NCT05531526

Brief Summary

This AR1001-ADP3-US01 protocol is a double-blind, randomized, placebo-controlled, multi- center, parallel-group comparison pivotal Phase 3 study to evaluate the efficacy and safety of AR1001 for the treatment of participants with early AD.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network
Enrollment
1,535

participants targeted

Target at P75+ for phase_3 alzheimer-disease

Timeline
19mo left

Started Dec 2022

Longer than P75 for phase_3 alzheimer-disease

Geographic Reach
13 countries

198 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress68%
Dec 2022Dec 2027

First Submitted

Initial submission to the registry

August 31, 2022

Completed
8 days until next milestone

First Posted

Study publicly available on registry

September 8, 2022

Completed
4 months until next milestone

Study Start

First participant enrolled

December 23, 2022

Completed
3.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2026

Completed
1.6 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2027

Expected
Last Updated

October 2, 2025

Status Verified

September 1, 2025

Enrollment Period

3.4 years

First QC Date

August 31, 2022

Last Update Submit

September 29, 2025

Conditions

Keywords

AR1001ADEarly AD

Outcome Measures

Primary Outcomes (1)

  • Clinical Dementia Rating Scale - Sum of Boxes (CDR-SB)

    Change in the CDR-SB from Baseline to Week 52

    52 weeks

Secondary Outcomes (4)

  • Alzheimer's Disease Assessment Scale, cognitive subscale (ADAS-Cog 13)

    52 weeks

  • Amsterdam-Instrumental Activities of Daily Living Questionnaire-Short Version (A-IADL-Q-SV)

    52 weeks

  • Geriatric Depression Scale (GDS)

    52 weeks

  • Mini-Mental Status Examination (MMSE)

    52 weeks

Other Outcomes (3)

  • Safety Analysis

    156 weeks

  • Biomarker Analysis

    156 weeks

  • Exploratory Analysis

    156 weeks

Study Arms (2)

Group A - Active Comparator

ACTIVE COMPARATOR

Active, AR1001 30 mg QD will be administered daily for 52 weeks during the Treatment Phase of the study. In the Extension Phase, all eligible participants who choose to participate will receive AR1001 30 mg QD for 52 weeks.

Drug: AR1001

Group B - Placebo Comparator

PLACEBO COMPARATOR

Placebo QD will be administered daily for 52 weeks during the Treatment Phase of the study. In the Extension Phase, all eligible participants who choose to participate will receive AR1001 30 mg QD for 52 weeks.

Drug: Placebo

Interventions

AR1001DRUG

AR1001 Active Oral Tablet

Group A - Active Comparator

Placebo Oral Tablet

Group B - Placebo Comparator

Eligibility Criteria

Age55 Years - 90 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Male or female participants aged 55 to 90 years of age inclusive at the time of signing the informed consent form
  • Mild cognitive impairment or mild dementia consistent with AD defined by stages 3 to 4 according to the National Institute on Aging and Alzheimer's Association (NIA-AA) at Screening
  • Participants with a history of subjective cognitive and memory decline with onset within 5 years before Screening, confirmed by study partner.
  • Participants who have a MMSE score greater than or equal to 20
  • Participants with a CDR global rating of 0.5 or 1
  • Participants with a RBANS score based on the Delayed Memory Index (DMI) score less than or equal to 85
  • If an historic magnetic resonance imaging (MRI) is available, findings must exclude other causes of dementia.
  • Positive biomarker for brain amyloid pathology as indicated by assessment of at least one of the following:
  • Current or historical CSF assessment with FDA-cleared assays, including Lumipulse® beta-amyloid ratio \[1-42/1-40\] ≤ 0.072, Elecsys® pTau 181/Aβ\[1-42\] greater than 0.023, Elecsys® tTau /Aβ\[1-42\] greater than 0.28, or other assays or cut-offs as they become FDA-cleared.
  • Historical amyloid positron emission tomography (PET) assessment confirmed by the Sponsor or Designee.
  • Participants (or participant's legally authorized representative) and caregiver (s) who can sign an informed consent to participate in the study.
  • Participants who have one (or more) identified adult study partners (s) who, in the opinion of the Investigator, has sufficient contact with and knowledge about the participant as to be able to report knowledgably about the participant's cognition, function, behavior, and safety, and compliance with the protocol. The informant/care partner must be available by phone to provide information to the Investigator and study staff about the participant as well as agree to attend in-person clinic visits that require partner input for scale completion. The informant/care partner must be literate and provide informed consent and should be available for the duration of the study. The same informant/care partner is required to be consistent across all study visits except under rare, unavoidable circumstances (e.g., unexpected informant health crisis) that are approved by the Investigator and Sponsor.

You may not qualify if:

  • Participants who are female and are either pregnant, nursing, or of childbearing potential and not practicing effective contraception
  • Participants who have signs of significant delirium which, in the opinion of the Investigator, would interfere with this study
  • Participants who have any diagnosis of dementia or cognitive decline other than that related to AD, including, but not limited to concomitant history of significant head trauma, alcohol abuse, frontotemporal dementia, Huntington Disease, Parkinsonism (e.g., Parkinson's disease, Dementia with Lewy Bodies, etc.), significant cerebrovascular disease, and/or significant seizure disorder
  • Participants with any current psychiatric diagnosis if, in the judgment of the Investigator, the psychiatric disorder (e.g., schizophrenia) or symptom is likely to confound interpretation of drug effect, affect cognitive assessments, or affect the participant's ability to complete the study
  • Participants with a history of vascular dementia
  • Participants with evidence of other neurological conditions thought to interfere with the evaluations in this study
  • Participants with a history of myocardial infarction, unstable angina, coronary artery disease, and/or New York Heart Association (NYHA) class III or IV heart failure within the last 12 months
  • Participants with uncontrolled hypertension (systolic blood pressure (BP) \>160 mmHg or diastolic BP \> 95 mmHg) or hypotension (systolic BP \<90 mmHg or diastolic BP \<50 mmHg). Participants may undergo repeated testing to ensure that accurate BP readings are obtained
  • Participants with a body mass index (BMI) \> 35 kg/m2
  • Participants with any of the following:
  • elevation (\>2.5x upper limit of normal \[ULN\]) of AST (aspartate aminotransferase, ALT (alanine transaminase, or total bilirubin (unless known prior history of Gilbert's syndrome)
  • deficiency (\< lower limit of normal \[LLN\]) of Vitamin B12
  • known history of HIV (human immunodeficiency virus) positivity or positive test for HIV 1/2 at screening
  • known history of Hepatitis C virus (HCV) or positive test for HCV antibody (HCVAb) at screening (unless negative on confirmatory PCR test)'
  • positive test for Hepatitis B surface antigen (HBsAg)
  • +21 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (198)

IMA Clinical Research Phoenix

Phoenix, Arizona, 85012, United States

Location

Perseverance Research Center, LLC

Scottsdale, Arizona, 85253, United States

Location

Clinical Endpoints - N. Scottsdale

Scottsdale, Arizona, 85258, United States

Location

Banner Sun Health Research Institute

Sun City, Arizona, 85351, United States

Location

Voyage Medical

Tempe, Arizona, 85282, United States

Location

Sutter Health's Palo Alto Medical Foundation

Burlingame, California, 94010, United States

Location

Neuro-Pain Medical Center

Fresno, California, 93710, United States

Location

Fullerton Neurology and Headache Center

Fullerton, California, 92835, United States

Location

Esperanza Clinical

Murrieta, California, 92562, United States

Location

Valley Clinical Trials, INC

Northridge, California, 91325, United States

Location

Havana Research Institute

Pasadena, California, 91105, United States

Location

Kaizen Brain Center

San Diego, California, 92037, United States

Location

Adaptive Research

San Jose, California, 95124, United States

Location

The Neuron Clinic

San Marcos, California, 92069, United States

Location

Stanford Neuroscience Health Center

Stanford, California, 94305, United States

Location

Denver Neurological Research

Denver, Colorado, 80210, United States

Location

Mile High Research Center

Denver, Colorado, 80218, United States

Location

CenExel Rocky Mountain Clinical Research

Englewood, Colorado, 80113, United States

Location

Topaz Clinical Research

Apopka, Florida, 32803, United States

Location

BayCare Health System, Inc

Clearwater, Florida, 33756, United States

Location

Vertex Research Group

Clermont, Florida, 34711, United States

Location

Arrow Clinical Trial

Daytona Beach, Florida, 32117, United States

Location

Brainstorm Research - Loxahatchee

Loxahatchee Groves, Florida, 33470, United States

Location

ClinCloud LLC

Maitland, Florida, 32751, United States

Location

Verus Clinical Research, Corp

Miami, Florida, 33135, United States

Location

Vitae Research Center, LLC

Miami, Florida, 33135, United States

Location

Caro Medcenter and Community Research

Miami, Florida, 33145, United States

Location

Allied Biomedical Research Institute, Inc

Miami, Florida, 33155, United States

Location

Future Life Clinical Trials

Miami, Florida, 33169, United States

Location

Brainstorm Research

Miami, Florida, 33176, United States

Location

Meridian International Research, Inc

Miami Gardens, Florida, 33014, United States

Location

Charter Research - Orlando

Orlando, Florida, 32803, United States

Location

K2 Medical Research

Orlando, Florida, 32806, United States

Location

JEM Research Institute

Palm Beach, Florida, 33462, United States

Location

Progressive Medical Research

Port Orange, Florida, 32127, United States

Location

Angels Clinical Research Institute, Inc. - Tampa

Tampa, Florida, 33614, United States

Location

Charter Research - The Villages

The Villages, Florida, 32162, United States

Location

ClinCloud, LLC Melbourn

Viera, Florida, 32940, United States

Location

Conquest Research

Winter Park, Florida, 32789, United States

Location

iResearch -Atlanta

Atlanta, Georgia, 30030, United States

Location

Atlanta Neuroscience Institute

Atlanta, Georgia, 30327, United States

Location

Accel Research Sites

Decatur, Georgia, 30030, United States

Location

iResearch

Savannah, Georgia, 31405, United States

Location

Re:Cognition Health - Chicago

Chicago, Illinois, 60611, United States

Location

Tandem Clinical Research

New Orleans, Louisiana, 70072, United States

Location

BTC of New Bedford

New Bedford, Massachusetts, 02740, United States

Location

Boston Center for Memory

Newton, Massachusetts, 02459, United States

Location

Headlands Research - Eastern MA

Plymouth, Massachusetts, 02360, United States

Location

Boston Paincare

Waltham, Massachusetts, 02451, United States

Location

Sharlin Health Neuroscience Research Center

Ozark, Missouri, 65721, United States

Location

Alivation

Lincoln, Nebraska, 68526, United States

Location

Wake Research- Clinical Research Center of Nevada, LLC

Las Vegas, Nevada, 89106, United States

Location

Advanced Memory Research Institute of New Jersey

Toms River, New Jersey, 08755, United States

Location

Dent Neuroscience Research Center

Amherst, New York, 14226, United States

Location

Mid-Hudson Medical Research, PLLC - New Windsor

New Windsor, New York, 12553, United States

Location

Triad Clinical Trials

Greensboro, North Carolina, 27410, United States

Location

AMC Research, LLC

Matthews, North Carolina, 28105, United States

Location

Accellacare of Winston-Salem, Triad Neurological Associates

Winston-Salem, North Carolina, 27103, United States

Location

NeuroScience Research Center, LLC

Canton, Ohio, 44718, United States

Location

American Clinical Research Institute, LLC

Dayton, Ohio, 45432, United States

Location

Rhode Island Mood and Memory REsearch

East Providence, Rhode Island, 02914, United States

Location

Health Concepts

Rapid City, South Dakota, 57702, United States

Location

Neurology Clinic, P.C.

Cordova, Tennessee, 38018, United States

Location

Center for Biomedical Research, LLC - Genesis Neuroscience Clinic

Knoxville, Tennessee, 37909, United States

Location

Gadolin Research LLC

Beaumont, Texas, 77702, United States

Location

Kerwin Medical Center

Dallas, Texas, 75231, United States

Location

Cognition Health Corporation- Texas

Houston, Texas, 77030, United States

Location

Clinical Trial Network - Houston

Houston, Texas, 77074, United States

Location

Bhupesh Dihenia, MD, PA

Lubbock, Texas, 79410, United States

Location

Wasatch Clinical Research, LLC

Salt Lake City, Utah, 84107, United States

Location

Cognition Health Corporation

Fairfax, Virginia, 22031, United States

Location

Integrated Neurology Services

Falls Church, Virginia, 22043, United States

Location

Kingfisher Cooperative

Spokane, Washington, 99201, United States

Location

Vaught Neurological Services, PLLC

Crab Orchard, West Virginia, 25827, United States

Location

Medical College of Wisconsin

Wauwatosa, Wisconsin, 53226, United States

Location

Centricity Research - Halifax

Halifax, Canada

Location

Okanagan Clinical Trials

Kelowna, Canada

Location

Parkwood Institute

London, Canada

Location

Baycrest Academy for Research and Education

North York, Canada

Location

Kawartha Centre - Redefining Healthy Aging

Peterborough, Canada

Location

Centricity Research

Toronto, Canada

Location

Sunnybrook Health Sciences Centre

Toronto, Canada

Location

University Health Network-Toronto Western Hospital

Toronto, Canada

Location

Royal Jubilee Hospital

Victoria, Canada

Location

Medical Arts Health Research Group - West Vancouver

West Vancouver, Canada

Location

Beijing Anding Hospital Capital Medical University

Beijing, China

Location

Beijing Friendship Hospital,Capital Medical University

Beijing, China

Location

Beijing Tiantan Hospital, Capital Medical University

Beijing, China

Location

Peking Union Medical College Hospital

Beijing, China

Location

Peking University Sixth Hospital

Beijing, China

Location

Xuanwu Hospital Capital Medical University

Beijing, China

Location

The First Hospital of Jilin University

Changchun, China

Location

Xiangya Hospital of Central South University

Changsha, China

Location

The First Affiliated Hospital of Chongqing Medical University

Chongqing, China

Location

Guangdong Provincial People's Hospital

Guangzhou, China

Location

The Affiliated Brain Hospital Of Guangzhou Medical University

Guangzhou, China

Location

The First Affiliated Hospital, Sun Yat-sen University

Guangzhou, China

Location

Anhui Provincial Hospital

Hefei, China

Location

Tianjin Medical University General Hospital

Heping, China

Location

The Second Affiliated Hospital of Nanchang University

Nanchang, China

Location

Nanjing Drum Tower Hospital

Nanjing, China

Location

The Second Affiliated Hospital of Nanjing Medical University

Nanjing, China

Location

Huashan Hospital affiliated to Fudan University

Shanghai, China

Location

Renji Hospital, Shanghai Jiao Tong University School of Medicine

Shanghai, China

Location

The First Hospital of Hebei Medical University

Shijiazhuang, China

Location

The First Affiliated Hospital of Soochow University

Suzhou, China

Location

The Second Affiliated Hospital of Soochow University

Suzhou, China

Location

Tongji Hospital Of Tongji University

Wuhan, China

Location

Xuzhou Central Hospital

Xuzhou, China

Location

Fakultni nemocnice u sv. Anny v Brne

Brno, Czechia

Location

NeuroHK s.r.o.

Choceň, Czechia

Location

Fakultni nemocnice Hradec Kralove

Hradec Králové, Czechia

Location

Ambulance Smrkova ulice (A-Shine s.r.o.)

Pilsen, Czechia

Location

FORBELI s.r.o

Prague, Czechia

Location

INEP Medical s.r.o.

Prague, Czechia

Location

Neuropsychiatrie s.r.o.

Prague, Czechia

Location

Vestra Clinics

Rychnov nad Kněžnou, 516 01, Czechia

Location

Sanos Clinic - Gandrup

Gandrup, 9362, Denmark

Location

Sanos Clinic - Herlev

Herlev, Denmark

Location

Sanos Clinic Syddanmark - Vejle

Vejle, Denmark

Location

CHU de Lille - Hopital Roger Salengro

Lille, France

Location

AP-HM - Hopital de la Timone

Marseille, France

Location

CHU de Nantes - Hopital Nord Laennec

Nantes, France

Location

AP-HP Hopital Broca

Paris, France

Location

AP-HP Hopital Lariboisiere

Paris, France

Location

AP-HP Hopital Pitie-Salpetriere

Paris, France

Location

CHU Rennes - Hopital Pontchaillou

Rennes, 35033, France

Location

CHRU de Strasbourg - Hopital de Hautepierre

Strasbourg, 67200, France

Location

CHU de Toulouse - Hopital La Grave

Toulouse, France

Location

HCL - Hopital des Charpennes

Villeurbanne, France

Location

Universitaetsklinikum Aachen, AoeR

Aachen, Germany

Location

Zentrum fuer klinische Forschung Dr. med. I. Schoell

Bad Homburg, Germany

Location

Charite Universitaetsmedizin Berlin

Berlin, Germany

Location

Bezirkskrankenhaus Guenzburg

Günzburg, Germany

Location

Universitaetsklinikum des Saarlandes

Homburg, Germany

Location

Institut fuer Studien zur Psychischen Gesundheit (ISPG)

Mannheim, 68165, Germany

Location

Azienda Ospedaliero-Universitaria delle Marche

Ancona, Italy

Location

IRCCS Istituto Centro San Giovanni di Dio Fatebenefratelli

Brescia, Italy

Location

ASST Grande Ospedale Metropolitano Niguarda

Milan, Italy

Location

IRCCS Ospedale San Raffaele

Milan, Italy

Location

Fondazione IRCCS San Gerardo dei Tintori

Monza, Italy

Location

Azienda Ospedaliero Universitaria Pisana

Pisa, Italy

Location

Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore

Roma, 00168, Italy

Location

Azienda Ospedaliero Universitaria Policlinico Umberto I

Roma, Italy

Location

AOU Citta della Salute e della Scienza di Torino - Ospedale le Molinette

Torino, Italy

Location

Pia Fondazione di Culto e Religione Cardinale Giovanni Panico

Tricase, Italy

Location

Brain Research Center Den Bosch

's-Hertogenbosch, 5223 LA, Netherlands

Location

Brain Research Center

Amsterdam, Netherlands

Location

Amphia Ziekenhuis - Locatie Breda Molengracht

Breda, Netherlands

Location

Brain Research Center Zwolle

Zwolle, Netherlands

Location

KLIMED Marek Klimkiewicz

Bialystok, Poland

Location

Podlaskie Centrum Psychogeriatrii

Bialystok, Poland

Location

NEURO-CARE Sp. z o.o. Sp. Komandytowa

Katowice, Poland

Location

Osrodek Alzheimerowski Sp. z o.o

Åšcinawa, Poland

Location

ETG Neurosciences Sp. Z.o.o.

Warsaw, Poland

Location

NZOZ Wroclawskie Centrum Alzheimerowski

Wroclaw, Poland

Location

Seoul National University Bundang Hospital

Seongnam-si, Bundang-gu, 13620, South Korea

Location

Chonnam National University Hospital

Gwangju, Dong-Gu, 61469, South Korea

Location

Ewha Womans University Seoul Hospital

Seoul, Gangseo-gu, 07804, South Korea

Location

Hallym University Chuncheon Sacred Heart Hospital

Chuncheon, Gangwon-do, 24253, South Korea

Location

Gachon University Gil Medical Center

Incheon, Namdong-Gu, 21565, South Korea

Location

Severance Hospital, Yonsei University Health System

Seoul, Seodaemun-gu, 03722, South Korea

Location

Ewha Womans University Mokdong Hospital

Seoul, Seoul, South Korea

Location

Soonchunhyang University Hospital Cheonan

Cheonan, South Korea

Location

Kyungpook National University Chilgok Hospital

Daegu, 41404, South Korea

Location

Yeungnam University Hospital

Daegu, South Korea

Location

Hanyang University Guri Hospital

Guri-si, South Korea

Location

Uijeongbu St. Mary's Hospital

Gyeonggi-do, South Korea

Location

Inha University Hospital

Incheon, 400-711, South Korea

Location

Catholic Kwandong University International St.Mary's Hospital

Incheon, South Korea

Location

Asan Medical Center

Seoul, 05505, South Korea

Location

The Catholic University of Korea Seoul St.Mary's Hospital

Seoul, 06591, South Korea

Location

Chung ang University Hospital

Seoul, 06973, South Korea

Location

Korea University Guro Hospital

Seoul, 08308, South Korea

Location

Eunpyeong St.Mary's Hospital

Seoul, South Korea

Location

Gangnam Severance Hospital, Yonsei University Health System

Seoul, South Korea

Location

Konkuk University Hospital

Seoul, South Korea

Location

Samsung Medical Center

Seoul, South Korea

Location

Severance Hospital, Yonsei University Health System

Seoul, South Korea

Location

Ajou University Hospital

Suwon, South Korea

Location

Fundacio ACE - Institut Catala de Neurociencies Aplicades (Alzheimer Research Center Memory Clinic)

Barcelona, Spain

Location

Hospital del Mar

Barcelona, Spain

Location

Hospital Universitario Vall d'Hebron

Barcelona, Spain

Location

Hospital Universitario Juan Ramon Jimenez

Huelva, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, Spain

Location

Clinica Universidad de Navarra - Pamplona

Pamplona, Spain

Location

Hospital Clinico Universitario de Salamanca

Salamanca, Spain

Location

Policlinica Gipuzkoa

San Sebastián, Spain

Location

Hospital Universitario Virgen del Rocio

Seville, 41013, Spain

Location

Hospital Universitari i Politecnic La Fe

Valencia, Spain

Location

Fulbourn Hospital

Cambridge, United Kingdom

Location

Cornwall Partnership NHS Foundation Trust

Cornwell, United Kingdom

Location

Royal Devon University Healthcare NHS Foundation Trust

Exeter, United Kingdom

Location

Hammersmith Hospital

London, W12 0HS, United Kingdom

Location

NeuroClin Glasgow

Motherwell, United Kingdom

Location

Warneford Hospital

Oxford, United Kingdom

Location

Southern Health NHS Foundation Trust

Southampton, United Kingdom

Location

MeSH Terms

Conditions

Alzheimer Disease

Condition Hierarchy (Ancestors)

DementiaBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesTauopathiesNeurodegenerative DiseasesNeurocognitive DisordersMental Disorders

Study Officials

  • James Rock

    AriBio Co., Ltd.

    STUDY DIRECTOR
  • Sharon Sha, MD, MS

    Stanford University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Masking Details
double-blind, randomized, placebo-controlled
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: A Phase 3 Double-blind, Randomized, Placebo-controlled, Multi-center Trial to Evaluate the Efficacy and Safety of AR1001 over 52 Weeks in Participants with Early Alzheimer's Disease
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 31, 2022

First Posted

September 8, 2022

Study Start

December 23, 2022

Primary Completion

May 1, 2026

Study Completion (Estimated)

December 1, 2027

Last Updated

October 2, 2025

Record last verified: 2025-09

Locations