Alzheimer DiseasePhase 2NCT06881836
A Study of ONO-2020 in Participants With Mild to Moderate Alzheimer's Disease
A Phase 2 study testing ONO-2020 in people with Alzheimer Disease, run by Ono Pharmaceutical Co., Ltd. at 80 sites in 2 countries.
Trial summary
This is a Phase 2, double-blind, parallel-group, placebo-controlled study to assess safety, tolerability, pharmacokinetics, and efficacy of ONO-2020 in participants with mild to moderate Alzheimer's disease (AD). Read more about this study
- Who is it for?
- Ages 55 to 85 years
- Conditions the study is for
- Alzheimer Disease
- What is being tested?
- ONO-2020
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 260 participants
- Where
- 80 sitesin 2 countries
- Study dates
- 24 Apr 2025to 1 Sep 2026
- Phase
- Phase 2
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 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Have a diagnosis of Alzheimer's disease according to the recommendations from the revised criteria for diagnosis and staging of Alzheimer's disease: Alzheimer's Association Workgroup , along with any positive AD-specific biomarker results (abnormal Core 1 or Core 2 biomarkers) from a previous diagnosis or at screening.
- Have a previous MRI or CT scan of the brain, which was performed within 1 year prior to enrollment in the study, to confirm that more recent neurological events (e.g., stroke) would not potentially constitute a confounder in the assessment of the etiology of the participant's cognitive status.
- MMSE score of 15 to 24, inclusive, and MMSE score cannot deviate more than 3 points in either direction between the screening and baseline visits.
- AD numeric clinical stage 4 or stage 5 based on NIA-AA criteria 2024, at screening and baseline visits
- Participants receiving concurrent AD treatment (acetylcholinesterase inhibitors and /or memantine) must be on a stable dose for at least 90 days prior to randomization, and the participant must be willing to remain on the same dose for the duration of the study.
- Have the ability to comply with procedures for cognitive and other tests in the opinion of the investigator
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- If female, postmenopausal for at least 1 year
- Non-vasectomized male participants with female partners of childbearing potential must agree to use an effective method of contraception from dosing on Day 1 until 3 months after the last administration of study intervention and agree not to donate sperm until 3 months after the last administration of study intervention.
- Participant must have a Caregiver who has frequent contact with the participant (defined as at least 8 hours per week spread across 3~4 visits per week) to provide support to the participant to ensure compliance with study requirements. The Caregiver must be willing to consent to participate in this study, to provide a rating of the extent and severity of change of the participant's memory, problem-solving abilities, or activities of daily living from prior abilities.
- General health status acceptable for participation in the study, and the participant must be able to ingest pills.
- Participant and his/her Caregiver have provided full written informed consent prior to the performance of any protocol-specified procedure; or if a participant is unable to provide informed consent due to cognitive status, he/she has provided assent, and a legally acceptable representative (LAR) has provided full written informed consent on behalf of the participant.
Who cannot join
- Participants with dementia or other memory impairment not due to Alzheimer's disease, including, but not limited to, dementia with Lewy bodies, vascular dementia, Parkinson's disease, Huntington disease, corticobasal degeneration, Creutzfeldt-Jakob disease, progressive supranuclear palsy, frontotemporal degeneration, normal pressure hydrocephalus, hypoxia, severe sleep apnea or other chronic sleep disturbance, or baseline intellectual disability.
- Participants with a history of stroke, well-documented transient ischemic attack, or pulmonary or cerebral embolism.
- History of significant psychiatric illness such as schizophrenia or bipolar affective disorder, or history or current major depressive disorder in the past year and any other significant psychiatric illness that in the opinion of the investigator could interfere with participation in the study.
- Participants with delirium or history of delirium within the 30 days prior to the screening visit.
- Have suicide ideation according to the investigator's clinical judgment as per the Columbia-Suicide Severity Rating Scale (C-SSRS) at screening or have made a suicide attempt in the 6 months prior to screening.
- Clinically significant ECG abnormality as judged by the investigator.
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- Confirmed absolute QTcF >450 msec for males or >470 msec for females.
- Positive results at screening for active viral infections that include human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), and hepatitis C virus (HCV) RNA PCR test.
- Participants with total bilirubin, alanine transaminase (ALT) or aspartate transaminase (AST) greater than 1.5×upper limit of normal (ULN), or international normalized ratio (INR) greater than 1.7 at screening.
- Participants with estimated creatinine clearance (CrCL, Cockcroft-Gault equation) ≤30 mL/min at screening.
- Participants with a history of treatment, and/or current treatment, with anti-Aβ antibodies
- Changes in any medications that, in the opinion of the investigator, may potentially impair participants' ability to perform cognitive testing or study procedures during the study period (from Screening to EOT), and their dosing should be stable for at least 1 month before Screening (such as benzodiazepines and sedatives/hypnotics). All concomitant medications must be kept as stable as medically possible during the study.
- Participants who have taken any investigational products, or used investigational medical devices, within 3 months or five half-lives of the therapy (whichever is longer) with respect to first dosing and throughout the study
Trial locations and contacts
80 sites in 2 countries.
80 sites in 2 countries
United States60 sites
Boston Clinical Trials
Boston, Massachusetts, 02131
University at Buffalo
Buffalo, New York, 14203
NeuroScience Research Center
Canton, Ohio, 44718
Profound Research LLC at The Neurology Center of Southern California
Carlsbad, California, 92011
New Hope Clinical Research
Charlotte, North Carolina, 28211
Re:Cognition Health-Chicago
Chicago, Illinois, 60611
University of Cincinnati
Cincinnati, Ohio, 45219
Cleveland Clinic
Cleveland, Ohio, 44195
The Ohio State University
Columbus, Ohio, 43221
Neurology Clinic, P.C.
Cordova, Tennessee, 38018
Horizon Clinical Research Group
Cypress, Texas, 77429
Texas Neurology
Dallas, Texas, 75206
Neurology Diagnostics Research
Dayton, Ohio, 45459
Sandhill Research, LLC d/b/a Accel Research Sites - NeuroStudies CRU
Decatur, Georgia, 30030
Brain Matters Research
Delray Beach, Florida, 33445
Ascension Alexian Brothers Medical Center
Elk Grove Village, Illinois, 60007
CenExel Rocky Mountain Clinical Research
Englewood, Colorado, 80113
Re:Cognition Health - Fairfax
Fairfax, Virginia, 22031
University of Kansas Medical Center
Fairway, Kansas, 66205
Neurology Center of North Orange County
Fullerton, California, 92835
Olympus Clinical Research - Katy
Katy, Texas, 77450
Cleveland Clinic Lou Ruvo Center for Brain Health
Las Vegas, Nevada, 89106
Vector Clinical Trials
Las Vegas, Nevada, 89128
Univ of Kentucky Sanders-Brown Center on Aging
Lexington, Kentucky, 40504
The Feinstein Institutes for Medical Research
Manhasset, New York, 11030
Velocity Clinical Research, Boise
Meridian, Idaho, 83642
ActivMed Research
Methuen, Massachusetts, 01844
Quantum Clinical Trials
Miami Beach, Florida, 33140
Vanderbilt UMC-Cognitive Med
Nashville, Tennessee, 37212
Suncoast Clinical Research
New Port Richey, Florida, 34652
NYU Center for Cognitive Neurology
New York, New York, 10016
Neuro Behavioral Clinical Research, Inc.
North Canton, Ohio, 44720
Renstar Medical Research
Ocala, Florida, 34470
Charter Research - Orlando
Orlando, Florida, 32803
Stanford University
Palo Alto, California, 94304
Penn Medicine
Philadelphia, Pennsylvania, 19104
Banner Alzheimer's Institute (BAI)
Phoenix, Arizona, 85006
Accel Research Sites - Brain and Spine Institute
Port Orange, Florida, 32127
Neural Net Research / Center for Cognitive Health
Portland, Oregon, 97225
Oregon Health & Science University
Portland, Oregon, 97239
Rhode Island Hospital
Providence, Rhode Island, 02903
Velocity Clinical Research at Raleigh Neurology
Raleigh, North Carolina, 27607
AD-CARE; University of Rochester
Rochester, New York, 14620
Be Well Clinical Studies
Round Rock, Texas, 78681
Central Texas Neurology Consultants
Round Rock, Texas, 78681
CenExel iResearch, LLC
Savannah, Georgia, 31405
Clinical Endpoints
Scottsdale, Arizona, 85258
Kingfisher Cooperative; LLC
Spokane, Washington, 99201
The Cognitive and Research Center of New Jersey
Springfield, New Jersey, 07081
Stony Brook University Hospital
Stony Brook, New York, 11794
Banner Sun Health Research Institute
Sun City, Arizona, 85351
USF Health Byrd Alzheimer's Institute
Tampa, Florida, 33613
ForCare Clinical Research
Tampa, Florida, 33647
Charter Research - The Villages
The Villages, Florida, 32162
Advanced Memory Research Institute of NJ (CenExel AMRI)
Toms River, New Jersey, 08755
Center for Neurosciences-Research
Tucson, Arizona, 85718
Sunwise Clinical Research
Walnut Creek, California, 94596
Advanced Clinical Institute Inc.
West Long Branch, New Jersey, 07764
Grayline Research Center
Wichita Falls, Texas, 76309
Conquest Research LLC
Winter Park, Florida, 32789
Japan20 sites
National Center for Geriatrics and Gerontology
Aichi
Inage Neurology and Memory Clinic
Chiba
Mabashi Clinic
Chiba
Southern Tohoku Medical Clinic
Fukushima
Ikuseikai Shinozuka Hospital
Gunma
Imon Yukari Neurology Clinic
Hiroshima
NHO Hiroshima-Nishi Medical Center
Hiroshima
Himeji Central Hospital Clinic
Hyōgo
Kobe City Medical Center General Hospital
Hyōgo
Memory Clinic Toride
Ibaraki
Kagawa Prefectural Central Hospital
Kagawa
Meiwakai Izaki Clinic
Nagasaki
Katayama Medical Clinic
Okayama
Takesato Hospital
Saitama
Jichiidai Station Brain Clinic
Tochigi
Ichiekai Itsuki Hospital
Tokushima
Federation of National Public Service Personnel Mutual Aid Associations Tachikawa Hospital
Tokyo
Memory Clinic Ochanomizu
Tokyo
Tokyo Medical University Hospital
Tokyo
Tokyo Metropolitan Institute for Geriatrics and Gerontology
Tokyo
About this study
Official title: A Phase II, 26-week, Double-blind, Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Efficacy of ONO-2020 in Patients With Mild to Moderate Alzheimer's Disease
This is a Phase 2, double-blind, parallel-group, placebo-controlled study to assess safety, tolerability, pharmacokinetics, and efficacy of ONO-2020 in participants with mild to moderate Alzheimer's disease (AD). This study aims to determine whether administering ONO-2020, an epigenetic regulator, may improve cognitive functions like memory and cognition in individuals with Alzheimer's disease dementia.
Read the detailed description
In the study, participants will undergo a screening period of up to 6 weeks (42 days). Eligible participants will be assigned to receive one of 2 dose levels of ONO-2020 or placebo control arm. ONO-2020 or placebo will be administered orally QD for 26 weeks. All participants who received study intervention will be followed up for 4 weeks after treatment discontinuation. The target sample size is 240 participants , out of which up to 45 participants will undergo additional special CSF biomarker evaluation. After enrollment, participants will be randomized in a 1:1:1 ratio to one of 3 treatment arms.
- Conditions
- Alzheimer Disease
- Being tested
- ONO-2020, Placebo
- Keywords
- Alzheimer Disease, mild to moderate Alzheimer Disease, Dementia, ONO-2020, mild Alzheimer Disease, moderate Alzheimer Disease
- Medical subject headings
- Alzheimer Disease, Dementia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ONO-2020Drug
ONO-2020 group: Two ONO-2020 tablets will be orally administered once daily.
PlaceboDrug
Placebo group: Two ONO-2020 placebo tablets will be orally administered once daily.
| Study group | Role | What they receive |
|---|---|---|
| ONO-2020 Dose 1 | Experimental | Participants will receive ONO-2020 Dose 1 administered orally, once a day (QD) for 26 weeks. Receives: Drug: ONO-2020 |
| ONO-2020 Dose 2 | Experimental | Participants will receive ONO-2020 Dose 2 administered orally, once a day (QD) for 26 weeks. Receives: Drug: ONO-2020 |
| Placebo | Placebo comparator | Participants will receive Placebo administered orally, once a day (QD) for 26 weeks. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence, severity, and type of treatment emergent adverse events (TEAEs)
The number and percentage of subjects reporting each TEAE will be summarized by both system organ class (SOC) and preferred term (PT).
Measured: From baseline up to 26 weeks
Clinically abnormal findings in Columbia Suicide Severity Rating Scale (C-SSRS)
The number and percentage of subjects with clinically abnormal finding will be tabulated at each time point.
Measured: From baseline up to 26 weeks
Change from baseline through week 26 in Alzheimer's Disease Assessment Scale-Cognitive Subscale 12 (ADAS-cog 12) score
Measured: From baseline up to 26 weeks
Secondary outcomes (10)
Change from baseline through week 26 in ADAS-cog 12 score in mild AD participants
Measured: From baseline up to 26 weeks
Change from baseline through week 26 in ADAS-cog 12 score in moderate AD participants
Measured: From baseline up to 26 weeks
Change from baseline through week 26 in ADAS-cog 11 and 13 scores
Measured: From baseline up to 26 weeks
Change from baseline through week 26 in Alzheimer's Disease Cooperative Study Group-Activities of Daily Living (ADCS-ADL) score
Measured: From baseline up to 26 weeks
Change from baseline through week 26 in Quick Dementia Rating System (QDRS)
Measured: From baseline up to 26 weeks
Change from baseline through week 26 in Mini-Mental State Examination (MMSE) score
Measured: From baseline up to 26 weeks
Change from baseline through week 26 in Neuropsychiatric Inventory Questionnaire (NPI-Q)
Measured: From baseline up to 26 weeks
Change from baseline through week 26 in Quality of Life-Alzheimer's Disease (QoL-AD) Scale
Measured: From baseline up to 26 weeks
Change from baseline through week 26 in Zarit Burden Interview Scale (ZBI)
Measured: From baseline up to 26 weeks
Change in plasma concentration of ONO-2020 by dose level and time point
The plasma concentrations of ONO-2020 will be listed, and descriptive summary statistics of them will be calculated by dose level and time point.
Measured: Day 1, Week 2, Week 10, and Week 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2025
- Study start24 Apr 2025
- Primary completion31 Jul 2026
- Study completion1 Sep 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alzheimer DiseaseStudies of the same condition, open and under way.
- More trials run by Ono Pharmaceutical Co., Ltd.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 260 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ono Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Project Leader, Study director, Ono Pharmaceutical Co., Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06881836
- Sponsor's protocol ID
- ONO-2020-02
- Other IDs
- jRCT2031240712
- Status last verified
- Oct 2026
- First submitted
- 2 Mar 2025
- Last updated
- 22 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No