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

NCT04770220 | Completed Phase 3 Results DMC FDA Drug

• 3 other identifiers: ALZ-801-AD301R01AG0652532020-005755-20
• Study Type: interventional
• Target: 325
• Locations: 9 countries
• Sites: 95

Brief 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.

Conditions

Early Alzheimer's Disease

Outcome Measures

Primary Outcomes (2)

• 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.

  Baseline to Week 78

• 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.

  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 (4)

• Key Secondary Endpoint (A-IADL-W)

  Baseline to Week 78

• Key Secondary Endpoint (CDR-SB)

  Baseline to Week 78

• Functional Assessment (DAD)

  Baseline to Week 78

• Global Cognition Assessment (MMSE)

  Baseline to Week 78

Other Outcomes (28)

• Imaging Biomarker Endpoint (Hippocampal Volume)

  Baseline to Week 78

• Imaging Biomarker Endpoint (Cortical Thickness [Whole Cortex])

  Baseline to Week 78

• Imaging Biomarker Endpoint (Cortical Thickness [Mayo Index])

  Baseline to Week 78

Study Arms (2)

ALZ-801

EXPERIMENTAL

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

PLACEBO COMPARATOR

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

Drug: Placebo Comparator: Placebo

Interventions

Experimental: ALZ-801 DRUG

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

ALZ-801

Placebo Comparator: Placebo DRUG

Placebo tablet BID

ALZ-801; Placebo

Eligibility Criteria

• Age: 50 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alzheon Inc.: lead
• National Institute on Aging (NIA): collaborator

Study Sites (95)

Xenoscience

Phoenix, Arizona, 85004, United States

Location

CCT Research

Scottsdale, Arizona, 85297, United States

Location

Banner Sun Health Research Institute

Sun City, Arizona, 85351, United States

Location

ATP Clinical Research

Costa Mesa, California, 92626, United States

Location

Tilda Research

Irvine, California, 92780, United States

Location

UCSD Shiley-Marcos Alzheimer's Disease Research Center

La Jolla, California, 92037, United States

Location

Torrance Clinical Research Institute

Lomita, California, 90717, United States

Location

Collaborative NeuroScience Network LLC

Long Beach, California, 90806, United States

Location

Stanford University

Palo Alto, California, 94304, United States

Location

SC3 Research Group

Pasadena, California, 91105, United States

Location

Sutter Health

Sacramento, California, 95816, United States

Location

JEM Research Institute, Headlands Site

Atlantis, Florida, 33462, United States

Location

Bradenton Research Center

Bradenton, Florida, 34205, United States

Location

Galiz Research

Hialeah, Florida, 33016, United States

Location

Alphab Global Research

Jupiter, Florida, 33458, United States

Location

Charter Research

Lady Lake, Florida, 32159, United States

Location

K2 Medical Research, LLC

Maitland, Florida, 32751, United States

Location

Miami Jewish Health

Miami, Florida, 33137, United States

Location

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

Miami, Florida, 33144, United States

Location

Mount Sinai Medical Center

Miami Beach, Florida, 33140, United States

Location

Aqualane Clinical Research

Naples, Florida, 34105, United States

Location

Charter Research

Orlando, Florida, 32803, United States

Location

Headlands Research Orlando

Orlando, Florida, 32819, United States

Location

Advanced Research Consultants

Palm Beach Gardens, Florida, 33410, United States

Location

Quantum CNS Clinical Research

Pompano Beach, Florida, 33064, United States

Location

Progressive Medical Research

Port Orange, Florida, 32127, United States

Location

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

Stuart, Florida, 34997, United States

Location

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

Wellington, Florida, 33414, United States

Location

Premiere Research Institute

West Palm Beach, Florida, 33407, United States

Location

Columbus Memory Center

Columbus, Georgia, 31909, United States

Location

Sandhill Research, LLC

Decatur, Georgia, 30030, United States

Location

Fort Wayne Neurological Center

Fort Wayne, Indiana, 46804, United States

Location

Louisiana State University Health Sciences Center (LSUHSC)

Shreveport, Louisiana, 71103, United States

Location

Headlands Research Eastern MA

Plymouth, Massachusetts, 02360, United States

Location

WR-CRCN

Las Vegas, Nevada, 89106, United States

Location

Advanced Memory Research Center

Toms River, New Jersey, 08755, United States

Location

Neurological Associates of Albany

Albany, New York, 12208, United States

Location

NYU Alzheimer's Disease Research Center

New York, New York, 10016, United States

Location

Kline Institute for Psychiatric Research

Orangeburg, New York, 10962, United States

Location

University of Rochester Medical Center

Rochester, New York, 14620, United States

Location

Richmond Behavioral Associates

Staten Island, New York, 10312, United States

Location

Triad Clinical Trials

Greensboro, North Carolina, 27410, United States

Location

AMC Research

Matthews, North Carolina, 28105, United States

Location

NeuroScience Research Center

Canton, Ohio, 44718, United States

Location

Neurology Diagnostics, Inc.

Dayton, Ohio, 45459, United States

Location

IPS Research

Oklahoma City, Oklahoma, 73106, United States

Location

Center for Cognitive Health

Portland, Oregon, 97225, United States

Location

Abington Neurological Associates

Abington, Pennsylvania, 19001, United States

Location

Lehigh Center for Clinical Research

Allentown, Pennsylvania, 18104, United States

Location

Rhode Island Mood & Memory Research Institute

East Providence, Rhode Island, 02914, United States

Location

Vanderbilt Center for Cognitive Medicine

Nashville, Tennessee, 37212, United States

Location

UT Health Science Center at Houston

Houston, Texas, 77054, United States

Location

Re:Cognition Health

Fairfax, Virginia, 22031, United States

Location

Northwest Clinical Research Center

Bellevue, Washington, 98007, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

OCT Research ULC (dba Okanagan Clinical Trials)

Kelowna, British Columbia, V1Y 1Z9, Canada

Location

Centricity Research

Halifax, Nova Scotia, B3S 1N2, Canada

Location

Centricity Research

New Minas, Nova Scotia, B4N 3R7, Canada

Location

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

Ottawa, Ontario, K1Z 1G3, Canada

Location

Kawartha Centre - Redefining Healthy Aging

Peterborough, Ontario, K9H 2P4, Canada

Location

Toronto Memory Program

Toronto, Ontario, M3B 2S7, Canada

Location

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

Gatineau, Quebec, J8T 8J1, Canada

Location

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

Brno, 602 00, Czechia

Location

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

Brno, 656 91, Czechia

Location

Neuropsychiatrie s.r.o.

Prague, 160 00, Czechia

Location

Vestra Clinics - Dedicated Research Clinics

Rychnov nad Kněžnou, 51601, Czechia

Location

Centre Hospitalier Universitaire de Marseille - Hôpital de la Timone

Marseille, 13385, France

Location

Hopital Gui de Chauliac - CHRU de Montpellier

Montpellier, 43431, France

Location

Hopital Lariboisiere - Fernand-Widal - AP-HP

Paris, 75475, France

Location

Centre Hospitalier Universitaire (CHU) Hopitaux de Rouen

Rouen, 76031, France

Location

Hopitaux Universitaires de Strasbourg Centre d'Investigation Clinique

Strasbourg, 67200, France

Location

Centre de Recherche Clinique du Gerontopole - CHU Toulouse

Toulouse, 31059, France

Location

Centre de Recherche-Hopital Geriatrique de Charpennes

Villeurbanne, 69100, France

Location

University Hospital RWTH Aachen Neurological Study Center

Aachen, 52074, Germany

Location

Klinikum der Universitaet Muenchen Innenstadt

München, 80336, Germany

Location

Universitätsklinik fuer Psychiatrie und Psychotherapie

Tübingen, 72076, Germany

Location

Studienzentrum Nord-West

Westerstede, 26655, Germany

Location

Memory Clinic, Landspitali University Hospital

Reykjavik, 101, Iceland

Location

Brain Research Center Den Bosch

's-Hertogenbosch, 5223 LA, Netherlands

Location

Brain Research Center Amsterdam

Amsterdam, 1081 GN, Netherlands

Location

Brain Research Center Zwolle

Zwolle, 8025 AZ, Netherlands

Location

Fundacia ACE - Institut Catala de Neurociencies Aplicadas

Barcelona, 08028, Spain

Location

Clinica Universidad de Navarra

Madrid, 28027, Spain

Location

Hospital Clinico San Carlos

Madrid, 28040, Spain

Location

Clinica Universidad de Navarra

Pamplona, 31008, Spain

Location

Hospital Universitari MutuaTerrassa

Terrassa, 08221, Spain

Location

Centro Hospital Universitario Dr. Preset

Valencia, 46017, Spain

Location

Re-Cognition Health Ltd Plymouth

Plymouth, Devon, PL6 8BT, United Kingdom

Location

Re-Cognition Health Ltd London

London, Greater London, W1G 9JF, United Kingdom

Location

NeuroClin Glasgow Ltd

Motherwell, North Lanarkshire, ML1 4UF, United Kingdom

Location

Re-Cognition Health Ltd Guildford

Guildford, Surrey, GU2 7YD, United Kingdom

Location

Re-Cognition Health Ltd Birmingham

Birmingham, West Midlands, B16 8LT, United Kingdom

Location

Re:Cognition Health Ltd Bristol

Bristol, BS32 4SY, United Kingdom

Location

St. Pancras Clinical Research

London, EC2Y 8EA, United Kingdom

Location

Re-Cognition Health Ltd Winchester

Winchester, SO21 1HU, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 41015981 DERIVED

MeSH Terms

Conditions

Alzheimer Disease

Condition Hierarchy (Ancestors)

Dementia; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tauopathies; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders

Results Point of Contact

• Title: Aidan Power
• Organization: Alzheon

aidan.power@alzheon.com

(508) 861-7709

Study Officials

• Susan Abushakra, MD

  Alzheon Inc.

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is a multi-center Phase 3, randomized, double-blind, placebo-controlled, parallel-group study.

Study Record Dates

• First Submitted: February 22, 2021
• First Posted: February 25, 2021
• Study Start: May 19, 2021
• Primary Completion: May 29, 2024
• Study Completion: July 29, 2024
• Last Updated: November 10, 2025
• Results First Posted: November 10, 2025

Record last verified: 2025-10

Locations

FR (7); DE (4); CZ (4); IC (1); NL (3); ES (6); GB (8); US (55); CA (7)