Imaging Biomarker Endpoint (Whole Brain Volume)

    Change from baseline in whole brain volume (uL) as measured by Magnetic Resonance Imaging (MRI).

    Time frame: Baseline to Week 78

  5. Imaging Biomarker Endpoint (Ventricular Volume)

    Change from baseline in total (bilateral) ventricular volume (uL) as measured by Magnetic Resonance Imaging (MRI).

    Time frame: Baseline to Week 78

  6. Imaging Biomarker Endpoint - DTI in Grey Matter Mean Diffusivity - Bilateral Caudate

    Change from baseline in Grey Matter Mean Diffusivity (Bilateral Caudate) as measured by Diffusion Tensor Imaging - Magnetic Resonance Imaging (DTI-MRI). The mean diffusivity (MD) is the average of the three main diffusion values (eigenvalues) obtained from the diffusion tensor imaging (DTI). It is expressed as mm2/s. This unit quantifies the average rate of water diffusion across all directions within a tissue, providing an overall measure of tissue microstructural properties. Lower MD value suggests better maintenance of microstructural integrity of a given brain tissue. Positive treatment effects of ALZ-801 on DTI would present as lower mean diffusivity compared with the placebo group.

    Time frame: Baseline to Week 78

  7. Imaging Biomarker Endpoint - DTI in White Matter Mean Diffusivity (Bilateral Fornix)

    Change from baseline in Bilateral Fornix White Matter Mean Diffusivity as measured by Diffusion Tensor Imaging - Magnetic Resonance Imaging (DTI-MRI). The mean diffusivity (MD) is the average of the three main diffusion values (eigenvalues) obtained from the diffusion tensor imaging (DTI). It is expressed as mm2/s. This unit quantifies the average rate of water diffusion across all directions within a tissue, providing an overall measure of tissue microstructural properties. Lower MD value suggests better maintenance of microstructural integrity of a given brain tissue. Positive treatment effects of ALZ-801 on DTI would present as lower mean diffusivity compared with the placebo group.

    Time frame: Baseline to Week 78

  8. Imaging Biomarker Endpoint - White Matter Fractional Anisotropy (Bilateral Fornix)

    Change from baseline in Bilateral Fornix White Matter Fractional Anisotropy as measured by Diffusion Tensor Imaging - Magnetic Resonance Imaging (DTI-MRI). Fractional anisotropy (FA) is a unitless, scalar value that measures the degree of anisotropic (directional) water diffusion within a voxel, ranging from 0 to 1. A value of 0 indicates perfectly isotropic (equal in all directions) diffusion, while a value of 1 indicates perfectly anisotropic (directional) diffusion. Higher FA value suggests better maintenance of microstructural integrity of a given brain tissue. Positive treatment effects of ALZ-801 on DTI would present as higher FA in the ALZ-801 group compared with the placebo group.

    Time frame: Baseline to Week 78

  9. Cognitive Efficacy Endpoint (ADAS-Cog 13) - MCI Subgroup

    Change from baseline in Alzheimer's Disease Assessment Scale - Cognitive Subscale 13 (ADAS-cog 13) scores at 78 weeks. The ADAS-Cog13 is a rater administered instrument that was designed to assess the severity of the dysfunction in the cognitive and noncognitive behaviors characteristic of persons with AD. The cognitive subscale of the ADAS-Cog13 consists of 13 items assessing areas of cognitive function most typically impaired in AD: orientation, verbal memory, language, praxis, delayed free recall, digit cancellation. The ADAS-Cog13 scale ranges from 0 to 85. Higher scores indicate greater disease severity.

    Time frame: Baseline to Week 78

  10. Cognitive Efficacy Endpoint (ADAS-Cog 13) - Mild Alzheimer's Disease(Mild AD) Subgroup

    Change from baseline in Alzheimer's Disease Assessment Scale - Cognitive Subscale 13 (ADAS-cog 13) scores at 78 weeks. The ADAS-Cog13 is a rater administered instrument that was designed to assess the severity of the dysfunction in the cognitive and noncognitive behaviors characteristic of persons with AD. The cognitive subscale of the ADAS-Cog13 consists of 13 items assessing areas of cognitive function most typically impaired in AD: orientation, verbal memory, language, praxis, delayed free recall, digit cancellation. The ADAS-Cog13 scale ranges from 0 to 85. Higher scores indicate greater disease severity.

    Time frame: Baseline to Week 78

  11. A-IADL-W - MCI Subgroup

    Change from baseline in Amsterdam - Instrumental Activities of Daily Living scores calculated using the weighted average method (A-IADL-W score). The A-IADL Questionnaire is a 70-item informant-based computerized questionnaire aimed at detecting deficits in complex functions at the early stages of AD. Instrumental ADL can be described as the activities necessary to function independently in society. These activities include, but are not limited to, cooking, doing finances, and shopping. They are complex everyday tasks, determined by multiple cognitive processes and controlled processing. They can be distinguished from basic ADL, which include basic self-care skills. The A-IADL-W has a score range of 0-100 and is calculated as follows: (sum of all scores / number of questions scored) × 25. For A-IADL-W, higher scores indicates worse functioning or more impairment.

    Time frame: Baseline to Week 78

  12. A-IADL-W - Mild AD Subgroup

    Change from baseline in Amsterdam - Instrumental Activities of Daily Living scores calculated using the weighted average method (A-IADL-W score). The A-IADL Questionnaire is a 70-item informant-based computerized questionnaire aimed at detecting deficits in complex functions at the early stages of AD. Instrumental ADL can be described as the activities necessary to function independently in society. These activities include, but are not limited to, cooking, doing finances, and shopping. They are complex everyday tasks, determined by multiple cognitive processes and controlled processing. They can be distinguished from basic ADL, which include basic self-care skills. The A-IADL-W has a score range of 0-100 and is calculated as follows: (sum of all scores / number of questions scored) × 25. For A-IADL-W, higher scores indicates worse functioning or more impairment.

    Time frame: Baseline to Week 78

  13. CDR-SB - MCI Subgroup

    Change from baseline in Clinical Dementia Rating - Sum of Boxes (CDR-SB) scores. CDR-SB is a semi-structured interview of participants and their caregivers. Participant's cognitive status is rated across 6 domains of functioning, including memory, orientation, judgment/problem solving, community affairs, home/hobbies, and personal care. Severity score assigned for each of 6 domains; Total score (SB) ranges from 0 to 18. Higher scores indicate greater disease severity.

    Time frame: Baseline to Week 78

  14. CDR-SB - Mild AD Subgroup

    Change from baseline in Clinical Dementia Rating - Sum of Boxes (CDR-SB) scores. CDR-SB is a semi-structured interview of participants and their caregivers. Participant's cognitive status is rated across 6 domains of functioning, including memory, orientation, judgment/problem solving, community affairs, home/hobbies, and personal care. Severity score assigned for each of 6 domains; Total score (SB) ranges from 0 to 18. Higher scores indicate greater disease severity.

    Time frame: Baseline to Week 78

  15. Functional Assessment (DAD) - MCI Subgroup

    Change from baseline in Disability Assessment for Dementia (DAD)scores. The DAD consists of 40 items with a score range of 0-100 to evaluate the basic and instrumental activities of daily living of subjects with dementia. It is administered through an interview with the caregiver. Higher DAD scores indicate less disability or better function.

    Time frame: Baseline to Week 78

  16. Functional Assessment (DAD) - Mild AD Subgroup

    Change from baseline in Disability Assessment for Dementia (DAD)scores. The DAD consists of 40 items with a score range of 0-100 to evaluate the basic and instrumental activities of daily living of subjects with dementia. It is administered through an interview with the caregiver. Higher DAD scores indicate less disability or better function.

    Time frame: Baseline to Week 78

  17. Global Cognition Assessment (MMSE) - MCI Subgroup

    Change from baseline in Mini-Mental State Examination (MMSE) score. The MMSE is a measure of global cognition that is widely used for clinical staging of Alzheimer's Disease. It consists of 11 domains items for a score range of 0-30 to assess general cognitive function. Higher score on MMSE means better cognitive skills.

    Time frame: Baseline to Week 78

  18. Global Cognition Assessment (MMSE) - Mild AD Subgroup

    Change from baseline in Mini-Mental State Examination (MMSE) score. The MMSE is a measure of global cognition that is widely used for clinical staging of Alzheimer's Disease. It consists of 11 domains items for a score range of 0-30 to assess general cognitive function. Higher score on MMSE means better cognitive skills.

    Time frame: Baseline to Week 78

  19. Hippocampal Volume (MCI Subgroup)

    Change from baseline in total (bilateral) hippocampal volume (HV) (uL) as measured by Magnetic Resonance Imaging (MRI). HV atrophy is an early event in AD patients, especially in APOE4/4 homozygotes who show accelerated atrophy compared to APOE3/3 patients with Early AD. HV may be a marker of synaptic loss and neurodegeneration.

    Time frame: Baseline to Week 78

  20. Hippocampal Volume (Mild AD Subgroup)

    Change from baseline in total (bilateral) hippocampal volume (HV) (uL) as measured by Magnetic Resonance Imaging (MRI). HV atrophy is an early event in AD patients, especially in APOE4/4 homozygotes who show accelerated atrophy compared to APOE3/3 patients with Early AD. HV may be a marker of synaptic loss and neurodegeneration.

    Time frame: Baseline to Week 78

  21. Cortical Thickness (Whole Cortex) (MCI Subgroup)

    Change from baseline in total (bilateral) cortical thickness (mm) as measured by MRI. Brain MRI studies in Alzheimer's Disease patients show progressive cortical atrophy, reflecting progressive neurodegeneration.

    Time frame: Baseline to Week 78

  22. Cortical Thickness (Whole Cortex) (Mild AD Subgroup)

    Change from baseline in total (bilateral) cortical thickness (mm) as measured by MRI. Brain MRI studies in Alzheimer's Disease patients show progressive cortical atrophy, reflecting progressive neurodegeneration.

    Time frame: Baseline to Week 78

  23. Cortical Thickness (Mayo Index) (MCI Subgroup)

    Change from baseline in total (bilateral) cortical thickness (mm) as measured by MRI. Brain MRI studies in Alzheimer's Disease patients show progressive cortical atrophy, reflecting progressive neurodegeneration. The Mayo Index refers specifically to the measurement of cortical thickness in the medial temporal lobe versus the whole cortex.

    Time frame: Baseline to Week 78

  24. Cortical Thickness (Mayo Index) (Mild AD Subgroup)

    Change from baseline in total (bilateral) cortical thickness (mm) as measured by MRI. Brain MRI studies in Alzheimer's Disease patients show progressive cortical atrophy, reflecting progressive neurodegeneration. The Mayo Index refers specifically to the measurement of cortical thickness in the medial temporal lobe versus the whole cortex.

    Time frame: Baseline to Week 78

  25. Whole Brain Volume (MCI Subgroup)

    Change from baseline in whole brain volume (uL) as measured by Magnetic Resonance Imaging (MRI).

    Time frame: Baseline to Week 78

  26. Whole Brain Volume (Mild AD Subgroup)

    Change from baseline in whole brain volume (uL) as measured by Magnetic Resonance Imaging (MRI).

    Time frame: Baseline to Week 78

  27. Ventricular Volume (MCI Subgroup)

    Change from baseline in total (bilateral) ventricular volume (uL) as measured by Magnetic Resonance Imaging (MRI).

    Time frame: Baseline to Week 78

  28. Ventricular Volume (Mild AD Subgroup)

    Change from baseline in total (bilateral) ventricular volume (uL) as measured by Magnetic Resonance Imaging (MRI).

    Time frame: Baseline to Week 78

  29. Cognitive Efficacy Endpoint (ADAS-Cog 13) - Female Subgroup of the FAS

    Change from baseline in Alzheimer's Disease Assessment Scale - Cognitive Subscale 13 (ADAS-cog 13) scores at 78 weeks. The ADAS-Cog13 is a rater administered instrument that was designed to assess the severity of the dysfunction in the cognitive and noncognitive behaviors characteristic of persons with AD. The cognitive subscale of the ADAS-Cog13 consists of 13 items assessing areas of cognitive function most typically impaired in AD: orientation, verbal memory, language, praxis, delayed free recall, digit cancellation. The ADAS-Cog13 scale ranges from 0 to 85. Higher scores indicate greater disease severity.

    Time frame: Baseline to Week 78

  30. Cognitive Efficacy Endpoint (ADAS-Cog 13) - Male Subgroup of the FAS

    Change from baseline in Alzheimer's Disease Assessment Scale - Cognitive Subscale 13 (ADAS-cog 13) scores at 78 weeks. The ADAS-Cog13 is a rater administered instrument that was designed to assess the severity of the dysfunction in the cognitive and noncognitive behaviors characteristic of persons with AD. The cognitive subscale of the ADAS-Cog13 consists of 13 items assessing areas of cognitive function most typically impaired in AD: orientation, verbal memory, language, praxis, delayed free recall, digit cancellation. The ADAS-Cog13 scale ranges from 0 to 85. Higher scores indicate greater disease severity.

    Time frame: Baseline to Week 78

  31. A-IADL-W - Female Subgroup

    Change from baseline in Amsterdam - Instrumental Activities of Daily Living scores calculated using the weighted average method (A-IADL-W score). The A-IADL Questionnaire is a 70-item informant-based computerized questionnaire aimed at detecting deficits in complex functions at the early stages of AD. Instrumental ADL can be described as the activities necessary to function independently in society. These activities include, but are not limited to, cooking, doing finances, and shopping. They are complex everyday tasks, determined by multiple cognitive processes and controlled processing. They can be distinguished from basic ADL, which include basic self-care skills. The A-IADL-W has a score range of 0-100 and is calculated as follows: (sum of all scores / number of questions scored) × 25. For A-IADL-W, higher scores indicates worse functioning or more impairment.

    Time frame: Baseline to Week 78

  32. A-IADL-W - Male Subgroup

    Change from baseline in Amsterdam - Instrumental Activities of Daily Living scores calculated using the weighted average method (A-IADL-W score). The A-IADL Questionnaire is a 70-item informant-based computerized questionnaire aimed at detecting deficits in complex functions at the early stages of AD. Instrumental ADL can be described as the activities necessary to function independently in society. These activities include, but are not limited to, cooking, doing finances, and shopping. They are complex everyday tasks, determined by multiple cognitive processes and controlled processing. They can be distinguished from basic ADL, which include basic self-care skills. The A-IADL-W has a score range of 0-100 and is calculated as follows: (sum of all scores / number of questions scored) × 25. For A-IADL-W, higher scores indicates worse functioning or more impairment.

    Time frame: Baseline to Week 78

  33. CDR-SB - Female Subgroup

    Change from baseline in Clinical Dementia Rating - Sum of Boxes (CDR-SB) scores. CDR-SB is a semi-structured interview of participants and their caregivers. Participant's cognitive status is rated across 6 domains of functioning, including memory, orientation, judgment/problem solving, community affairs, home/hobbies, and personal care. Severity score assigned for each of 6 domains; Total score (SB) ranges from 0 to 18. Higher scores indicate greater disease severity.

    Time frame: Baseline to Week 78

  34. CDR-SB - Male Subgroup

    Change from baseline in Clinical Dementia Rating - Sum of Boxes (CDR-SB) scores. CDR-SB is a semi-structured interview of participants and their caregivers. Participant's cognitive status is rated across 6 domains of functioning, including memory, orientation, judgment/problem solving, community affairs, home/hobbies, and personal care. Severity score assigned for each of 6 domains; Total score (SB) ranges from 0 to 18. Higher scores indicate greater disease severity.

    Time frame: Baseline to Week 78

  35. Functional Assessment (DAD) - Female Subgroup

    Change from baseline in Disability Assessment for Dementia (DAD)scores. The DAD consists of 40 items with a score range of 0-100 to evaluate the basic and instrumental activities of daily living of subjects with dementia. It is administered through an interview with the caregiver. Higher DAD scores indicate less disability or better function.

    Time frame: Baseline to Week 78

  36. Functional Assessment (DAD) - Male Subgroup

    Change from baseline in Disability Assessment for Dementia (DAD)scores. The DAD consists of 40 items with a score range of 0-100 to evaluate the basic and instrumental activities of daily living of subjects with dementia. It is administered through an interview with the caregiver. Higher DAD scores indicate less disability or better function.

    Time frame: Baseline to Week 78

  37. Treatment Emergent Adverse Events (TEAE) - Females

    Safety and tolerability as measured by incidence of treatment emergent adverse events (TEAE), serious TEAEs, and TEAEs experienced by at least 5% of participants in either treatment group

    Time frame: Entire study: approximately 82 weeks. (first dose of study drug until end of Safety Follow-up Visit at 28 +/- 7 days after the last dose (ie, 78-week treatment period plus 4-weeks follow-up after last dose up to total of 82 weeks)

  38. Treatment Emergent Adverse Events (TEAE) - Males

    Safety and tolerability as measured by incidence of treatment emergent adverse events (TEAE), serious TEAEs, and TEAEs experienced by at least 5% of participants in either treatment group

    Time frame: Entire study: approximately 82 weeks. (first dose of study drug until end of Safety Follow-up Visit at 28 +/- 7 days after the last dose (ie, 78-week treatment period plus 4-weeks follow-up after last dose up to total of 82 weeks)

07

Results

Posted Nov 10, 2025

Participant flow

Participant flow — Overall Study
MilestonePlaceboALZ-801
Started162163
Completed148132
Not completed1431
Withdrew: Adverse event211
Withdrew: Withdrawal by subject511
Withdrew: Lost to follow-up43
Withdrew: Not otherwise specified23
Withdrew: Physician decision13

Outcome measures

PrimaryPrimary Cognitive Efficacy Endpoint (ADAS-Cog13)

Change from baseline in Alzheimer's Disease Assessment Scale - Cognitive Subscale 13 (ADAS-Cog13) scores. The ADAS-Cog13 is a rater administered instrument that was designed to assess the severity of the dysfunction in the cognitive and noncognitive behaviors characteristic of persons with AD. The cognitive subscale of the ADAS-Cog13 consists of 13 items assessing areas of cognitive function most typically impaired in AD: orientation, verbal memory, language, praxis, delayed free recall, digit cancellation. The ADAS-Cog13 scale ranges from 0 to 85. Higher scores indicate greater disease severity.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · Score on a scale
Primary Cognitive Efficacy Endpoint (ADAS-Cog13)
Score on a scalePlaceboALZ-801
Primary Cognitive Efficacy Endpoint (ADAS-Cog13)4.40 ± 0.693.89 ± 0.69
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.6058 (Statistical testing was two-sided at the α=0.05 significance level. Between-treatment group comparison (effect in ALZ-801 minus effect in placebo): LSM difference and p-value.) · Least squares mean difference: -0.51ALZ-801 minus placebo
PrimaryIncidence, 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 TEAEs, and TEAEs leading to withdrawal.

Time frame:
Entire study: approximately 82 weeks. (first dose of study drug until end of Safety Follow-up Visit at 28 +/- 7 days after the last dose (ie, 78-week treatment period plus 4-weeks follow-up after last dose up to total of 82 weeks)
Reported as:
Count of participants · Participants
Incidence, Nature, and Severity of Treatment Emergent Adverse Events (TEAE)
ParticipantsPlaceboALZ-801
TEAE137140
Study drug related TEAE4475
Serious TEAE1314
Serious study drug related TEAE01
TEAE leading to discontinuation of study drug413
Study drug related TEAE leading to study drug discontinuation110
SecondaryKey Secondary Endpoint (A-IADL-W)

Change from baseline in Amsterdam - Instrumental Activities of Daily Living scores calculated using the weighted average method (A-IADL-W score). The A-IADL Questionnaire is a 70-item informant-based computerized questionnaire aimed at detecting deficits in complex functions at the early stages of AD. Instrumental ADL can be described as the activities necessary to function independently in society. These activities include, but are not limited to, cooking, doing finances, and shopping. They are complex everyday tasks, determined by multiple cognitive processes and controlled processing. They can be distinguished from basic ADL, which include basic self-care skills. The A-IADL-W has a score range of 0-100 and is calculated as follows: (sum of all scores / number of questions scored) × 25. For A-IADL-W, higher scores indicates worse functioning or more impairment.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
Key Secondary Endpoint (A-IADL-W)
score on a scalePlaceboALZ-801
Key Secondary Endpoint (A-IADL-W)13.59 ± 2.0213.60 ± 2.00
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.9966 (Nominal p-value) · Least squares mean difference: 0.01ALZ-801 minus placebo
SecondaryKey Secondary Endpoint (CDR-SB)

Change from baseline in Clinical Dementia Rating - Sum of Boxes (CDR-SB) scores. CDR-SB is a semi-structured interview of participants and their caregivers. Participant's cognitive status is rated across 6 domains of functioning, including memory, orientation, judgment/problem solving, community affairs, home/hobbies, and personal care. Severity score assigned for each of 6 domains; Total score (SB) ranges from 0 to 18. Higher scores indicate greater disease severity.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
Key Secondary Endpoint (CDR-SB)
score on a scalePlaceboALZ-801
Key Secondary Endpoint (CDR-SB)1.36 ± 0.211.05 ± 0.22
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.3090 (Nominal p-value) · Least squares mean difference: -0.31ALZ-801 minus placebo
SecondaryFunctional Assessment (DAD)

Change from baseline in Disability Assessment for Dementia (DAD)scores. The DAD consists of 40 items with a score range of 0-100 to evaluate the basic and instrumental activities of daily living of subjects with dementia. It is administered through an interview with the caregiver. Higher DAD scores indicate less disability or better function.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
Functional Assessment (DAD)
score on a scalePlaceboALZ-801
Functional Assessment (DAD)-9.2 ± 1.7-6.5 ± 1.7
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.2763 (Nominal p-value) · Least squares mean difference: 2.6ALZ-801 minus placebo
SecondaryGlobal Cognition Assessment (MMSE)

Change from baseline in Mini-Mental State Examination (MMSE) score. The MMSE is a measure of global cognition that is widely used for clinical staging of Alzheimer's Disease. It consists of 11 domains items for a score range of 0-30 to assess general cognitive function. Higher score on MMSE means better cognitive skills.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
Global Cognition Assessment (MMSE)
score on a scalePlaceboALZ-801
Global Cognition Assessment (MMSE)-2.03 ± 0.33-1.60 ± 0.33
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.4484 (Nominal p-value) · Least squares mean difference: 0.35ALZ-801 minus placebo
Other pre-specifiedImaging Biomarker Endpoint (Hippocampal Volume)

Change from baseline in total (bilateral) hippocampal volume (HV) (uL) as measured by Magnetic Resonance Imaging (MRI). HV atrophy is an early event in AD patients, especially in APOE4/4 homozygotes who show accelerated atrophy compared to APOE3/3 patients with Early AD. HV may be a marker of synaptic loss and neurodegeneration.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · microliters
Imaging Biomarker Endpoint (Hippocampal Volume)
microlitersPlaceboALZ-801
Imaging Biomarker Endpoint (Hippocampal Volume)-418.5 ± 21.46-345.0 ± 22.01
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0174 (Nominal p-value) · Least squares mean difference: 73.505ALZ-801 minus placebo.
Other pre-specifiedImaging Biomarker Endpoint (Cortical Thickness [Whole Cortex])

Change from baseline in total (bilateral) cortical thickness (mm) as measured by MRI. Brain MRI studies in Alzheimer's Disease patients show progressive cortical atrophy, reflecting progressive neurodegeneration.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · mm
Imaging Biomarker Endpoint (Cortical Thickness [Whole Cortex])
mmPlaceboALZ-801
Imaging Biomarker Endpoint (Cortical Thickness [Whole Cortex])-0.060 ± 0.003-0.048 ± 0.003
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0020 (Nominal p-value) · Least squares mean difference: 0.012ALZ-801 minus placebo
Other pre-specifiedImaging Biomarker Endpoint (Cortical Thickness [Mayo Index])

Change from baseline in total (bilateral) cortical thickness (mm) as measured by MRI. Brain MRI studies in Alzheimer's Disease patients show progressive cortical atrophy, reflecting progressive neurodegeneration. The Mayo Index refers specifically to the measurement of cortical thickness in the medial temporal lobe.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · mm
Imaging Biomarker Endpoint (Cortical Thickness [Mayo Index])
mmPlaceboALZ-801
Imaging Biomarker Endpoint (Cortical Thickness [Mayo Index])-0.121 ± 0.005-0.099 ± 0.005
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0040 (Nominal p-value) · Least squares mean difference: 0.021ALZ-801 minus placebo
Other pre-specifiedImaging Biomarker Endpoint (Whole Brain Volume)

Change from baseline in whole brain volume (uL) as measured by Magnetic Resonance Imaging (MRI).

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · microliters
Imaging Biomarker Endpoint (Whole Brain Volume)
microlitersPlaceboALZ-801
Imaging Biomarker Endpoint (Whole Brain Volume)-17877.0 ± 955.98-15056.0 ± 979.86
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0400 (Nominal p-value) · Least squares mean difference: 2821.027ALZ-801 minus placebo
Other pre-specifiedImaging Biomarker Endpoint (Ventricular Volume)

Change from baseline in total (bilateral) ventricular volume (uL) as measured by Magnetic Resonance Imaging (MRI).

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · microliters
Imaging Biomarker Endpoint (Ventricular Volume)
microlitersPlaceboALZ-801
Imaging Biomarker Endpoint (Ventricular Volume)5186.3 ± 256.194028.9 ± 262.86
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0018 (Nominal p-value) · Least squares mean difference: -1157
Other pre-specifiedImaging Biomarker Endpoint - DTI in Grey Matter Mean Diffusivity - Bilateral Caudate

Change from baseline in Grey Matter Mean Diffusivity (Bilateral Caudate) as measured by Diffusion Tensor Imaging - Magnetic Resonance Imaging (DTI-MRI). The mean diffusivity (MD) is the average of the three main diffusion values (eigenvalues) obtained from the diffusion tensor imaging (DTI). It is expressed as mm2/s. This unit quantifies the average rate of water diffusion across all directions within a tissue, providing an overall measure of tissue microstructural properties. Lower MD value suggests better maintenance of microstructural integrity of a given brain tissue. Positive treatment effects of ALZ-801 on DTI would present as lower mean diffusivity compared with the placebo group.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · square millimeters per second (mm²/s)
Imaging Biomarker Endpoint - DTI in Grey Matter Mean Diffusivity - Bilateral Caudate
square millimeters per second (mm²/s)PlaceboALZ-801
Imaging Biomarker Endpoint - DTI in Grey Matter Mean Diffusivity - Bilateral Caudate0.000028 ± 0.000007750.000007439 ± 0.00000792
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.054 (Nominal p-value) · Least squares mean difference: -0.0000214 · 95% CI -0.0000432 to 0.00000037ALZ-801 minus placebo
Other pre-specifiedImaging Biomarker Endpoint - DTI in White Matter Mean Diffusivity (Bilateral Fornix)

Change from baseline in Bilateral Fornix White Matter Mean Diffusivity as measured by Diffusion Tensor Imaging - Magnetic Resonance Imaging (DTI-MRI). The mean diffusivity (MD) is the average of the three main diffusion values (eigenvalues) obtained from the diffusion tensor imaging (DTI). It is expressed as mm2/s. This unit quantifies the average rate of water diffusion across all directions within a tissue, providing an overall measure of tissue microstructural properties. Lower MD value suggests better maintenance of microstructural integrity of a given brain tissue. Positive treatment effects of ALZ-801 on DTI would present as lower mean diffusivity compared with the placebo group.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · square millimeters per second (mm²/s)
Imaging Biomarker Endpoint - DTI in White Matter Mean Diffusivity (Bilateral Fornix)
square millimeters per second (mm²/s)PlaceboALZ-801
Imaging Biomarker Endpoint - DTI in White Matter Mean Diffusivity (Bilateral Fornix)0.0000237 ± 0.00000580.000000843 ± 0.00000593
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0064 (Nominal p-value) · Least squares mean difference: -0.0000228 · 95% CI -0.0000391 to -0.0000065
Other pre-specifiedImaging Biomarker Endpoint - White Matter Fractional Anisotropy (Bilateral Fornix)

Change from baseline in Bilateral Fornix White Matter Fractional Anisotropy as measured by Diffusion Tensor Imaging - Magnetic Resonance Imaging (DTI-MRI). Fractional anisotropy (FA) is a unitless, scalar value that measures the degree of anisotropic (directional) water diffusion within a voxel, ranging from 0 to 1. A value of 0 indicates perfectly isotropic (equal in all directions) diffusion, while a value of 1 indicates perfectly anisotropic (directional) diffusion. Higher FA value suggests better maintenance of microstructural integrity of a given brain tissue. Positive treatment effects of ALZ-801 on DTI would present as higher FA in the ALZ-801 group compared with the placebo group.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · Units on a scale (fractions 0-1)
Imaging Biomarker Endpoint - White Matter Fractional Anisotropy (Bilateral Fornix)
Units on a scale (fractions 0-1)PlaceboALZ-801
Imaging Biomarker Endpoint - White Matter Fractional Anisotropy (Bilateral Fornix)-0.0154 ± 0.00332-0.002089 ± 0.00336
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0465 (Nominal p-value) · Least squares mean difference: 0.009455 · 95% CI 0.0001469 to 0.01876
Other pre-specifiedCognitive Efficacy Endpoint (ADAS-Cog 13) - MCI Subgroup

Change from baseline in Alzheimer's Disease Assessment Scale - Cognitive Subscale 13 (ADAS-cog 13) scores at 78 weeks. The ADAS-Cog13 is a rater administered instrument that was designed to assess the severity of the dysfunction in the cognitive and noncognitive behaviors characteristic of persons with AD. The cognitive subscale of the ADAS-Cog13 consists of 13 items assessing areas of cognitive function most typically impaired in AD: orientation, verbal memory, language, praxis, delayed free recall, digit cancellation. The ADAS-Cog13 scale ranges from 0 to 85. Higher scores indicate greater disease severity.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · Score on a scale
Cognitive Efficacy Endpoint (ADAS-Cog 13) - MCI Subgroup
Score on a scalePlaceboALZ-801
Cognitive Efficacy Endpoint (ADAS-Cog 13) - MCI Subgroup4.10 ± 0.811.97 ± 0.83
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0416 (Nominal p-value) · Least squares mean difference: -2.14
Other pre-specifiedCognitive Efficacy Endpoint (ADAS-Cog 13) - Mild Alzheimer's Disease(Mild AD) Subgroup

Change from baseline in Alzheimer's Disease Assessment Scale - Cognitive Subscale 13 (ADAS-cog 13) scores at 78 weeks. The ADAS-Cog13 is a rater administered instrument that was designed to assess the severity of the dysfunction in the cognitive and noncognitive behaviors characteristic of persons with AD. The cognitive subscale of the ADAS-Cog13 consists of 13 items assessing areas of cognitive function most typically impaired in AD: orientation, verbal memory, language, praxis, delayed free recall, digit cancellation. The ADAS-Cog13 scale ranges from 0 to 85. Higher scores indicate greater disease severity.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
Cognitive Efficacy Endpoint (ADAS-Cog 13) - Mild Alzheimer's Disease(Mild AD) Subgroup
score on a scalePlaceboALZ-801
Cognitive Efficacy Endpoint (ADAS-Cog 13) - Mild Alzheimer's Disease(Mild AD) Subgroup4.79 ± 0.755.66 ± 0.75
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.3945 (Nominal p-value) · Least squares mean difference: 0.87
Other pre-specifiedA-IADL-W - MCI Subgroup

Change from baseline in Amsterdam - Instrumental Activities of Daily Living scores calculated using the weighted average method (A-IADL-W score). The A-IADL Questionnaire is a 70-item informant-based computerized questionnaire aimed at detecting deficits in complex functions at the early stages of AD. Instrumental ADL can be described as the activities necessary to function independently in society. These activities include, but are not limited to, cooking, doing finances, and shopping. They are complex everyday tasks, determined by multiple cognitive processes and controlled processing. They can be distinguished from basic ADL, which include basic self-care skills. The A-IADL-W has a score range of 0-100 and is calculated as follows: (sum of all scores / number of questions scored) × 25. For A-IADL-W, higher scores indicates worse functioning or more impairment.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
A-IADL-W - MCI Subgroup
score on a scalePlaceboALZ-801
A-IADL-W - MCI Subgroup4.84 ± 2.451.43 ± 2.50
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.2682 (Nominal p-value) · Least squares mean difference: -3.41
Other pre-specifiedA-IADL-W - Mild AD Subgroup

Change from baseline in Amsterdam - Instrumental Activities of Daily Living scores calculated using the weighted average method (A-IADL-W score). The A-IADL Questionnaire is a 70-item informant-based computerized questionnaire aimed at detecting deficits in complex functions at the early stages of AD. Instrumental ADL can be described as the activities necessary to function independently in society. These activities include, but are not limited to, cooking, doing finances, and shopping. They are complex everyday tasks, determined by multiple cognitive processes and controlled processing. They can be distinguished from basic ADL, which include basic self-care skills. The A-IADL-W has a score range of 0-100 and is calculated as follows: (sum of all scores / number of questions scored) × 25. For A-IADL-W, higher scores indicates worse functioning or more impairment.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
A-IADL-W - Mild AD Subgroup
score on a scalePlaceboALZ-801
A-IADL-W - Mild AD Subgroup18.55 ± 2.2122.20 ± 2.21
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.2178 (Nominal p-value) · Least squares mean difference: 3.66
Other pre-specifiedCDR-SB - MCI Subgroup

Change from baseline in Clinical Dementia Rating - Sum of Boxes (CDR-SB) scores. CDR-SB is a semi-structured interview of participants and their caregivers. Participant's cognitive status is rated across 6 domains of functioning, including memory, orientation, judgment/problem solving, community affairs, home/hobbies, and personal care. Severity score assigned for each of 6 domains; Total score (SB) ranges from 0 to 18. Higher scores indicate greater disease severity.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
CDR-SB - MCI Subgroup
score on a scalePlaceboALZ-801
CDR-SB - MCI Subgroup0.63 ± 0.26-0.02 ± 0.27
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0533 (Nominal p-value) · Least squares mean difference: -0.646
Other pre-specifiedCDR-SB - Mild AD Subgroup

Change from baseline in Clinical Dementia Rating - Sum of Boxes (CDR-SB) scores. CDR-SB is a semi-structured interview of participants and their caregivers. Participant's cognitive status is rated across 6 domains of functioning, including memory, orientation, judgment/problem solving, community affairs, home/hobbies, and personal care. Severity score assigned for each of 6 domains; Total score (SB) ranges from 0 to 18. Higher scores indicate greater disease severity.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
CDR-SB - Mild AD Subgroup
score on a scalePlaceboALZ-801
CDR-SB - Mild AD Subgroup1.84 ± 0.231.97 ± 0.24
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.6854 (Nominal p-value) · Least squares mean difference: 0.127
Other pre-specifiedFunctional Assessment (DAD) - MCI Subgroup

Change from baseline in Disability Assessment for Dementia (DAD)scores. The DAD consists of 40 items with a score range of 0-100 to evaluate the basic and instrumental activities of daily living of subjects with dementia. It is administered through an interview with the caregiver. Higher DAD scores indicate less disability or better function.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
Functional Assessment (DAD) - MCI Subgroup
score on a scalePlaceboALZ-801
Functional Assessment (DAD) - MCI Subgroup-6.3 ± 1.9-0.2 ± 2.0
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0161 (Nominal p-value) · Least squares mean difference: 6.10
Other pre-specifiedFunctional Assessment (DAD) - Mild AD Subgroup

Change from baseline in Disability Assessment for Dementia (DAD)scores. The DAD consists of 40 items with a score range of 0-100 to evaluate the basic and instrumental activities of daily living of subjects with dementia. It is administered through an interview with the caregiver. Higher DAD scores indicate less disability or better function.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
Functional Assessment (DAD) - Mild AD Subgroup
score on a scalePlaceboALZ-801
Functional Assessment (DAD) - Mild AD Subgroup-11.1 ± 1.8-11.6 ± 1.8
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.8387 (Nominal p-value) · Least squares mean difference: -0.51
Other pre-specifiedGlobal Cognition Assessment (MMSE) - MCI Subgroup

Change from baseline in Mini-Mental State Examination (MMSE) score. The MMSE is a measure of global cognition that is widely used for clinical staging of Alzheimer's Disease. It consists of 11 domains items for a score range of 0-30 to assess general cognitive function. Higher score on MMSE means better cognitive skills.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · Score on a scale
Global Cognition Assessment (MMSE) - MCI Subgroup
Score on a scalePlaceboALZ-801
Global Cognition Assessment (MMSE) - MCI Subgroup-1.4 ± 0.4-0.5 ± 0.4
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0795 (Nominal p-value) · Least squares mean difference: 0.9 · 95% CI -0.103 to 1.845
Other pre-specifiedGlobal Cognition Assessment (MMSE) - Mild AD Subgroup

Change from baseline in Mini-Mental State Examination (MMSE) score. The MMSE is a measure of global cognition that is widely used for clinical staging of Alzheimer's Disease. It consists of 11 domains items for a score range of 0-30 to assess general cognitive function. Higher score on MMSE means better cognitive skills.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
Global Cognition Assessment (MMSE) - Mild AD Subgroup
score on a scalePlaceboALZ-801
Global Cognition Assessment (MMSE) - Mild AD Subgroup-2.6 ± 0.4-2.7 ± 0.4
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.8169 (Nominal p-value) · Least squares mean difference: -0.1 · 95% CI -1.070 to 0.845
Other pre-specifiedHippocampal Volume (MCI Subgroup)

Change from baseline in total (bilateral) hippocampal volume (HV) (uL) as measured by Magnetic Resonance Imaging (MRI). HV atrophy is an early event in AD patients, especially in APOE4/4 homozygotes who show accelerated atrophy compared to APOE3/3 patients with Early AD. HV may be a marker of synaptic loss and neurodegeneration.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · microliters
Hippocampal Volume (MCI Subgroup)
microlitersPlaceboALZ-801
Hippocampal Volume (MCI Subgroup)-411.2 ± 29.60-303.1 ± 30.16
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0042 (Nominal p-value) · Least squares mean difference: 108.1
Other pre-specifiedHippocampal Volume (Mild AD Subgroup)

Change from baseline in total (bilateral) hippocampal volume (HV) (uL) as measured by Magnetic Resonance Imaging (MRI). HV atrophy is an early event in AD patients, especially in APOE4/4 homozygotes who show accelerated atrophy compared to APOE3/3 patients with Early AD. HV may be a marker of synaptic loss and neurodegeneration.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · microliters
Hippocampal Volume (Mild AD Subgroup)
microlitersPlaceboALZ-801
Hippocampal Volume (Mild AD Subgroup)-423.2 ± 23.27-371.8 ± 24.75
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.1145 (Nominal p-value) · Least squares mean difference: 51.3
Other pre-specifiedCortical Thickness (Whole Cortex) (MCI Subgroup)

Change from baseline in total (bilateral) cortical thickness (mm) as measured by MRI. Brain MRI studies in Alzheimer's Disease patients show progressive cortical atrophy, reflecting progressive neurodegeneration.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · mm
Cortical Thickness (Whole Cortex) (MCI Subgroup)
mmPlaceboALZ-801
Cortical Thickness (Whole Cortex) (MCI Subgroup)-0.059 ± 0.004-0.038 ± 0.004
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = < 0.0001 (Nominal p-value) · Least squares mean difference: 0.020
Other pre-specifiedCortical Thickness (Whole Cortex) (Mild AD Subgroup)

Change from baseline in total (bilateral) cortical thickness (mm) as measured by MRI. Brain MRI studies in Alzheimer's Disease patients show progressive cortical atrophy, reflecting progressive neurodegeneration.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · mm
Cortical Thickness (Whole Cortex) (Mild AD Subgroup)
mmPlaceboALZ-801
Cortical Thickness (Whole Cortex) (Mild AD Subgroup)-0.061 ± 0.003-0.054 ± 0.003
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0985 (Nominal p-value) · Least squares mean difference: 0.007
Other pre-specifiedCortical Thickness (Mayo Index) (MCI Subgroup)

Change from baseline in total (bilateral) cortical thickness (mm) as measured by MRI. Brain MRI studies in Alzheimer's Disease patients show progressive cortical atrophy, reflecting progressive neurodegeneration. The Mayo Index refers specifically to the measurement of cortical thickness in the medial temporal lobe versus the whole cortex.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · mm
Cortical Thickness (Mayo Index) (MCI Subgroup)
mmPlaceboALZ-801
Cortical Thickness (Mayo Index) (MCI Subgroup)-0.117 ± 0.007-0.084 ± 0.007
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0002 (Nominal p-value) · Least squares mean difference: 0.033
Other pre-specifiedCortical Thickness (Mayo Index) (Mild AD Subgroup)

Change from baseline in total (bilateral) cortical thickness (mm) as measured by MRI. Brain MRI studies in Alzheimer's Disease patients show progressive cortical atrophy, reflecting progressive neurodegeneration. The Mayo Index refers specifically to the measurement of cortical thickness in the medial temporal lobe versus the whole cortex.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · mm
Cortical Thickness (Mayo Index) (Mild AD Subgroup)
mmPlaceboALZ-801
Cortical Thickness (Mayo Index) (Mild AD Subgroup)-0.123 ± 0.006-0.109 ± 0.006
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0699 (Nominal p-value) · Least squares mean difference: 0.014
Other pre-specifiedWhole Brain Volume (MCI Subgroup)

Change from baseline in whole brain volume (uL) as measured by Magnetic Resonance Imaging (MRI).

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · microliters
Whole Brain Volume (MCI Subgroup)
microlitersPlaceboALZ-801
Whole Brain Volume (MCI Subgroup)-17559.5 ± 1417.91-13715.7 ± 1449.14
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0267 (Nominal p-value) · Least squares mean difference: 3843.824
Other pre-specifiedWhole Brain Volume (Mild AD Subgroup)

Change from baseline in whole brain volume (uL) as measured by Magnetic Resonance Imaging (MRI).

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · microliters
Whole Brain Volume (Mild AD Subgroup)
microlitersPlaceboALZ-801
Whole Brain Volume (Mild AD Subgroup)-18081.4 ± 1056.74-15917.1 ± 1143.24
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.1386 (Nominal p-value) · Least squares mean difference: 2164.299
Other pre-specifiedVentricular Volume (MCI Subgroup)

Change from baseline in total (bilateral) ventricular volume (uL) as measured by Magnetic Resonance Imaging (MRI).

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · microliters
Ventricular Volume (MCI Subgroup)
microlitersPlaceboALZ-801
Ventricular Volume (MCI Subgroup)4490.5 ± 343.653178.2 ± 350.44
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0029 (Nominal p-value) · Least squares mean difference: -1312
Other pre-specifiedVentricular Volume (Mild AD Subgroup)

Change from baseline in total (bilateral) ventricular volume (uL) as measured by Magnetic Resonance Imaging (MRI).

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · microliters
Ventricular Volume (Mild AD Subgroup)
microlitersPlaceboALZ-801
Ventricular Volume (Mild AD Subgroup)5630.4 ± 274.914573.4 ± 294.71
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0065 (Nominal p-value) · Least squares mean difference: -1057
Post-hocCerebellar Volume

Change from baseline in total (bilateral) cerebellar volume (uL) as measured by Magnetic Resonance Imaging (MRI).

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · microliters
Cerebellar Volume
microlitersPlaceboALZ-801
Cerebellar Volume-577.0 ± 62.50-427.2 ± 64.70
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.0966 (Nominal p-value) · Least squares mean difference: 149.800
Post-hocCerebellar Volume (MCI Subgroup)

Change from baseline in total (bilateral) cerebellar volume (uL) as measured by Magnetic Resonance Imaging (MRI).

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · microliters
Cerebellar Volume (MCI Subgroup)
microlitersPlaceboALZ-801
Cerebellar Volume (MCI Subgroup)-566.8 ± 91.78-428.8 ± 94.09
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.2200 (Nominal p-value) · Least squares mean difference: 137.943
Post-hocCerebellar Volume (Mild AD Subgroup)

Change from baseline in total (bilateral) cerebellar volume (uL) as measured by Magnetic Resonance Imaging (MRI).

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · microliters
Cerebellar Volume (Mild AD Subgroup)
microlitersPlaceboALZ-801
Cerebellar Volume (Mild AD Subgroup)-583.6 ± 68.62-426.2 ± 75.18
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.1005 (Nominal p-value) · Least squares mean difference: 157.414
Other pre-specifiedCognitive Efficacy Endpoint (ADAS-Cog 13) - Female Subgroup of the FAS

Change from baseline in Alzheimer's Disease Assessment Scale - Cognitive Subscale 13 (ADAS-cog 13) scores at 78 weeks. The ADAS-Cog13 is a rater administered instrument that was designed to assess the severity of the dysfunction in the cognitive and noncognitive behaviors characteristic of persons with AD. The cognitive subscale of the ADAS-Cog13 consists of 13 items assessing areas of cognitive function most typically impaired in AD: orientation, verbal memory, language, praxis, delayed free recall, digit cancellation. The ADAS-Cog13 scale ranges from 0 to 85. Higher scores indicate greater disease severity.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
Cognitive Efficacy Endpoint (ADAS-Cog 13) - Female Subgroup of the FAS
score on a scalePlaceboALZ-801
Cognitive Efficacy Endpoint (ADAS-Cog 13) - Female Subgroup of the FAS4.12 ± 0.894.43 ± 0.91
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.8045 (Nominal p-value) · Least squares mean difference: 0.32
Other pre-specifiedCognitive Efficacy Endpoint (ADAS-Cog 13) - Male Subgroup of the FAS

Change from baseline in Alzheimer's Disease Assessment Scale - Cognitive Subscale 13 (ADAS-cog 13) scores at 78 weeks. The ADAS-Cog13 is a rater administered instrument that was designed to assess the severity of the dysfunction in the cognitive and noncognitive behaviors characteristic of persons with AD. The cognitive subscale of the ADAS-Cog13 consists of 13 items assessing areas of cognitive function most typically impaired in AD: orientation, verbal memory, language, praxis, delayed free recall, digit cancellation. The ADAS-Cog13 scale ranges from 0 to 85. Higher scores indicate greater disease severity.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
Cognitive Efficacy Endpoint (ADAS-Cog 13) - Male Subgroup of the FAS
score on a scalePlaceboALZ-801
Cognitive Efficacy Endpoint (ADAS-Cog 13) - Male Subgroup of the FAS4.74 ± 0.873.72 ± 0.88
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.4114 (Nominal p-value) · Least squares mean difference: -1.02
Other pre-specifiedA-IADL-W - Female Subgroup

Change from baseline in Amsterdam - Instrumental Activities of Daily Living scores calculated using the weighted average method (A-IADL-W score). The A-IADL Questionnaire is a 70-item informant-based computerized questionnaire aimed at detecting deficits in complex functions at the early stages of AD. Instrumental ADL can be described as the activities necessary to function independently in society. These activities include, but are not limited to, cooking, doing finances, and shopping. They are complex everyday tasks, determined by multiple cognitive processes and controlled processing. They can be distinguished from basic ADL, which include basic self-care skills. The A-IADL-W has a score range of 0-100 and is calculated as follows: (sum of all scores / number of questions scored) × 25. For A-IADL-W, higher scores indicates worse functioning or more impairment.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
A-IADL-W - Female Subgroup
score on a scalePlaceboALZ-801
A-IADL-W - Female Subgroup11.52 ± 2.5416.22 ± 2.64
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.2006 (Nominal p-value) · Least squares mean difference: 4.70
Other pre-specifiedA-IADL-W - Male Subgroup

Change from baseline in Amsterdam - Instrumental Activities of Daily Living scores calculated using the weighted average method (A-IADL-W score). The A-IADL Questionnaire is a 70-item informant-based computerized questionnaire aimed at detecting deficits in complex functions at the early stages of AD. Instrumental ADL can be described as the activities necessary to function independently in society. These activities include, but are not limited to, cooking, doing finances, and shopping. They are complex everyday tasks, determined by multiple cognitive processes and controlled processing. They can be distinguished from basic ADL, which include basic self-care skills. The A-IADL-W has a score range of 0-100 and is calculated as follows: (sum of all scores / number of questions scored) × 25. For A-IADL-W, higher scores indicates worse functioning or more impairment.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
A-IADL-W - Male Subgroup
score on a scalePlaceboALZ-801
A-IADL-W - Male Subgroup16.20 ± 2.5612.36 ± 2.56
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.2863 (Nominal p-value) · Least squares mean difference: -3.84
Other pre-specifiedCDR-SB - Female Subgroup

Change from baseline in Clinical Dementia Rating - Sum of Boxes (CDR-SB) scores. CDR-SB is a semi-structured interview of participants and their caregivers. Participant's cognitive status is rated across 6 domains of functioning, including memory, orientation, judgment/problem solving, community affairs, home/hobbies, and personal care. Severity score assigned for each of 6 domains; Total score (SB) ranges from 0 to 18. Higher scores indicate greater disease severity.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
CDR-SB - Female Subgroup
score on a scalePlaceboALZ-801
CDR-SB - Female Subgroup1.09 ± 0.270.95 ± 0.29
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.7278 (Nominal p-value) · Least squares mean difference: -0.14
Other pre-specifiedCDR-SB - Male Subgroup

Change from baseline in Clinical Dementia Rating - Sum of Boxes (CDR-SB) scores. CDR-SB is a semi-structured interview of participants and their caregivers. Participant's cognitive status is rated across 6 domains of functioning, including memory, orientation, judgment/problem solving, community affairs, home/hobbies, and personal care. Severity score assigned for each of 6 domains; Total score (SB) ranges from 0 to 18. Higher scores indicate greater disease severity.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
CDR-SB - Male Subgroup
score on a scalePlaceboALZ-801
CDR-SB - Male Subgroup1.65 ± 0.271.21 ± 0.27
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.2434 (Nominal p-value) · Least squares mean difference: -0.45
Other pre-specifiedFunctional Assessment (DAD) - Female Subgroup

Change from baseline in Disability Assessment for Dementia (DAD)scores. The DAD consists of 40 items with a score range of 0-100 to evaluate the basic and instrumental activities of daily living of subjects with dementia. It is administered through an interview with the caregiver. Higher DAD scores indicate less disability or better function.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
Functional Assessment (DAD) - Female Subgroup
score on a scalePlaceboALZ-801
Functional Assessment (DAD) - Female Subgroup-7.9 ± 2.2-7.1 ± 2.3
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.7995 (Nominal p-value) · Least squares mean difference: 0.8
Other pre-specifiedFunctional Assessment (DAD) - Male Subgroup

Change from baseline in Disability Assessment for Dementia (DAD)scores. The DAD consists of 40 items with a score range of 0-100 to evaluate the basic and instrumental activities of daily living of subjects with dementia. It is administered through an interview with the caregiver. Higher DAD scores indicate less disability or better function.

Time frame:
Baseline to Week 78
Reported as:
Least squares mean · score on a scale
Functional Assessment (DAD) - Male Subgroup
score on a scalePlaceboALZ-801
Functional Assessment (DAD) - Male Subgroup-10.5 ± 2.2-6.5 ± 2.2
Statistical analysis
  • Placebo vs ALZ-801 · MMRM · p = 0.1863 (Nominal p-value) · Least squares mean difference: 4.1
Other pre-specifiedTreatment Emergent Adverse Events (TEAE) - Females

Safety and tolerability as measured by incidence of treatment emergent adverse events (TEAE), serious TEAEs, and TEAEs experienced by at least 5% of participants in either treatment group

Time frame:
Entire study: approximately 82 weeks. (first dose of study drug until end of Safety Follow-up Visit at 28 +/- 7 days after the last dose (ie, 78-week treatment period plus 4-weeks follow-up after last dose up to total of 82 weeks)
Reported as:
Count of participants · Participants
Treatment Emergent Adverse Events (TEAE) - Females
ParticipantsPlaceboALZ-801
TEAE6273
Serious TEAE48
Abdominal pain upper25
Gastrooesophageal reflux disease53
Nausea528
Vomiting114
Fatigue53
COVID-191616
Upper respiratory tract infection25
Urinary tract infection96
Fall56
Weight decreased513
Decreased appetite39
Arthalgia15
Back pain63
Amyloid related imaging abnormality-microhaemorrhages and haemosiderin deposits51
Cerebral microhaemorrhage56
Dizziness25
Headache75
Syncope36
Depression15
Insomnia53
Hypertension55
Other pre-specifiedTreatment Emergent Adverse Events (TEAE) - Males

Safety and tolerability as measured by incidence of treatment emergent adverse events (TEAE), serious TEAEs, and TEAEs experienced by at least 5% of participants in either treatment group

Time frame:
Entire study: approximately 82 weeks. (first dose of study drug until end of Safety Follow-up Visit at 28 +/- 7 days after the last dose (ie, 78-week treatment period plus 4-weeks follow-up after last dose up to total of 82 weeks)
Reported as:
Count of participants · Participants
Treatment Emergent Adverse Events (TEAE) - Males
ParticipantsPlaceboALZ-801
TEAE7567
Serious TEAE96
Nausea314
Fatigue24
COVID-191518
Upper respiratory tract infection62
Urinary tract infection24
Fall64
Weight decreased710
Decreased appetite07
Arthalgia43
Back pain52
Amyloid related imaging abnormality-microhaemorrhages and haemosiderin deposits45
Cerebral microhaemorrhage62
Dizziness74
Headache54
Depression62
Insomnia24
Hypertension42
Diarrhoea43
Agitation43
Tremor41

Adverse events

Collected over All AEs (deaths, Serious Adverse Events, Other Adverse Events) were recorded for up to 95 weeks (13 weeks screening + 78-week treatment + 4 weeks follow-up). All AEs were collected from ICF signing at screening through Safety Follow-up Visit 4 weeks after last efficacy visit at week 78. Medical conditions present at screening were captured as medical history. However, if a condition deteriorated, it was recorded as an AE.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Placebo0/162 (0%)13/162 (8%)94/162 (58%)
ALZ-8010/163 (0%)15/163 (9.2%)104/163 (63.8%)
Most frequent serious events
Showing 10 of 41
Most frequent serious events
EventPlaceboALZ-801
SyncopeNervous system disorders0/1622/163
Angina PectorisCardiac disorders1/1620/163
BradycardiaCardiac disorders1/1620/163
Coronary Artery StenosisCardiac disorders1/1620/163
Myocardial InfarctionCardiac disorders1/1621/163
Intestinal ObstructionGastrointestinal disorders1/1620/163
Chest PainGeneral disorders1/1620/163
COVID-19Infections and infestations1/1620/163
Cholecystitis InfectiveInfections and infestations1/1620/163
Pyelonephritis AcuteInfections and infestations1/1620/163
Most frequent other events
Showing 10 of 15
Most frequent other events
EventPlaceboALZ-801
NauseaGastrointestinal disorders8/16242/163
COVID-19Infections and infestations33/16235/163
Weight DecreasedInvestigations12/16223/163
Decreased AppetiteMetabolism and nutrition disorders3/16216/163
VomitingGastrointestinal disorders2/16216/163
HeadacheNervous system disorders13/1629/163
Urinary Tract InfectionInfections and infestations13/16210/163
Cerebral MicrohaemorrhageNervous system disorders12/1628/163
FallInjury, poisoning and procedural complications11/16210/163
Back painMusculoskeletal and connective tissue disorders11/1625/163

Baseline characteristics

Age, Continuous
Age, Continuous(years)PlaceboALZ-801Total
Mean68.5 ± 5.9368.4 ± 6.3668.5 ± 6.14
Age, Customized
Age, Customized(Participants)PlaceboALZ-801Total
50 to <=65464995
>65 to 80116114230
Sex: Female, Male
Sex: Female, Male(Participants)PlaceboALZ-801Total
Female8285167
Male8078158
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)PlaceboALZ-801Total
American Indian or Alaska Native000
Asian123
Black or African American6713
Native Hawaiian or Other Pacific Islander000
White145144289
Multiple Races112
Race Not Reported/Missing9918
Hispanic/Latino7512
Not Hispanic/Latino141147288
Ethnicity Not Reported141125
Alzheimer's Diagnosis based on MMSE
Alzheimer's Diagnosis based on MMSE(Participants)PlaceboALZ-801Total
Mild cognitive impairment (MCI) MMSE 27-306067127
Mild Alzheimer's Disease (Mild AD) MMSE 22-2610296198
Concomitant AChEI
Concomitant AChEI(Participants)PlaceboALZ-801Total
Yes6254116
No100109209
Baseline Clinical Outcomes Assessments (Safety Population)
Baseline Clinical Outcomes Assessments (Safety Population)(units on a scale)PlaceboALZ-801Total
ADAS-Cog1324.31 ± 8.8623.554 ± 8.2623.93 ± 8.56
A-IADL-W17.46 ± 20.6516.00 ± 18.4216.73 ± 19.55
CDR-SB2.97 ± 1.453.04 ± 1.533.00 ± 1.49
DAD93.0 ± 10.3493.3 ± 9.9493.2 ± 10.13
MMSE25.1 ± 2.725.5 ± 2.725.3 ± 2.7
08

Study locations

95 sites
  • Xenoscience
    Phoenix, Arizona 85004, United States
  • CCT Research
    Scottsdale, Arizona 85297, United States
  • Banner Sun Health Research Institute
    Sun City, Arizona 85351, United States
  • ATP Clinical Research
    Costa Mesa, California 92626, United States
  • Tilda Research
    Irvine, California 92780, United States
  • UCSD Shiley-Marcos Alzheimer's Disease Research Center
    La Jolla, California 92037, United States
  • Torrance Clinical Research Institute
    Lomita, California 90717, United States
  • Collaborative NeuroScience Network LLC
    Long Beach, California 90806, United States
  • Stanford University
    Palo Alto, California 94304, United States
  • SC3 Research Group
    Pasadena, California 91105, United States
  • Sutter Health
    Sacramento, California 95816, United States
  • JEM Research Institute, Headlands Site
    Atlantis, Florida 33462, United States
  • Bradenton Research Center
    Bradenton, Florida 34205, United States
  • Galiz Research
    Hialeah, Florida 33016, United States
  • Alphab Global Research
    Jupiter, Florida 33458, United States
  • Charter Research
    Lady Lake, Florida 32159, United States
  • K2 Medical Research, LLC
    Maitland, Florida 32751, United States
  • Miami Jewish Health
    Miami, Florida 33137, United States
  • Y & L Advance Health Care, Inc /DBA Elite Clinical Research
    Miami, Florida 33144, United States
  • Mount Sinai Medical Center
    Miami Beach, Florida 33140, United States
  • Aqualane Clinical Research
    Naples, Florida 34105, United States
  • Charter Research
    Orlando, Florida 32803, United States
  • Headlands Research Orlando
    Orlando, Florida 32819, United States
  • Advanced Research Consultants
    Palm Beach Gardens, Florida 33410, United States
  • Quantum CNS Clinical Research
    Pompano Beach, Florida 33064, United States
  • Progressive Medical Research
    Port Orange, Florida 32127, United States
  • ALZ Research and Treatment Center (A.R.T.C.)
    Stuart, Florida 34997, United States
  • ALZ Research and Treatment Center (A.R.T.C.)
    Wellington, Florida 33414, United States
  • Premiere Research Institute
    West Palm Beach, Florida 33407, United States
  • Columbus Memory Center
    Columbus, Georgia 31909, United States
  • Sandhill Research, LLC
    Decatur, Georgia 30030, United States
  • Fort Wayne Neurological Center
    Fort Wayne, Indiana 46804, United States
  • Louisiana State University Health Sciences Center (LSUHSC)
    Shreveport, Louisiana 71103, United States
  • Headlands Research Eastern MA
    Plymouth, Massachusetts 02360, United States
  • WR-CRCN
    Las Vegas, Nevada 89106, United States
  • Advanced Memory Research Center
    Toms River, New Jersey 08755, United States
  • Neurological Associates of Albany
    Albany, New York 12208, United States
  • NYU Alzheimer's Disease Research Center
    New York, New York 10016, United States
  • Kline Institute for Psychiatric Research
    Orangeburg, New York 10962, United States
  • University of Rochester Medical Center
    Rochester, New York 14620, United States
  • Richmond Behavioral Associates
    Staten Island, New York 10312, United States
  • Triad Clinical Trials
    Greensboro, North Carolina 27410, United States
  • AMC Research
    Matthews, North Carolina 28105, United States
  • NeuroScience Research Center
    Canton, Ohio 44718, United States
  • Neurology Diagnostics, Inc.
    Dayton, Ohio 45459, United States
  • IPS Research
    Oklahoma City, Oklahoma 73106, United States
  • Center for Cognitive Health
    Portland, Oregon 97225, United States
  • Abington Neurological Associates
    Abington, Pennsylvania 19001, United States
  • Lehigh Center for Clinical Research
    Allentown, Pennsylvania 18104, United States
  • Rhode Island Mood & Memory Research Institute
    East Providence, Rhode Island 02914, United States
  • Vanderbilt Center for Cognitive Medicine
    Nashville, Tennessee 37212, United States
  • UT Health Science Center at Houston
    Houston, Texas 77054, United States
  • Re:Cognition Health
    Fairfax, Virginia 22031, United States
  • Northwest Clinical Research Center
    Bellevue, Washington 98007, United States
  • Medical College of Wisconsin
    Milwaukee, Wisconsin 53226, United States
  • OCT Research ULC (dba Okanagan Clinical Trials)
    Kelowna, British Columbia V1Y 1Z9, Canada
  • Centricity Research
    Halifax, Nova Scotia B3S 1N2, Canada
  • Centricity Research
    New Minas, Nova Scotia B4N 3R7, Canada
  • Recherches Neuro-Hippocampe Inc., d/b/a Ottawa Memory Clinic
    Ottawa, Ontario K1Z 1G3, Canada
  • Kawartha Centre - Redefining Healthy Aging
    Peterborough, Ontario K9H 2P4, Canada
  • Toronto Memory Program
    Toronto, Ontario M3B 2S7, Canada
  • Recherches Neuro-Hippocampe Inc. d/b/a Clinique de la Mémoire de l'Outaouais
    Gatineau, Quebec J8T 8J1, Canada
  • Medica 111, Spol. s r.o. - Neurologicka a Rehabilitacni Ambulance Brno, Centralni Pracoviste v Brne
    Brno, 602 00, Czechia
  • Fakultní nemocnice u sv. Anny v Brně (St. Anne's University Hospital)
    Brno, 656 91, Czechia
  • Neuropsychiatrie s.r.o.
    Prague, 160 00, Czechia
  • Vestra Clinics - Dedicated Research Clinics
    Rychnov nad Kněžnou, 51601, Czechia
  • Centre Hospitalier Universitaire de Marseille - Hôpital de la Timone
    Marseille, 13385, France
  • Hopital Gui de Chauliac - CHRU de Montpellier
    Montpellier, 43431, France
  • Hopital Lariboisiere - Fernand-Widal - AP-HP
    Paris, 75475, France
  • Centre Hospitalier Universitaire (CHU) Hopitaux de Rouen
    Rouen, 76031, France
  • Hopitaux Universitaires de Strasbourg Centre d'Investigation Clinique
    Strasbourg, 67200, France
  • Centre de Recherche Clinique du Gerontopole - CHU Toulouse
    Toulouse, 31059, France
  • Centre de Recherche-Hopital Geriatrique de Charpennes
    Villeurbanne, 69100, France
  • University Hospital RWTH Aachen Neurological Study Center
    Aachen, 52074, Germany
  • Klinikum der Universitaet Muenchen Innenstadt
    München, 80336, Germany
  • Universitätsklinik fuer Psychiatrie und Psychotherapie
    Tübingen, 72076, Germany
  • Studienzentrum Nord-West
    Westerstede, 26655, Germany
  • Memory Clinic, Landspitali University Hospital
    Reykjavik, 101, Iceland
  • Brain Research Center Den Bosch
    's-Hertogenbosch, 5223 LA, Netherlands
  • Brain Research Center Amsterdam
    Amsterdam, 1081 GN, Netherlands
  • Brain Research Center Zwolle
    Zwolle, 8025 AZ, Netherlands
  • Fundacia ACE - Institut Catala de Neurociencies Aplicadas
    Barcelona, 08028, Spain
  • Clinica Universidad de Navarra
    Madrid, 28027, Spain
  • Hospital Clinico San Carlos
    Madrid, 28040, Spain
  • Clinica Universidad de Navarra
    Pamplona, 31008, Spain
  • Hospital Universitari MutuaTerrassa
    Terrassa, 08221, Spain
  • Centro Hospital Universitario Dr. Preset
    Valencia, 46017, Spain
  • Re-Cognition Health Ltd Plymouth
    Plymouth, Devon PL6 8BT, United Kingdom
  • Re-Cognition Health Ltd London
    London, Greater London W1G 9JF, United Kingdom
  • NeuroClin Glasgow Ltd
    Motherwell, North Lanarkshire ML1 4UF, United Kingdom
  • Re-Cognition Health Ltd Guildford
    Guildford, Surrey GU2 7YD, United Kingdom
  • Re-Cognition Health Ltd Birmingham
    Birmingham, West Midlands B16 8LT, United Kingdom
  • Re:Cognition Health Ltd Bristol
    Bristol, BS32 4SY, United Kingdom
  • St. Pancras Clinical Research
    London, EC2Y 8EA, United Kingdom
  • Re-Cognition Health Ltd Winchester
    Winchester, SO21 1HU, United Kingdom
09

References and documents

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 ↗
  • Abushakra S, Power A, Watson D, Porsteinsson A, Sabbagh M, MacSweeney E, Cohen S, Boada Rovira M, Doraiswamy PM, Liang E, Flint S, Kesslak JP, McLaine R, Albayrak A, Schaefer J, Yu J, Tolar L, Dickson S, Hey JA, Tolar M. Clinical Efficacy, Safety and Imaging Effects of Oral Valiltramiprosate in APOEepsilon4/epsilon4 Homozygotes with Early Alzheimer's Disease: Results of the Phase III, Randomized, Double-Blind, Placebo-Controlled, 78-Week APOLLOE4 Trial. Drugs. 2025 Nov;85(11):1455-1472. doi: 10.1007/s40265-025-02250-5. Epub 2025 Sep 28. PubMed 41015981 ↗

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

10

Timeline

1 registry update since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from May 2021 to primary completion in May 2024, 36 months.

Results were first posted in Nov 2025, 1 year 5 months after primary completion. A results delay was requested in May 2025.

Of 75 industry-sponsored phase 3 interventional studies under Alzheimer Disease, half reached primary completion within 42 months of starting (the median), counting those still running; a quarter within 28 and three quarters within 61. This study's 36 months is below that.

Counting every sponsor, half of 101 studies reached it within 47 months; a quarter within 28 and three quarters within 73.

How these figures are worked out, and which studies are left out of them.

Registered
Feb 22, 2021
First posted
Feb 25, 2021
StartActual
May 19, 2021
Primary completionActual
May 29, 2024
CompletionActual
Jul 29, 2024
Results delay requested
May 8, 2025
Results first posted
Nov 10, 2025
Last update
Sep 29, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Results
Posted results revised
revised Sep 29, 2026
Publications
1 publication citing this study linked from PubMed, e.g. PubMed 42678603 ↗
Sep 29, 2026
Secondary outcomes
10 added
What changed in the outcomes
  • Addedother outcomeCognitive Efficacy Endpoint (ADAS-Cog 13) - Female Subgroup of the FASTime frame: Baseline to Week 78
  • Addedother outcomeCognitive Efficacy Endpoint (ADAS-Cog 13) - Male Subgroup of the FASTime frame: Baseline to Week 78
  • Addedother outcomeA-IADL-W - Female SubgroupTime frame: Baseline to Week 78
  • Addedother outcomeA-IADL-W - Male SubgroupTime frame: Baseline to Week 78
  • Addedother outcomeCDR-SB - Female SubgroupTime frame: Baseline to Week 78
  • Addedother outcomeCDR-SB - Male SubgroupTime frame: Baseline to Week 78
  • Addedother outcomeFunctional Assessment (DAD) - Female SubgroupTime frame: Baseline to Week 78
  • Addedother outcomeFunctional Assessment (DAD) - Male SubgroupTime frame: Baseline to Week 78
  • Addedother outcomeTreatment Emergent Adverse Events (TEAE) - FemalesTime frame: Entire study: approximately 82 weeks. (first dose of study drug until end of Safety Follow-up Visit at 28 +/- 7 days after the last dose (ie, 78-week treatment period plus 4-weeks follow-up after last dose up to total of 82 weeks)
  • Addedother outcomeTreatment Emergent Adverse Events (TEAE) - MalesTime frame: Entire study: approximately 82 weeks. (first dose of study drug until end of Safety Follow-up Visit at 28 +/- 7 days after the last dose (ie, 78-week treatment period plus 4-weeks follow-up after last dose up to total of 82 weeks)
Sep 29, 2026
Show all 1 update
  1. Sep 29, 2026
    Posted results revised
    Publications 1 publication citing this study linked from PubMed, e.g. PubMed 42678603 ↗
    Secondary outcomes 10 added
    What changed in the outcomes
    • Addedother outcomeCognitive Efficacy Endpoint (ADAS-Cog 13) - Female Subgroup of the FASTime frame: Baseline to Week 78
    • Addedother outcomeCognitive Efficacy Endpoint (ADAS-Cog 13) - Male Subgroup of the FASTime frame: Baseline to Week 78
    • Addedother outcomeA-IADL-W - Female SubgroupTime frame: Baseline to Week 78
    • Addedother outcomeA-IADL-W - Male SubgroupTime frame: Baseline to Week 78
    • Addedother outcomeCDR-SB - Female SubgroupTime frame: Baseline to Week 78
    • Addedother outcomeCDR-SB - Male SubgroupTime frame: Baseline to Week 78
    • Addedother outcomeFunctional Assessment (DAD) - Female SubgroupTime frame: Baseline to Week 78
    • Addedother outcomeFunctional Assessment (DAD) - Male SubgroupTime frame: Baseline to Week 78
    • Addedother outcomeTreatment Emergent Adverse Events (TEAE) - FemalesTime frame: Entire study: approximately 82 weeks. (first dose of study drug until end of Safety Follow-up Visit at 28 +/- 7 days after the last dose (ie, 78-week treatment period plus 4-weeks follow-up after last dose up to total of 82 weeks)
    • Addedother outcomeTreatment Emergent Adverse Events (TEAE) - MalesTime frame: Entire study: approximately 82 weeks. (first dose of study drug until end of Safety Follow-up Visit at 28 +/- 7 days after the last dose (ie, 78-week treatment period plus 4-weeks follow-up after last dose up to total of 82 weeks)
    + 1 other change: verification date

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

11

Registry details

Key details

Study ID
NCT04770220
Lead sponsor
Alzheon Inc.
Collaborators
National Institute on Aging (NIA)
Responsible party
Sponsor
First posted
Feb 25, 2021
Start date
May 19, 2021
Primary completion
May 29, 2024
Completion
Jul 29, 2024
Results posted
Nov 10, 2025
Last update
Sep 29, 2026

Study contacts

Susan Abushakra, MD
principal investigator · Alzheon Inc.

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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Studies of the drug tested here: ALZ-801

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