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Mild to Moderate Alzheimer's DiseasePhase 2NCT03625622

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

A Phase 2 study testing AR1001 in people with Mild to Moderate Alzheimer's Disease, run by AriBio Co., Ltd. at 21 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 55 to 80 years
Conditions the study is for
Mild to Moderate Alzheimer's Disease
What is being tested?
AR1001
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
210 participants
Where
21 sitesUnited States
Study dates
1 Apr 2019to 28 Jun 2021
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 55 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.
Show all 23Show fewer
  • 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.
  • Subjects who have any other clinically significant abnormal result in laboratory tests such as abnormally low B12 or high TSH levels, as determined by the Investigator.
  • Subjects with any current psychiatric diagnosis other than AD if, in the judgment of the investigator, the psychiatric disorder or symptom is likely to confound interpretation of drug effect, affect cognitive assessments, or affect the subject's ability to complete the study.
  • Subjects whose treatment with FDA-approved AD medication (donepezil, galantamine, memantine, rivastigmine or their combinations) has not been stable for at least 3 months prior to screening. Treatment and dosing should remain stable, with no changes throughout the trial.
  • Subjects who are currently receiving (or unable to stop use for at least 21 days [3 weeks] prior to receiving the first dose of the AR1001 and throughout the study) prescription or non-prescription medications or other products known to be moderate or potent inhibitors/inducers of CYP3A4.
  • Subjects who have had any intake of grapefruit, grapefruit juice, Seville oranges, Seville orange marmalade, or other products containing grapefruit or Seville oranges within 7 days of the first administration of the AR1001 and throughout the study.
  • Subjects, in the opinion of the Investigator, who are unsuitable to participate in the study.
  • Extension Phase Continuation Criterion
  • 1. Subjects enrolled in AR1001-ADP2-US01 and completed 26 weeks of assigned dosing.

Trial locations and contacts

21 sites in 1 country.

21 sites in 1 country

United States21 sites

  • Advanced Clinical Research, Inc.

    Banning, California, 92220

  • Rapid Medical Research

    Beachwood, Ohio, 44122

  • Advanced Clinical Research - Cedar Park

    Cedar Park, Texas, 78613

  • NeuroStudies, LLC

    Decatur, Georgia, 30033

  • Meridien Research

    Lakeland, Florida, 33805

  • FMC Science

    Lampasas, Texas, 76550

  • Wake Research - CRCNV

    Las Vegas, Nevada, 89106

  • Meridien - Maitland

    Maitland, Florida, 32751

  • Advanced Clinical Research

    Meridian, Idaho, 83642

  • The Neurology Research Group

    Miami, Florida, 33176

  • Lynn Health Science Institute

    Oklahoma City, Oklahoma, 73112

  • Accelerated Enrollment Solutions (AES)

    Orlando, Florida, 32806

  • IMIC, Inc

    Palmetto Bay, Florida, 33157

  • Northern California Research

    Sacramento, California, 95821

  • Syrentis Clinical Research

    Santa Ana, California, 92705

  • Kingfisher Cooperative, LLC

    Spokane, Washington, 99202

  • Meridien Research - Spring Hill

    Spring Hill, Florida, 34609

  • Meridien Research - St Petersburg

    St. Petersburg, Florida, 33709

  • Palmetto Clinical Research

    Summerville, South Carolina, 29485

  • Meridien Research - Tampa

    Tampa, Florida, 33634

  • Advanced Clinical Research, Inc.

    West Jordan, Utah, 84088

Other trials in United States

About this study

Official title: A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate Efficacy and Safety of 26-Week Treatment of AR1001 in Patients With Mild to Moderate Alzheimer's Disease

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.

Read the detailed description

Alzheimer's Disease (AD) is the most prevalent neurodegenerative disorder in The United States affecting approximately 5.4 million Americans. AD is characterized by progressive loss in memory and as well as a decline in the ability to learn that is associated with neuronal death. Well known hallmarks of AD are neuritic plaques and neurofibrillary tangles and extensive inflammation. Currently, no treatment has been developed to fully cure or prevent the progression of dementia that is associated with AD.

AR1001 is a polypharmacological drug candidate being developed as a treatment for AD and shows great potential with favorable attributes for a central nervous system (CNS) drug (i.e., high specificity and potency, as well as good pharmacokinetic, bioavailability, CNS penetration, and ensured safety).

The clinical study of AR1001 aims to evaluate the efficacy and safety of AR1001 as a potential treatment for AD. Based on the preclinical results, AR1001 could be an effective treatment option with a mechanism of action that has not been explored for AD indication.

Being tested
AR1001, Placebo
Keywords
Alzheimer's disease
Medical subject headings
Alzheimer Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AR1001Drug

    AR1001 Active Oral Tablet

  • PlaceboDrug

    Placebo Oral Tablet

Study groups in NCT03625622
Study groupRoleWhat they receive
PlaceboPlacebo comparatorPlacebo, orally administered once daily for 26 weeks.
Receives: Drug: Placebo
AR1001 - 10 mgActive comparatorActive, AR1001 - 10 mg, orally administered once daily for 26 weeks.
Receives: Drug: AR1001
AR1001 - 30 mgActive comparatorActive, AR1001 - 30 mg, orally administered once daily for 26 weeks.
Receives: Drug: AR1001

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. ADAS-Cog 13

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

    Measured: 26 weeks

  2. ADCS-CGIC

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

    Measured: 26 weeks

Secondary outcomes (6)

  1. MMSE-2

    Change of MMSE (Mini-mental status examination) from baseline at Week 26

    Measured: 26 weeks

  2. NPI

    Changes of NPI (Neuropsychiatric Inventory) from baseline at Week 26

    Measured: 26 weeks

  3. GDS

    Changes of GDS (Geriatric Depression Scale) from baseline at Week 26

    Measured: 26 weeks

  4. C-SSRS

    Changes of C-SSRS (Columbia Suicide Severity Rating Scale) from baseline at Week 26

    Measured: 26 weeks

  5. QOL-AD

    Changes of QOL-AD (Quality of Life in Alzheimer's Disease) from baseline at Week 26

    Measured: 26 weeks

  6. Treatment related adverse events

    Number of subjects with treatment related adverse events as assessed by analysis of adverse events including symptoms and abnormal findings on physical examination, vital signs, ECG, and standard laboratory examination results

    Measured: 26 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Aug 2018
  2. Study start1 Apr 2019
  3. Primary completion22 Dec 2020
  4. Study completion28 Jun 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
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.
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
210 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03625622
Sponsor's protocol ID
AR1001-ADP2-US01
Status last verified
Jul 2021
First submitted
8 Aug 2018
Last updated
21 Jul 2021
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • 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.

    PubMed 40912996

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing AR1001 in people with Mild to Moderate Alzheimer's Disease, run by AriBio Co., Ltd. at 21 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 21 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 21 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

ADAS-Cog 13 (time frame: 26 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03625622, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jul 2021.

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