MedPath

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

Phase 3
Completed
Conditions
Early Alzheimer's Disease
Registration Number
NCT04770220
Lead Sponsor
Alzheon Inc.
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.

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.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
325
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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Primary Outcome Measures
NameTimeMethod
Primary cognitive efficacy endpointWeek 78

Change from baseline in Alzheimer's Disease Assessment Scale - Cognitive Subscale 13 (ADAS-cog 13) scores at 78 weeks

Incidence, Nature, and Severity of Treatment Emergent Adverse events (TEAE)Week 78

Safety and tolerability as measured by incidence, nature and severity of treatment emergent adverse events (TEAE), serious TEAE, and TEAE leading to withdrawal.

Secondary Outcome Measures
NameTimeMethod
Functional assessment 1Week 78

Change from baseline in Amsterdam - Instrumental Activities of Daily Living scores

Global assessmentWeek 78

Change from baseline in Clinical Dementia Rating - Sum of Boxes (CDR-SB) scores

Functional assessment 2Week 78

Change from baseline in Disability Assessment for Dementia (DAD)scores

Trial Locations

Locations (91)

Brain Research Center Zwolle

🇳🇱

Zwolle, Netherlands

Clinica Universidad de Navarra

🇪🇸

Pamplona, Spain

Hospital Clinico San Carlos

🇪🇸

Madrid, Spain

Hospital Universitari MutuaTerrassa

🇪🇸

Terrassa, Spain

Centro Hospital Universitario Dr. Preset

🇪🇸

Valencia, Spain

Re:Cognition Health Ltd Bristol

🇬🇧

Bristol, United Kingdom

St. Pancras Clinical Research

🇬🇧

London, United Kingdom

Neuropsychiatrie s.r.o.

🇨🇿

Praha, Czechia

Brain Research Center Den Bosch

🇳🇱

Den Bosch, Netherlands

Tilda Research

🇺🇸

Irvine, California, United States

Sandhill Research, LLC

🇺🇸

Decatur, Georgia, United States

WR-CRCN

🇺🇸

Las Vegas, Nevada, United States

Neurology Diagnostics, Inc.

🇺🇸

Dayton, Ohio, United States

Vanderbilt Center for Cognitive Medicine

🇺🇸

Nashville, Tennessee, United States

Xenoscience

🇺🇸

Phoenix, Arizona, United States

CCT Research

🇺🇸

Scottsdale, Arizona, United States

Banner Sun Health Research Institute

🇺🇸

Sun City, Arizona, United States

ATP Clinical Research

🇺🇸

Costa Mesa, California, United States

UCSD Shiley-Marcos Alzheimer's Disease Research Center

🇺🇸

La Jolla, California, United States

Torrance Clinical Research Institute

🇺🇸

Lomita, California, United States

Collaborative NeuroScience Network LLC

🇺🇸

Long Beach, California, United States

Stanford University

🇺🇸

Palo Alto, California, United States

SC3 Research Group

🇺🇸

Pasadena, California, United States

Sutter Health

🇺🇸

Sacramento, California, United States

JEM Research Institute, Headlands Site

🇺🇸

Atlantis, Florida, United States

Bradenton Research Center

🇺🇸

Bradenton, Florida, United States

Galiz Research

🇺🇸

Hialeah, Florida, United States

Alphab Global Research

🇺🇸

Jupiter, Florida, United States

Charter Research

🇺🇸

Orlando, Florida, United States

K2 Medical Research, LLC

🇺🇸

Maitland, Florida, United States

Mount Sinai Medical Center

🇺🇸

Miami Beach, Florida, United States

Miami Jewish Health

🇺🇸

Miami, Florida, United States

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

🇺🇸

Miami, Florida, United States

Aqualane Clinical Research

🇺🇸

Naples, Florida, United States

Headlands Research Orlando

🇺🇸

Orlando, Florida, United States

Advanced Research Consultants

🇺🇸

Palm Beach Gardens, Florida, United States

Quantum CNS Clinical Research

🇺🇸

Pompano Beach, Florida, United States

Progressive Medical Research

🇺🇸

Port Orange, Florida, United States

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

🇺🇸

Wellington, Florida, United States

Premiere Research Institute

🇺🇸

West Palm Beach, Florida, United States

Columbus Memory Center

🇺🇸

Columbus, Georgia, United States

Fort Wayne Neurological Center

🇺🇸

Fort Wayne, Indiana, United States

Louisiana State University Health Sciences Center (LSUHSC)

🇺🇸

Shreveport, Louisiana, United States

Headlands Research Eastern MA

🇺🇸

Plymouth, Massachusetts, United States

Advanced Memory Research Center

🇺🇸

Toms River, New Jersey, United States

Neurological Associates of Albany

🇺🇸

Albany, New York, United States

NYU Alzheimer's Disease Research Center

🇺🇸

New York, New York, United States

Kline Institute for Psychiatric Research

🇺🇸

Orangeburg, New York, United States

University of Rochester Medical Center

🇺🇸

Rochester, New York, United States

Richmond Behavioral Associates

🇺🇸

Staten Island, New York, United States

Triad Clinical Trials

🇺🇸

Greensboro, North Carolina, United States

AMC Research

🇺🇸

Matthews, North Carolina, United States

NeuroScience Research Center

🇺🇸

Canton, Ohio, United States

IPS Research

🇺🇸

Oklahoma City, Oklahoma, United States

Center for Cognitive Health

🇺🇸

Portland, Oregon, United States

Abington Neurological Associates

🇺🇸

Abington, Pennsylvania, United States

Lehigh Center for Clinical Research

🇺🇸

Allentown, Pennsylvania, United States

Rhode Island Mood & Memory Research Institute

🇺🇸

East Providence, Rhode Island, United States

UT Health Science Center at Houston

🇺🇸

Houston, Texas, United States

Re:Cognition Health

🇺🇸

Fairfax, Virginia, United States

Northwest Clinical Research Center

🇺🇸

Bellevue, Washington, United States

Medical College of Wisconsin

🇺🇸

Milwaukee, Wisconsin, United States

OCT Research ULC (dba Okanagan Clinical Trials)

🇨🇦

Kelowna, British Columbia, Canada

Centricity Research

🇨🇦

New Minas, Nova Scotia, Canada

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

🇨🇦

Ottawa, Ontario, Canada

Kawartha Centre - Redefining Healthy Aging

🇨🇦

Peterborough, Ontario, Canada

Toronto Memory Program

🇨🇦

Toronto, Ontario, Canada

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

🇨🇦

Gatineau, Quebec, Canada

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

🇨🇿

Brno, Czechia

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

🇨🇿

Brno, Czechia

Centre Hospitalier Universitaire de Marseille - Hôpital de la Timone

🇫🇷

Marseille, France

Vestra Clinics - Dedicated Research Clinics

🇨🇿

Rychnov Nad Kněžnou, Czechia

Hopital Gui de Chauliac - CHRU de Montpellier

🇫🇷

Montpellier Cedex 5, France

Hopital Lariboisiere - Fernand-Widal - AP-HP

🇫🇷

Paris Cedex 10, France

Centre Hospitalier Universitaire (CHU) Hopitaux de Rouen

🇫🇷

Rouen, France

Hopitaux Universitaires de Strasbourg Centre d'Investigation Clinique

🇫🇷

Strasbourg, France

Centre de Recherche Clinique du Gerontopole - CHU Toulouse

🇫🇷

Toulouse, France

Centre de Recherche-Hopital Geriatrique de Charpennes

🇫🇷

Villeurbanne, France

University Hospital RWTH Aachen Neurological Study Center

🇩🇪

Aachen, Germany

Klinikum der Universitaet Muenchen Innenstadt

🇩🇪

Muenchen, Germany

Universitätsklinik fuer Psychiatrie und Psychotherapie

🇩🇪

Tuebingen, Germany

Studienzentrum Nord-West

🇩🇪

Westerstede, Germany

Memory Clinic, Landspitali University Hospital

🇮🇸

Reykjavík, Iceland

Brain Research Center Amsterdam

🇳🇱

Amsterdam, Netherlands

Fundacia ACE - Institut Catala de Neurociencies Aplicadas

🇪🇸

Barcelona, Spain

Re-Cognition Health Ltd Plymouth

🇬🇧

Plymouth, Devon, United Kingdom

Re-Cognition Health Ltd London

🇬🇧

London, Greater London, United Kingdom

NeuroClin Glasgow Ltd

🇬🇧

Motherwell, North Lanarkshire, United Kingdom

Re-Cognition Health Ltd Guildford

🇬🇧

Guildford, Surrey, United Kingdom

Re-Cognition Health Ltd Birmingham

🇬🇧

Birmingham, West Midlands, United Kingdom

Re-Cognition Health Ltd Winchester

🇬🇧

Winchester, United Kingdom

© Copyright 2025. All Rights Reserved by MedPath