Phase 3
N=325
An Efficacy and Safety Study of ALZ-801 in APOE4/4 Early AD Subjects
Early Alzheimer's Disease
Bottom Line
View on ClinicalTrials.gov: NCT04770220 ↗Enrolled (actual)
325
Serious AEs
8.6%
Results posted
Nov 2025
Primary outcome: Primary: Primary Cognitive Efficacy Endpoint (ADAS-Cog13) — 4.40; 3.89 Score on a scale — p=0.6058
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Experimental: ALZ-801 (Drug); Placebo Comparator: Placebo (Drug)
- Age
- Adult, Older Adult · 50+ yrs
- Sex
- All
- Sponsor
- Alzheon Inc.
- Primary completion
- May 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Primary Cognitive Efficacy Endpoint (ADAS-Cog13) |
4.40; 3.89 | 0.6058 |
| PRIMARY Incidence, Nature, and Severity of Treatment Emergent Adverse Events (TEAE) |
137; 140; 44; 75; 13; 14 | — |
| SECONDARY Key Secondary Endpoint (A-IADL-W) |
13.59; 13.60 | 0.9966 |
| SECONDARY Key Secondary Endpoint (CDR-SB) |
1.36; 1.05 | 0.3090 |
| SECONDARY Functional Assessment (DAD) |
-9.2; -6.5 | 0.2763 |
| SECONDARY Global Cognition Assessment (MMSE) |
-2.03; -1.60 | 0.4484 |
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.
Eligibility Criteria
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).
- MMSE 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
- 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 transient ischemic attack within one year prior to screening.
Data sourced from ClinicalTrials.gov (NCT04770220). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.