NCT04770220

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

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled Study of the Efficacy, Safety and Biomarker Effects of ALZ-801 in Subjects With Early Alzheimer's Disease and APOE4/4 Genotype

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 325 (actual)
• Sponsor: Alzheon Inc. · Industry
• Sex: All
• Age: 50 Years – 80 Years
• Healthy volunteers: Not accepted

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.

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.

Conditions

• Early Alzheimer's Disease

Interventions

Type	Name	Description
DRUG	Experimental: ALZ-801	ALZ-801 tablet 265 mg once daily in the evening for the first 2 weeks, then ALZ-801 tablet 265 mg BID
DRUG	Placebo Comparator: Placebo	Placebo tablet BID

Timeline

• Start date: 2021-05-19
• Primary completion: 2024-05-29
• Completion: 2024-07-29
• First posted: 2021-02-25
• Last updated: 2025-11-10
• Results posted: 2025-11-10

Locations

95 sites across 9 countries: United States, Canada, Czechia, France, Germany, Iceland, Netherlands, Spain, United Kingdom

Regulatory

• FDA-regulated drug study