IRB Study Number 25-528
Status Recruiting
Phase Phase 2
Locations U Building - Mellen Center, Cleveland Clinic Main Campus
Institute Neurological Institute
Description
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.
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 (every day) 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.
Inclusion Criteria
- Have a diagnosis of Alzheimer's disease.
- Have a previous MRI or CT scan of the brain, which was performed within 1 year prior to enrollment.
- MMSE score of 15 to 24, inclusive.
- AD numeric clinical stage 4 or stage 5 at screening and baseline visits.
- Participants receiving concurrent AD treatment must be on a stable dose for at least 90 days prior to randomization.
- Have the ability to comply with procedures for cognitive and other tests in the opinion of the investigator.
- 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 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 to provide support to the participant to ensure compliance with study requirements.
- General health status acceptable for participation in the study, and the participant must be able to ingest pills.
- Capacity to provide consent or have a legally authorized representative (LAR) to sign on their behalf.
Exclusion Criteria
- Participants with dementia or other memory impairment not due to Alzheimer's disease.
- Participants with a history of stroke, well-documented transient ischemic attack, or pulmonary or cerebral embolism.
- History of significant psychiatric illness or history or current major depressive disorder in the past year.
- Participants with delirium or history of delirium within the 30 days prior to the screening visit.
- Have suicide ideation or have made a suicide attempt in the 6 months prior to screening.
- Clinically significant ECG abnormality as judged by the investigator.
- Confirmed absolute QTcF >450 msec for males or >470 msec for females.
- Positive results at screening for active viral infections.
- Participants with elevated total bilirubin, alanine transaminase (ALT) or aspartate transaminase (AST) at screening.
- Participants with estimated creatinine clearance ≤30 mL/min at screening.
- Participants with a history of treatment, and/or current treatment, with anti-Aβ antibodies
- Changes in any medications that may potentially impair participants' ability to perform cognitive testing or study procedures during the study period.
- 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.
