# Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study - COVID supplement

> **NIH NIH R01** · ALZHEON, INC. · 2022 · $3,500,000

## Abstract

Project Summary
We are developing ALZ-801 (also known as valiltramiprosate), an oral brain-penetrant small molecule that
inhibits the formation of toxic amyloid oligomers. With support from NIA through R01-AG065253, we are
conducting 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
(NCT04770220). This study (ALZ-801-AD301, APOLLOE4) evaluates the efficacy, safety, fluid biomarkers, and
MRI effects of ALZ-801 265 mg bid or placebo in 300 patients over 78 weeks (150/arm). The study is being
conducted in the US, Canada, Iceland, United Kingdom, Czech Republic, Netherlands, Germany, France, and
Spain. A major clinical research organization (CRO), ICON plc, is contracted to manage the study.
Study start-up commenced in July 2020, in the middle of the first year of the COVID-19 pandemic and the first
subject was randomized in June 2021. The original goal for the study was to complete randomization within 18
months (i.e., by October 2022) with a goal of completing the study by May 2024. Due to the complications of the
pandemic, the study has faced many complications that have increased both the cost and duration of the study:
• many sites initially chosen for the study were unable to participate or were severely delayed in participation
 due to COVID as many sites were unable to conduct on-site visits or were unable to see potential new
 subjects for evaluation and recruitment – as a result an additional 14 sites are being brought to site initiation
 and a new country (Czech Republic) was added to the study
• especially at some of the larger institutions, administrative support was focused on COVID studies and
 contracting was for other studies was severely delayed
• enhancement or initiation of alternate strategies for recruitment were employed to address the very limitation
 application of traditional, local outreach strategies
• supply chain issues have lately had a significant impact, especially on the provision of laboratory supplies,
 particularly kits to support screening activities causing some sites to pause screening activities
The sum of these challenges has resulted in a delay of approximately two months in recruitment with subsequent
increases in project management. This delay would likely have been significantly longer without the increase in
investment already applied to the study by the sponsor.

## Key facts

- **NIH application ID:** 10610578
- **Project number:** 3R01AG065253-03S1
- **Recipient organization:** ALZHEON, INC.
- **Principal Investigator:** Susan Abushakra
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $3,500,000
- **Award type:** 3
- **Project period:** 2020-08-15 → 2025-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10610578

## Citation

> US National Institutes of Health, RePORTER application 10610578, Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study - COVID supplement (3R01AG065253-03S1). Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/grant/nih/10610578. Licensed CC0.

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