Phase 3, Double-blind, Randomized, Placebo-controlled Trial to Evaluate the Efficacy and Safety of AR1001 in Participants With Early Alzheimer's Disease (Polaris-AD)

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 (Polaris-AD)

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Alzheimer Disease
• Intervention / Treatment: Drug: AR1001; Drug: Placebo

Detailed Description

The purpose of this Study is to evaluate the efficacy and safety of AR1001 in participants with Early Alzheimer's Disease (AD).

AR1001 is a small molecule that has demonstrated its potential as a therapeutic agent for AD via its polypharmacological characteristics with multiple mechanisms to ameliorate AD pathology.

• Study Type: Interventional
• Enrollment (Actual): 1535
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • Halifax, Canada
    • Centricity Research - Halifax
  • Kelowna, Canada
    • Okanagan Clinical Trials
  • London, Canada
    • Parkwood Institute
  • North York, Canada
    • Baycrest Academy for Research and Education
  • Peterborough, Canada
    • Kawartha Centre - Redefining Healthy Aging
  • Toronto, Canada
    • Sunnybrook Health Sciences Centre
  • Toronto, Canada
    • Centricity Research
  • Toronto, Canada
    • University Health Network-Toronto Western Hospital
  • Victoria, Canada
    • Royal Jubilee Hospital
  • West Vancouver, Canada
    • Medical Arts Health Research Group - West Vancouver
• China
  • Beijing, China
    • Peking Union Medical College Hospital
  • Beijing, China
    • Beijing Friendship Hospital,Capital Medical University
  • Beijing, China
    • Xuanwu Hospital Capital Medical University
  • Beijing, China
    • Peking University Sixth Hospital
  • Beijing, China
    • Beijing Tiantan Hospital, Capital Medical University
  • Beijing, China
    • Beijing Anding Hospital Capital Medical University
  • Changchun, China
    • The First Hospital of Jilin University
  • Changsha, China
    • Xiangya Hospital of Central South University
  • Chongqing, China
    • The First Affiliated Hospital of Chongqing Medical University
  • Guangzhou, China
    • Guangdong Provincial People's Hospital
  • Guangzhou, China
    • The First Affiliated Hospital, Sun Yat-sen University
  • Guangzhou, China
    • The Affiliated Brain Hospital of Guangzhou Medical University
  • Hefei, China
    • Anhui Provincial Hospital
  • Heping, China
    • Tianjin Medical University General Hospital
  • Nanchang, China
    • The Second Affiliated Hospital of Nanchang University
  • Nanjing, China
    • Nanjing Drum Tower Hospital
  • Nanjing, China
    • The Second Affiliated Hospital of Nanjing Medical University
  • Shanghai, China
    • Huashan Hospital affiliated to Fudan University
  • Shanghai, China
    • Renji Hospital, Shanghai Jiao Tong University School of Medicine
  • Shijiazhuang, China
    • The First Hospital of Hebei Medical University
  • Suzhou, China
    • The First Affiliated Hospital of Soochow University
  • Suzhou, China
    • The Second Affiliated Hospital of Soochow University
  • Wuhan, China
    • Tongji Hospital of Tongji University
  • Xuzhou, China
    • Xuzhou Central Hospital
• Czechia
  • Brno, Czechia
    • Fakultni nemocnice u sv. Anny v Brne
  • Choceň, Czechia
    • NEUROHK s.r.o.
  • Hradec Králové, Czechia
    • Fakultni nemocnice Hradec Kralove
  • Pilsen, Czechia
    • Ambulance Smrkova ulice (A-Shine s.r.o.)
  • Prague, Czechia
    • Forbeli s.r.o
  • Prague, Czechia
    • INEP Medical s.r.o.
  • Prague, Czechia
    • Neuropsychiatrie s.r.o.
  • Rychnov nad Kněžnou, Czechia, 516 01
    • Vestra Clinics
• Denmark
  • Gandrup, Denmark, 9362
    • Sanos Clinic - Gandrup
  • Herlev, Denmark
    • Sanos Clinic - Herlev
  • Vejle, Denmark
    • Sanos Clinic Syddanmark - Vejle
• France
  • Lille, France
    • CHU de Lille - Hôpital Roger Salengro
  • Marseille, France
    • AP-HM - Hôpital de la Timone
  • Nantes, France
    • CHU de Nantes - Hopital Nord Laennec
  • Paris, France
    • AP-HP Hopital Broca
  • Paris, France
    • Ap-Hp Hopital Lariboisiere
  • Paris, France
    • AP-HP Hopital Pitie-Salpetriere
  • Rennes, France, 35033
    • CHU Rennes - Hôpital Pontchaillou
  • Strasbourg, France, 67200
    • CHRU de Strasbourg - Hopital de Hautepierre
  • Toulouse, France
    • CHU de Toulouse - Hopital La Grave
  • Villeurbanne, France
    • HCL - Hopital des Charpennes
• Germany
  • Aachen, Germany
    • Universitaetsklinikum Aachen, AoeR
  • Bad Homburg, Germany
    • Zentrum fuer klinische Forschung Dr. med. I. Schoell
  • Berlin, Germany
    • Charité Universitaetsmedizin Berlin
  • Günzburg, Germany
    • Bezirkskrankenhaus Guenzburg
  • Homburg, Germany
    • Universitaetsklinikum des Saarlandes
  • Mannheim, Germany, 68165
    • Institut fuer Studien zur Psychischen Gesundheit (ISPG)
• Italy
  • Ancona, Italy
    • Azienda Ospedaliero-Universitaria delle Marche
  • Brescia, Italy
    • IRCCS Istituto Centro San Giovanni di Dio Fatebenefratelli
  • Milan, Italy
    • IRCCS Ospedale San Raffaele
  • Milan, Italy
    • ASST Grande Ospedale Metropolitano Niguarda
  • Monza, Italy
    • Fondazione IRCCS San Gerardo dei Tintori
  • Pisa, Italy
    • Azienda Ospedaliero Universitaria Pisana
  • Roma, Italy, 00168
    • Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
  • Roma, Italy
    • Azienda Ospedaliero Universitaria Policlinico Umberto I
  • Torino, Italy
    • AOU Citta della Salute e della Scienza di Torino - Ospedale le Molinette
  • Tricase, Italy
    • Pia Fondazione di Culto e Religione Cardinale Giovanni Panico
• Netherlands
  • 's-Hertogenbosch, Netherlands, 5223 LA
    • Brain Research Center Den Bosch
  • Amsterdam, Netherlands
    • Brain Research Center
  • Breda, Netherlands
    • Amphia Ziekenhuis - Locatie Breda Molengracht
  • Zwolle, Netherlands
    • Brain Research Center Zwolle
• Poland
  • Bialystok, Poland
    • KLIMED Marek Klimkiewicz
  • Bialystok, Poland
    • Podlaskie Centrum Psychogeriatrii
  • Katowice, Poland
    • NEURO-CARE Sp. z o.o. Sp. Komandytowa
  • Warsaw, Poland
    • ETG Neurosciences Sp. Z.o.o.
  • Wroclaw, Poland
    • NZOZ Wroclawskie Centrum Alzheimerowski
  • Ścinawa, Poland
    • Osrodek Alzheimerowski Sp. z o.o
• South Korea
  • Cheonan, South Korea
    • SoonChunHyang University Hospital Cheonan
  • Daegu, South Korea, 41404
    • Kyungpook National University Chilgok Hospital
  • Daegu, South Korea
    • Yeungnam University Hospital
  • Guri-si, South Korea
    • Hanyang University Guri Hospital
  • Gyeonggi-do, South Korea
    • Uijeongbu St. Mary's Hospital
  • Incheon, South Korea, 400-711
    • Inha University Hospital
  • Incheon, South Korea
    • Catholic Kwandong University International St.Mary's Hospital
  • Seoul, South Korea, 05505
    • Asan Medical Center
  • Seoul, South Korea
    • Samsung Medical Center
  • Seoul, South Korea, 08308
    • Korea University Guro Hospital
  • Seoul, South Korea
    • Severance Hospital, Yonsei University Health System
  • Seoul, South Korea, 06591
    • The Catholic University of Korea Seoul St.Mary's Hospital
  • Seoul, South Korea
    • Konkuk University Hospital
  • Seoul, South Korea, 06973
    • Chung Ang University Hospital
  • Seoul, South Korea
    • Eunpyeong St.Mary's Hospital
  • Seoul, South Korea
    • Gangnam Severance Hospital, Yonsei University Health System
  • Suwon, South Korea
    • Ajou University Hospital
  • Bundang-gu
    • Seongnam-si, Bundang-gu, South Korea, 13620
      • Seoul National University Bundang Hospital
  • Dong-Gu
    • Gwangju, Dong-Gu, South Korea, 61469
      • Chonnam National University Hospital
  • Gangseo-gu
    • Seoul, Gangseo-gu, South Korea, 07804
      • Ewha Womans University Seoul Hospital
  • Gangwon-do
    • Chuncheon, Gangwon-do, South Korea, 24253
      • Hallym University Chuncheon Sacred Heart Hospital
  • Namdong-Gu
    • Incheon, Namdong-Gu, South Korea, 21565
      • Gachon University Gil Medical Center
  • Seodaemun-gu
    • Seoul, Seodaemun-gu, South Korea, 03722
      • Severance Hospital, Yonsei University Health System
  • Seoul
    • Seoul, Seoul, South Korea
      • Ewha Womans University Mokdong Hospital
• Spain
  • Barcelona, Spain
    • Hospital del Mar
  • Barcelona, Spain
    • Hospital Universitario Vall d'Hebron
  • Barcelona, Spain
    • Fundacio ACE - Institut Catala de Neurociencies Aplicades (Alzheimer Research Center Memory Clinic)
  • Huelva, Spain
    • Hospital Universitario Juan Ramon Jimenez
  • Madrid, Spain
    • Hospital Universitario 12 de Octubre
  • Madrid, Spain
    • Hospital Universitario Ramón y Cajal
  • Pamplona, Spain
    • Clinica Universidad de Navarra - Pamplona
  • Salamanca, Spain
    • Hospital Clínico Universitario de Salamanca
  • San Sebastián, Spain
    • Policlinica Gipuzkoa
  • Seville, Spain, 41013
    • Hospital Universitario Virgen del Rocio
  • Valencia, Spain
    • Hospital Universitari i Politècnic La Fe
• United Kingdom
  • Cambridge, United Kingdom
    • Fulbourn Hospital
  • Cornwell, United Kingdom
    • Cornwall Partnership NHS Foundation Trust
  • Exeter, United Kingdom
    • Royal Devon University Healthcare NHS Foundation Trust
  • London, United Kingdom, W12 0HS
    • Hammersmith Hospital
  • Motherwell, United Kingdom
    • NeuroClin Glasgow
  • Oxford, United Kingdom
    • Warneford Hospital
  • Southampton, United Kingdom
    • Southern Health NHS Foundation Trust
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85012
      • IMA Clinical Research Phoenix
    • Scottsdale, Arizona, United States, 85253
      • Perseverance Research Center, LLC
    • Scottsdale, Arizona, United States, 85258
      • Clinical Endpoints - N. Scottsdale
    • Sun City, Arizona, United States, 85351
      • Banner Sun Health Research Institute
    • Tempe, Arizona, United States, 85282
      • Voyage Medical
  • California
    • Burlingame, California, United States, 94010
      • Sutter Health's Palo Alto Medical Foundation
    • Fresno, California, United States, 93710
      • Neuro-Pain Medical Center
    • Fullerton, California, United States, 92835
      • Fullerton Neurology and Headache Center
    • Murrieta, California, United States, 92562
      • Esperanza Clinical
    • Northridge, California, United States, 91325
      • Valley Clinical Trials, INC
    • Pasadena, California, United States, 91105
      • Havana Research Institute
    • San Diego, California, United States, 92037
      • Kaizen Brain Center
    • San Jose, California, United States, 95124
      • Adaptive Research
    • San Marcos, California, United States, 92069
      • The Neuron Clinic
    • Stanford, California, United States, 94305
      • Stanford Neuroscience Health Center
  • Colorado
    • Denver, Colorado, United States, 80218
      • Mile High Research Center
    • Denver, Colorado, United States, 80210
      • Denver Neurological Research
    • Englewood, Colorado, United States, 80113
      • CenExel Rocky Mountain Clinical Research
  • Florida
    • Apopka, Florida, United States, 32803
      • Topaz Clinical Research
    • Clearwater, Florida, United States, 33756
      • BayCare Health System, Inc
    • Clermont, Florida, United States, 34711
      • Vertex Research Group
    • Daytona Beach, Florida, United States, 32117
      • Arrow Clinical Trial
    • Loxahatchee Groves, Florida, United States, 33470
      • Brainstorm Research - Loxahatchee
    • Maitland, Florida, United States, 32751
      • ClinCloud LLC
    • Miami, Florida, United States, 33176
      • Brainstorm Research
    • Miami, Florida, United States, 33155
      • Allied Biomedical Research Institute, Inc
    • Miami, Florida, United States, 33135
      • Vitae Research Center, LLC
    • Miami, Florida, United States, 33135
      • Verus Clinical Research, Corp
    • Miami, Florida, United States, 33169
      • Future Life Clinical Trials
    • Miami, Florida, United States, 33145
      • Caro Medcenter and Community Research
    • Miami Gardens, Florida, United States, 33014
      • Meridian International Research, Inc
    • Orlando, Florida, United States, 32803
      • Charter Research - Orlando
    • Orlando, Florida, United States, 32806
      • K2 Medical Research
    • Palm Beach, Florida, United States, 33462
      • JEM Research Institute
    • Port Orange, Florida, United States, 32127
      • Progressive Medical Research
    • Tampa, Florida, United States, 33614
      • Angels Clinical Research Institute, Inc. - Tampa
    • The Villages, Florida, United States, 32162
      • Charter Research - The Villages
    • Viera, Florida, United States, 32940
      • ClinCloud, LLC Melbourn
    • Winter Park, Florida, United States, 32789
      • Conquest Research
  • Georgia
    • Atlanta, Georgia, United States, 30327
      • Atlanta NeuroScience Institute
    • Atlanta, Georgia, United States, 30030
      • iResearch -Atlanta
    • Decatur, Georgia, United States, 30030
      • Accel Research Sites
    • Savannah, Georgia, United States, 31405
      • IResearch
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Re:Cognition Health - Chicago
  • Louisiana
    • New Orleans, Louisiana, United States, 70072
      • Tandem Clinical Research
  • Massachusetts
    • New Bedford, Massachusetts, United States, 02740
      • Btc of New Bedford
    • Newton, Massachusetts, United States, 02459
      • Boston Center for Memory
    • Plymouth, Massachusetts, United States, 02360
      • Headlands Research - Eastern MA
    • Waltham, Massachusetts, United States, 02451
      • Boston Paincare
  • Missouri
    • Ozark, Missouri, United States, 65721
      • Sharlin Health Neuroscience Research Center
  • Nebraska
    • Lincoln, Nebraska, United States, 68526
      • Alivation
  • Nevada
    • Las Vegas, Nevada, United States, 89106
      • Wake Research- Clinical Research Center of Nevada, LLC
  • New Jersey
    • Toms River, New Jersey, United States, 08755
      • Advanced Memory Research Institute of New Jersey
  • New York
    • Amherst, New York, United States, 14226
      • Dent Neuroscience Research Center
    • New Windsor, New York, United States, 12553
      • Mid-Hudson Medical Research, PLLC - New Windsor
  • North Carolina
    • Greensboro, North Carolina, United States, 27410
      • Triad Clinical Trials
    • Matthews, North Carolina, United States, 28105
      • AMC Research, LLC
    • Winston-Salem, North Carolina, United States, 27103
      • Accellacare of Winston-Salem, Triad Neurological Associates
  • Ohio
    • Canton, Ohio, United States, 44718
      • Neuroscience Research Center, LLC
    • Dayton, Ohio, United States, 45432
      • American Clinical Research Institute, LLC
  • Rhode Island
    • East Providence, Rhode Island, United States, 02914
      • Rhode Island Mood and Memory REsearch
  • South Dakota
    • Rapid City, South Dakota, United States, 57702
      • Health Concepts
  • Tennessee
    • Cordova, Tennessee, United States, 38018
      • Neurology Clinic, P.C.
    • Knoxville, Tennessee, United States, 37909
      • Center for Biomedical Research, LLC - Genesis Neuroscience Clinic
  • Texas
    • Beaumont, Texas, United States, 77702
      • Gadolin Research LLC
    • Dallas, Texas, United States, 75231
      • Kerwin Medical Center
    • Houston, Texas, United States, 77074
      • Clinical Trial Network - Houston
    • Houston, Texas, United States, 77030
      • Cognition Health Corporation- Texas
    • Lubbock, Texas, United States, 79410
      • Bhupesh Dihenia, MD, PA
  • Utah
    • Salt Lake City, Utah, United States, 84107
      • Wasatch Clinical Research, LLC
  • Virginia
    • Fairfax, Virginia, United States, 22031
      • Cognition Health Corporation
    • Falls Church, Virginia, United States, 22043
      • Integrated Neurology Services
  • Washington
    • Spokane, Washington, United States, 99201
      • Kingfisher Cooperative
  • West Virginia
    • Crab Orchard, West Virginia, United States, 25827
      • Vaught Neurological Services, PLLC
  • Wisconsin
    • Wauwatosa, Wisconsin, United States, 53226
      • Medical College of Wisconsin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 55 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

1. Male or female participants aged 55 to 90 years of age inclusive at the time of signing the informed consent form
2. 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
3. Participants with a history of subjective cognitive and memory decline with onset within 5 years before Screening, confirmed by study partner.
4. Participants who have a MMSE score greater than or equal to 20
5. Participants with a CDR global rating of 0.5 or 1
6. Participants with a RBANS score based on the Delayed Memory Index (DMI) score less than or equal to 85
7. If an historic magnetic resonance imaging (MRI) is available, findings must exclude other causes of dementia.

8. Positive biomarker for brain amyloid pathology as indicated by assessment of at least one of the following:

   1. 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.
   2. Historical amyloid positron emission tomography (PET) assessment confirmed by the Sponsor or Designee.
9. Participants (or participant's legally authorized representative) and caregiver (s) who can sign an informed consent to participate in the study.
10. 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.

Exclusion Criteria:

1. Participants who are female and are either pregnant, nursing, or of childbearing potential and not practicing effective contraception
2. Participants who have signs of significant delirium which, in the opinion of the Investigator, would interfere with this study
3. 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
4. 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
5. Participants with a history of vascular dementia
6. Participants with evidence of other neurological conditions thought to interfere with the evaluations in this study
7. 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
8. 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
9. Participants with a body mass index (BMI) > 35 kg/m2

10. Participants with any of the following:

    1. 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)
    2. deficiency (< lower limit of normal [LLN]) of Vitamin B12
    3. known history of HIV (human immunodeficiency virus) positivity or positive test for HIV 1/2 at screening
    4. known history of Hepatitis C virus (HCV) or positive test for HCV antibody (HCVAb) at screening (unless negative on confirmatory PCR test)'
    5. positive test for Hepatitis B surface antigen (HBsAg)
    6. known history of neurosyphilis or positive test for RPR at screening

11. Participants who have history of cancer or malignant tumor within 5 years prior to screening with the exception of:

    1. Basal or squamous cell carcinoma of the skin or cervical dysplasia, which has been adequately treated
    2. In situ Grade 1 cervical cancer, fully treated at least 2 years prior to screening, and without recurrence.
    3. Prostate cancer, confined to the prostate gland, which has been adequately treated (e.g., surgery and/or radiation or watchful waiting) with normal or low and stable prostate-specific antigen (PSA) levels for 2 years prior to Screening
    4. Adequately treated non-metastatic breast cancer
12. Participants who in the opinion of the Investigator have an inadequately treated thyroid disorder
13. Participants with inherited degenerative retinal disease
14. Participants who have an undiagnosed or uncontrolled seizure disorder (and/or an epileptic syndrome), which has or could lead to cognitive impairment either from repeated seizures or the medications used to control the seizure disorder
15. Participants who are being treated, or likely to require treatment during the study, with any medications prohibited by the study protocol
16. Participants who have participated in any investigational drug or device trial within the previous 30 days or five half-lives of an investigational drug at Screening, whichever is longer
17. Participants taking an oral cholinesterase inhibitor and/or memantine not on a stable dose for at least 3 months prior to screening. Treatment and dosing should remain stable, with no changes throughout the trial.
18. Participants who have been and/or are currently being treated with anti-amyloid, anti-tau, or any other investigational therapies for AD
19. Participants who currently take any other PDE-5 Inhibitors (e.g., sildenafil)
20. Participants who are currently receiving (or unable to stop use for at least 14 days [2 weeks] prior to receiving the first dose of the AR1001 and throughout the study) prescription or nonprescription medications or other products known to be potent inhibitors of cytochrome P450 isozyme 3A4 (CYP3A4)
21. Alcohol or substance use disorder within the past 5 years according to Diagnostic and Statistical Manual of Mental Disorders (DSM-5)
22. Participants who have previously participated in a clinical trial with AR1001
23. Participants, in the opinion of the Investigator, who are unsuitable to participate in the trial
24. Participants who in the opinion of the Investigator are at significant risk of suicide.
25. GDS-15 score greater than equal to 8 at Screening
26. Participants, in the opinion of the Investigator, who have any who have any contraindications to undergoing LP. Participants receiving ongoing anticoagulant therapy or antiplatelet therapy (other than aspirin and non-steroidal anti-inflammatory drugs [NSAIDs]) should also be excluded if it is considered unsafe to temporarily discontinue the therapy

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Group A - 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; AR1001 Active Oral Tablet
Placebo Comparator: Group B - 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; Placebo Oral Tablet

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Clinical Dementia Rating Scale - Sum of Boxes (CDR-SB)	Change in the CDR-SB from Baseline to Week 52	52 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Alzheimer's Disease Assessment Scale, cognitive subscale (ADAS-Cog 13)	Change in ADAS-Cog 13 from Baseline to Week 52	52 weeks
Amsterdam-Instrumental Activities of Daily Living Questionnaire-Short Version (A-IADL-Q-SV)	Change in the A-IADL-Q-SV from Baseline to Week 52	52 weeks
Geriatric Depression Scale (GDS)	Change in the GDS from Baseline to Week 52	52 weeks
Mini-Mental Status Examination (MMSE)	Change in the MMSE from Baseline to Week 52	52 weeks

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety Analysis	Frequency of treatment emergent adverse events (TEAEs) and treatment-emergent serious adverse events (TESAEs); Changes in C-SSRS (Columbia Suicide Severity Rating Scale)	156 weeks
Biomarker Analysis	Change in plasma/CSF biomarker levels from Baseline to Week 52 and the end of the Extension Phase.	156 weeks
Exploratory Analysis	Change in both primary and secondary endpoints from Baseline and Week 52 to the end of the extension study.	156 weeks

Collaborators and Investigators

• Sponsor: AriBio Co., Ltd.
• Investigators: Principal Investigator: Sharon Sha, MD, MS, Stanford University; Study Director: James Rock, AriBio Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): December 23, 2022
• Primary Completion (Estimated): May 1, 2026
• Study Completion (Estimated): December 1, 2027

Study Registration Dates

• First Submitted: August 31, 2022
• First Submitted That Met QC Criteria: September 2, 2022
• First Posted (Actual): September 8, 2022

Study Record Updates

• Last Update Posted (Estimated): October 2, 2025
• Last Update Submitted That Met QC Criteria: September 29, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: AD; AR1001; Early AD
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Mental Disorders; Neurocognitive Disorders; Dementia; Tauopathies; Neurodegenerative Diseases; Alzheimer Disease
• Other Study ID Numbers: AR1001-ADP3-US01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No