Efficacy and Safety of 26-Week Treatment of AR1001 in Patients With Mild to Moderate Alzheimer's Disease

NCT03625622 | Completed Phase 2 FDA Drug

• 1 other identifier: AR1001-ADP2-US01
• Study Type: interventional
• Target: 210
• Locations: 1 country
• Sites: 21

Brief Summary

A double-blinded, randomized, placebo-controlled study will be performed to evaluate the efficacy and safety of treating AR1001 in patients with mild to moderate Alzheimer's disease for 26 weeks.

Conditions

Mild to Moderate Alzheimer's Disease

Keywords

Alzheimer's disease

Outcome Measures

Primary Outcomes (2)

• ADAS-Cog 13

  Change of ADAS-Cog (Alzheimer's Disease Assessment Scale-cognitive subscale) from baseline at Week 26

  26 weeks

• ADCS-CGIC

  Change of ADCS-CGIC (Alzheimer's disease Cooperative Study-Clinical Global Impression of Change)

  26 weeks

Secondary Outcomes (6)

• MMSE-2

  26 weeks

• NPI

  26 weeks

• GDS

  26 weeks

• C-SSRS

  26 weeks

• QOL-AD

  26 weeks

Study Arms (3)

Placebo

PLACEBO COMPARATOR

Placebo, orally administered once daily for 26 weeks.

Drug: Placebo

AR1001 - 10 mg

ACTIVE COMPARATOR

Active, AR1001 - 10 mg, orally administered once daily for 26 weeks.

Drug: AR1001

AR1001 - 30 mg

ACTIVE COMPARATOR

Active, AR1001 - 30 mg, orally administered once daily for 26 weeks.

Drug: AR1001

Interventions

AR1001 DRUG

AR1001 Active Oral Tablet

AR1001 - 10 mg; AR1001 - 30 mg

Placebo DRUG

Placebo Oral Tablet

Placebo

Eligibility Criteria

• Age: 55 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female subjects aged 55-80 years at the time of signing the Informed Consent Form.
• Subjects (or subject's legally acceptable representative) and caregiver(s) who can sign an Informed Consent to participate in the study. Same caregiver(s) must assist the subject throughout the entire duration of the study.
• Subjects who have a diagnosis of probable Alzheimer's disease according to the NIA-AA (National Institute of Aging and Alzheimer's Associations, 2011) criteria with mild to moderate dementia (stage 4 - 5) at screening.
• Subjects who have mild-to-moderate cognitive impairment with MMSE Score of 16-26 at screening.
• Subjects who have an MRI (either 1.5T or 3T) or CT scan performed after onset of symptoms and prior to randomization with findings consistent with the diagnosis of dementia due to Alzheimer's disease and without any other clinically significant comorbid pathologies.
• Subjects who have one (or more) identified adult study partner(s) who, in the opinion of the investigator, has sufficient contact with and knowledge about the subject as to be able to report knowledgeably about the subject's safety, compliance and adherence, cognition, function, and behavior.

You may not qualify if:

• Subjects who are female who are pregnant, nursing, or of childbearing potential and not practicing effective contraception.
• Subjects who have signs of delirium.
• Subjects who have had a cortical stroke within the preceding 2 years.
• Subjects who have any diagnosis of dementia other than that related to Alzheimer's Disease, including concomitant vascular dementia.
• Subjects who have a PET scan performed after onset of symptoms with negative amyloid results.
• Subjects with a history of myocardial infarction, unstable angina, New York Heart Association (NYHA) class III or IV heart failure or stroke within the last 12 months.
• Subjects with uncontrolled hypertension (systolic blood pressure \>160mm Hg or diastolic blood pressure \> 95mm Hg) or hypotension (systolic blood pressure \<90mm Hg or diastolic blood pressure \<50mm Hg).
• Subjects who have clinically significant renal impairment (creatinine \> 1.5x ULN) or hepatic impairment (AST or ALT \> 2.5x ULN or total bilirubin \> 1.5x ULN).
• Subjects who have history of cancer or malignant tumor within 5 years prior to screening with the exception of:
• Basal or squamous cell carcinoma of the skin or cervical dysplasia which has been adequately treated.
• In situ Grade 1 cervical cancer, fully treated at least 2 years prior to screening and without recurrence.
• Prostate cancer, confined to the prostate gland, which has been adequately treated (surgery and/or radiation) with normal or low and stable PSA levels for 2 years prior to screening.
• Subjects who have history of untreated thyroid disorder or a seizure disorder.
• Subjects who are being treated, or likely to require treatment during the study, with any medications prohibited by the study protocol.
• Subjects who have participated in any investigational drug or device trial within the previous 30 days or five half-lives of the investigational drug at screening, whichever one is longer.

Sponsors & Collaborators

• AriBio Co., Ltd.: lead

Study Sites (21)

Advanced Clinical Research, Inc.

Banning, California, 92220, United States

Location

Northern California Research

Sacramento, California, 95821, United States

Location

Syrentis Clinical Research

Santa Ana, California, 92705, United States

Location

Meridien Research

Lakeland, Florida, 33805, United States

Location

Meridien - Maitland

Maitland, Florida, 32751, United States

Location

The Neurology Research Group

Miami, Florida, 33176, United States

Location

Accelerated Enrollment Solutions (AES)

Orlando, Florida, 32806, United States

Location

IMIC, Inc

Palmetto Bay, Florida, 33157, United States

Location

Meridien Research - Spring Hill

Spring Hill, Florida, 34609, United States

Location

Meridien Research - St Petersburg

St. Petersburg, Florida, 33709, United States

Location

Meridien Research - Tampa

Tampa, Florida, 33634, United States

Location

NeuroStudies, LLC

Decatur, Georgia, 30033, United States

Location

Advanced Clinical Research

Meridian, Idaho, 83642, United States

Location

Wake Research - CRCNV

Las Vegas, Nevada, 89106, United States

Location

Rapid Medical Research

Beachwood, Ohio, 44122, United States

Location

Lynn Health Science Institute

Oklahoma City, Oklahoma, 73112, United States

Location

Palmetto Clinical Research

Summerville, South Carolina, 29485, United States

Location

Advanced Clinical Research - Cedar Park

Cedar Park, Texas, 78613, United States

Location

FMC Science

Lampasas, Texas, 76550, United States

Location

Advanced Clinical Research, Inc.

West Jordan, Utah, 84088, United States

Location

Kingfisher Cooperative, LLC

Spokane, Washington, 99202, United States

Location

Related Publications (1)

• Greeley D, Nash M, Herskowitz B, Kim F, Rock J, Prins N, Kim S, Xi T, Busam JA, Tete B, Choung JJ, Sha SJ. A phase 2 randomized, placebo-controlled study on the efficacy and safety of AR1001, a phosphodiesterase-5 inhibitor, in patients with mild-to-moderate Alzheimer's disease. J Prev Alzheimers Dis. 2025 Nov;12(9):100337. doi: 10.1016/j.tjpad.2025.100337. Epub 2025 Sep 5.

  PMID: 40912996 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

Study Officials

• James Rock

  SVP of global development

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double-blind for study site and participants
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: After the first 26 weeks, subjects can participate in an optional extension study to receive either 10 mg or 30 mg AR1001 for another 26 weeks.

Study Record Dates

• First Submitted: August 8, 2018
• First Posted: August 10, 2018
• Study Start: April 1, 2019
• Primary Completion: December 22, 2020
• Study Completion: June 28, 2021
• Last Updated: July 21, 2021

Record last verified: 2021-07

Locations

US (21)