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

NCT ID: NCT04770220

Last Updated: 2025-11-10

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

325 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-05-19

Study Completion Date

2024-07-29

Brief Summary

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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

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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

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Early Alzheimer's Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

This is a multi-center Phase 3, randomized, double-blind, placebo-controlled, parallel-group study.
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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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).

Group Type EXPERIMENTAL

Experimental: ALZ-801

Intervention Type DRUG

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

Placebo Comparator: Placebo

Intervention Type DRUG

Placebo tablet BID

Placebo

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

Group Type PLACEBO_COMPARATOR

Placebo Comparator: Placebo

Intervention Type DRUG

Placebo tablet BID

Interventions

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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

Intervention Type DRUG

Placebo Comparator: Placebo

Placebo tablet BID

Intervention Type DRUG

Other Intervention Names

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valiltramiprosate tramiprosate prodrug

Eligibility Criteria

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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.
Minimum Eligible Age

50 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Institute on Aging (NIA)

NIH

Sponsor Role collaborator

Alzheon Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Susan Abushakra, MD

Role: PRINCIPAL_INVESTIGATOR

Alzheon Inc.

Locations

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Xenoscience

Phoenix, Arizona, United States

Site Status

CCT Research

Scottsdale, Arizona, United States

Site Status

Banner Sun Health Research Institute

Sun City, Arizona, United States

Site Status

ATP Clinical Research

Costa Mesa, California, United States

Site Status

Tilda Research

Irvine, California, United States

Site Status

UCSD Shiley-Marcos Alzheimer's Disease Research Center

La Jolla, California, United States

Site Status

Torrance Clinical Research Institute

Lomita, California, United States

Site Status

Collaborative NeuroScience Network LLC

Long Beach, California, United States

Site Status

Stanford University

Palo Alto, California, United States

Site Status

SC3 Research Group

Pasadena, California, United States

Site Status

Sutter Health

Sacramento, California, United States

Site Status

JEM Research Institute, Headlands Site

Atlantis, Florida, United States

Site Status

Bradenton Research Center

Bradenton, Florida, United States

Site Status

Galiz Research

Hialeah, Florida, United States

Site Status

Alphab Global Research

Jupiter, Florida, United States

Site Status

Charter Research

Lady Lake, Florida, United States

Site Status

K2 Medical Research, LLC

Maitland, Florida, United States

Site Status

Miami Jewish Health

Miami, Florida, United States

Site Status

Y & L Advance Health Care, Inc /DBA Elite Clinical Research

Miami, Florida, United States

Site Status

Mount Sinai Medical Center

Miami Beach, Florida, United States

Site Status

Aqualane Clinical Research

Naples, Florida, United States

Site Status

Charter Research

Orlando, Florida, United States

Site Status

Headlands Research Orlando

Orlando, Florida, United States

Site Status

Advanced Research Consultants

Palm Beach Gardens, Florida, United States

Site Status

Quantum CNS Clinical Research

Pompano Beach, Florida, United States

Site Status

Progressive Medical Research

Port Orange, Florida, United States

Site Status

ALZ Research and Treatment Center (A.R.T.C.)

Stuart, Florida, United States

Site Status

ALZ Research and Treatment Center (A.R.T.C.)

Wellington, Florida, United States

Site Status

Premiere Research Institute

West Palm Beach, Florida, United States

Site Status

Columbus Memory Center

Columbus, Georgia, United States

Site Status

Sandhill Research, LLC

Decatur, Georgia, United States

Site Status

Fort Wayne Neurological Center

Fort Wayne, Indiana, United States

Site Status

Louisiana State University Health Sciences Center (LSUHSC)

Shreveport, Louisiana, United States

Site Status

Headlands Research Eastern MA

Plymouth, Massachusetts, United States

Site Status

WR-CRCN

Las Vegas, Nevada, United States

Site Status

Advanced Memory Research Center

Toms River, New Jersey, United States

Site Status

Neurological Associates of Albany

Albany, New York, United States

Site Status

NYU Alzheimer's Disease Research Center

New York, New York, United States

Site Status

Kline Institute for Psychiatric Research

Orangeburg, New York, United States

Site Status

University of Rochester Medical Center

Rochester, New York, United States

Site Status

Richmond Behavioral Associates

Staten Island, New York, United States

Site Status

Triad Clinical Trials

Greensboro, North Carolina, United States

Site Status

AMC Research

Matthews, North Carolina, United States

Site Status

NeuroScience Research Center

Canton, Ohio, United States

Site Status

Neurology Diagnostics, Inc.

Dayton, Ohio, United States

Site Status

IPS Research

Oklahoma City, Oklahoma, United States

Site Status

Center for Cognitive Health

Portland, Oregon, United States

Site Status

Abington Neurological Associates

Abington, Pennsylvania, United States

Site Status

Lehigh Center for Clinical Research

Allentown, Pennsylvania, United States

Site Status

Rhode Island Mood & Memory Research Institute

East Providence, Rhode Island, United States

Site Status

Vanderbilt Center for Cognitive Medicine

Nashville, Tennessee, United States

Site Status

UT Health Science Center at Houston

Houston, Texas, United States

Site Status

Re:Cognition Health

Fairfax, Virginia, United States

Site Status

Northwest Clinical Research Center

Bellevue, Washington, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

OCT Research ULC (dba Okanagan Clinical Trials)

Kelowna, British Columbia, Canada

Site Status

Centricity Research

Halifax, Nova Scotia, Canada

Site Status

Centricity Research

New Minas, Nova Scotia, Canada

Site Status

Recherches Neuro-Hippocampe Inc., d/b/a Ottawa Memory Clinic

Ottawa, Ontario, Canada

Site Status

Kawartha Centre - Redefining Healthy Aging

Peterborough, Ontario, Canada

Site Status

Toronto Memory Program

Toronto, Ontario, Canada

Site Status

Recherches Neuro-Hippocampe Inc. d/b/a Clinique de la Mémoire de l'Outaouais

Gatineau, Quebec, Canada

Site Status

Medica 111, Spol. s r.o. - Neurologicka a Rehabilitacni Ambulance Brno, Centralni Pracoviste v Brne

Brno, , Czechia

Site Status

Fakultní nemocnice u sv. Anny v Brně (St. Anne's University Hospital)

Brno, , Czechia

Site Status

Neuropsychiatrie s.r.o.

Prague, , Czechia

Site Status

Vestra Clinics - Dedicated Research Clinics

Rychnov nad Kněžnou, , Czechia

Site Status

Centre Hospitalier Universitaire de Marseille - Hôpital de la Timone

Marseille, , France

Site Status

Hopital Gui de Chauliac - CHRU de Montpellier

Montpellier, , France

Site Status

Hopital Lariboisiere - Fernand-Widal - AP-HP

Paris, , France

Site Status

Centre Hospitalier Universitaire (CHU) Hopitaux de Rouen

Rouen, , France

Site Status

Hopitaux Universitaires de Strasbourg Centre d'Investigation Clinique

Strasbourg, , France

Site Status

Centre de Recherche Clinique du Gerontopole - CHU Toulouse

Toulouse, , France

Site Status

Centre de Recherche-Hopital Geriatrique de Charpennes

Villeurbanne, , France

Site Status

University Hospital RWTH Aachen Neurological Study Center

Aachen, , Germany

Site Status

Klinikum der Universitaet Muenchen Innenstadt

München, , Germany

Site Status

Universitätsklinik fuer Psychiatrie und Psychotherapie

Tübingen, , Germany

Site Status

Studienzentrum Nord-West

Westerstede, , Germany

Site Status

Memory Clinic, Landspitali University Hospital

Reykjavik, , Iceland

Site Status

Brain Research Center Den Bosch

's-Hertogenbosch, , Netherlands

Site Status

Brain Research Center Amsterdam

Amsterdam, , Netherlands

Site Status

Brain Research Center Zwolle

Zwolle, , Netherlands

Site Status

Fundacia ACE - Institut Catala de Neurociencies Aplicadas

Barcelona, , Spain

Site Status

Clinica Universidad de Navarra

Madrid, , Spain

Site Status

Hospital Clinico San Carlos

Madrid, , Spain

Site Status

Clinica Universidad de Navarra

Pamplona, , Spain

Site Status

Hospital Universitari MutuaTerrassa

Terrassa, , Spain

Site Status

Centro Hospital Universitario Dr. Preset

Valencia, , Spain

Site Status

Re-Cognition Health Ltd Plymouth

Plymouth, Devon, United Kingdom

Site Status

Re-Cognition Health Ltd London

London, Greater London, United Kingdom

Site Status

NeuroClin Glasgow Ltd

Motherwell, North Lanarkshire, United Kingdom

Site Status

Re-Cognition Health Ltd Guildford

Guildford, Surrey, United Kingdom

Site Status

Re-Cognition Health Ltd Birmingham

Birmingham, West Midlands, United Kingdom

Site Status

Re:Cognition Health Ltd Bristol

Bristol, , United Kingdom

Site Status

St. Pancras Clinical Research

London, , United Kingdom

Site Status

Re-Cognition Health Ltd Winchester

Winchester, , United Kingdom

Site Status

Countries

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United States Canada Czechia France Germany Iceland Netherlands Spain United Kingdom

References

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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 Sep 28. doi: 10.1007/s40265-025-02250-5. Online ahead of print.

Reference Type DERIVED
PMID: 41015981 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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R01AG065253

Identifier Type: NIH

Identifier Source: secondary_id

View Link

2020-005755-20

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ALZ-801-AD301

Identifier Type: -

Identifier Source: org_study_id