NCT01862640

Brief Summary

To compare the efficacy of 2 fixed doses of brexpiprazole with placebo in participants with agitation associated with dementia of the Alzheimer's type.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
433

participants targeted

Target at P50-P75 for phase_3

Timeline
Completed

Started Jul 2013

Typical duration for phase_3

Geographic Reach
7 countries

85 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 22, 2013

Completed
2 days until next milestone

First Posted

Study publicly available on registry

May 24, 2013

Completed
2 months until next milestone

Study Start

First participant enrolled

July 11, 2013

Completed
3.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 15, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 15, 2017

Completed
3.3 years until next milestone

Results Posted

Study results publicly available

June 16, 2020

Completed
Last Updated

December 31, 2020

Status Verified

December 1, 2020

Enrollment Period

3.7 years

First QC Date

May 22, 2013

Results QC Date

April 16, 2020

Last Update Submit

December 8, 2020

Conditions

Keywords

OPC-34712brexpiprazoleDementiaAlzheimer's DiseaseCognitive DisordersAgitation

Outcome Measures

Primary Outcomes (1)

  • Change From Baseline In The Cohen-Mansfield Agitation Inventory (CMAI) Total Score After 12 Weeks Of Brexpiprazole Treatment

    To compare the efficacy of 2 fixed doses (1 mg/day and 2 mg/day) of brexpiprazole with placebo in participants with agitation associated with dementia of the Alzheimer's type, by the assessment of CMAI after 12 weeks of treatment. The CMAI assesses the frequency of agitated behaviors in elderly persons, such as hitting, cursing, and restlessness. It consists of 29 items all rated on a 1 to 7 scale with 1 being the "best" rating and 7 being the "worst" rating. The minimum possible CMAI total score is 29, and the maximum possible CMAI total score is 203. A decrease in score indicates improvement in symptoms. To control the overall type I error at 0.05 level when making 2 comparisons of brexpiprazole doses versus placebo, statistical testing was carried out using a hierarchical testing procedure in the order of: 1) comparison of 2 mg/day brexpiprazole versus placebo, and 2) comparison of 1 mg/day brexpiprazole versus placebo.

    Baseline, Week 12/Early Termination (ET)

Secondary Outcomes (1)

  • Change From Baseline In The Clinical Global Impression-Severity Of Illness (CGI-S) Score, As Related To Symptoms Of Agitation After 12 Weeks Of Brexpiprazole Treatment

    Baseline, Week 12/ET

Study Arms (3)

Placebo

PLACEBO COMPARATOR

Matching placebo once daily

Drug: Placebo Oral Tablet

Brexpiprazole 1 mg

EXPERIMENTAL

Titrate up from 0.25 milligrams (mg)/day brexpiprazole to 1 mg/day brexpiprazole

Drug: Brexpiprazole, OPC-34712

Brexpiprazole 2 mg

EXPERIMENTAL

Titrate up from 0.25 mg/day brexpiprazole to 2 mg/day brexpiprazole

Drug: Brexpiprazole, OPC-34712

Interventions

Once-daily, tablets

Brexpiprazole 1 mgBrexpiprazole 2 mg

Once-daily, tablets

Placebo

Eligibility Criteria

Age55 Years - 90 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Male and female participants 55 to 90 years of age, inclusive, at the time of informed consent.
  • Participants who are residing at their current location for at least 14 days before screening and are expected to remain at the same location for the duration of the trial.
  • Participants with a diagnosis of probable Alzheimer's disease according to National Institute of Neurological and Communicative Disorders and Stroke and the Alzheimer's Disease and Related Disorders Association.
  • Participants with a Mini-Mental State Exam score of 5 to 22, inclusive, at screening and baseline visits.
  • Participants with onset of symptoms of agitation at least 2 weeks prior to the screening visit.
  • Participants with a score of ≥ 4 on the agitation/aggression item of the Neuropsychiatric Inventory-Nursing Home at the screening and baseline visits.
  • Participants who require pharmacotherapy for treatment of agitation per the investigator's judgment, after an evaluation for reversible factors (for example, pain, infection, polypharmacy) and a trial of nonpharmacological intervention.
  • Participants must have a previous magnetic resonance imaging or computed tomography of the brain, which was performed after the onset of symptoms of dementia, with findings consistent with the diagnosis of Alzheimer's disease.

You may not qualify if:

  • Participants with dementia or other memory impairment not due to Alzheimer's disease
  • Participants with a history of stroke, well-documented transient ischemic attack, pulmonary or cerebral embolism.
  • Participants who currently have clinically significant neurological, hepatic, renal, metabolic, hematological, immunological, cardiovascular, pulmonary, gastrointestinal, or psychiatric disorders.
  • Participants who have been diagnosed with an Axis I disorder (Diagnostic and Statistical Manual of Mental Disorders, 4th edition, text revision criteria)
  • Participants with uncontrolled hypertension
  • Participants with uncontrolled insulin-dependent diabetes mellitus
  • Participants with epilepsy or a history of seizures
  • Participants considered in poor general health based on the investigator's judgment.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (85)

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Tuscaloosa, Alabama, 35404, United States

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Phoenix, Arizona, 85013, United States

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Bellflower, California, 90706, United States

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Costa Mesa, California, 92627, United States

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Downey, California, 90706, United States

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Orange, California, 92868, United States

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Redlands, California, 92373, United States

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Yorba Linda, California, 92886, United States

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Norwalk, Connecticut, 06851, United States

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Coconut Creek, Florida, 33024, United States

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Hialeah, Florida, 33012, United States

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Hialeah, Florida, 33013, United States

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Hialeah, Florida, 33018, United States

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Lauderhill, Florida, 33319, United States

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Miami, Florida, 33122, United States

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Miami, Florida, 33126, United States

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Miami, Florida, 33136, United States

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Miami, Florida, 33137, United States

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Miami, Florida, 33145, United States

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Miami, Florida, 33161, United States

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Miami, Florida, 33174, United States

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Miami Springs, Florida, 33166, United States

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Sarasota, Florida, 34239, United States

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Decatur, Georgia, 30033, United States

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Suwanee, Georgia, 30024, United States

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Weymouth, Massachusetts, 02190, United States

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St Louis, Missouri, 63128, United States

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St Louis, Missouri, 63141, United States

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Las Vegas, Nevada, 89148, United States

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Mount Arlington, New Jersey, 07856, United States

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Paterson, New Jersey, 08759, United States

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Toms River, New Jersey, 08755, United States

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Hickory, North Carolina, 28601, United States

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Oklahoma City, Oklahoma, 73118, United States

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Franklin, Tennessee, 37064, United States

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Bedford, Texas, 76022, United States

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Waukesha, Wisconsin, 53188, United States

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Rijeka, 57000, Croatia

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Zadar, 23000, Croatia

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Zagreb, 10000, Croatia

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Zagreb, 10090, Croatia

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Mittweida, Saxony, 09648, Germany

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Achim, 28832, Germany

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Berlin, 12209, Germany

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Bielefeld, 33647, Germany

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Bochum, 44791, Germany

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Cologne, 50935, Germany

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Hamburg, 22083, Germany

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Ostfildern, 73760, Germany

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Westerstede, 26655, Germany

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Saint Petersburg, 190000, Russia

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Saint Petersburg, 190005, Russia

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Saint Petersburg, 191119, Russia

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Saint Petersburg, 192019, Russia

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Saint Petersburg, 197341, Russia

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Saint Petersburg, 198510, Russia

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Samara, 443016, Russia

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Saratov, 410060, Russia

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Tonnel’nyy, 357034, Russia

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Yekaterinburg, 620030, Russia

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Belgrade, 11000, Serbia

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Kovin, 26220, Serbia

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Kragujevac, 34000, Serbia

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Niš, 18000, Serbia

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Novi Kneževac, 23330, Serbia

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Novi Sad, 21000, Serbia

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Vršac, 26300, Serbia

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Barcelona, 08028, Spain

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Getafe, 28905, Spain

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Girona, 17190, Spain

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Madrid, 28034, Spain

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Madrid, 28040, Spain

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Madrid, 28049, Spain

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Pamplona, 31014, Spain

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Salamanca, 37003, Spain

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Valencia, 46010, Spain

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Valencia, 46026, Spain

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Zamora, 49021, Spain

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Kharkiv, 61068, Ukraine

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Kherson, 73488, Ukraine

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Kiev, 04080, Ukraine

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Lviv, 79021, Ukraine

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Odesa, 65006, Ukraine

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Odesa, 67513, Ukraine

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Poltava, 36013, Ukraine

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Related Publications (7)

  • Cummings JL, Chumki SR, Chang D, Zhang Z, Brubaker M, Hefting N, Such P, Wang D, Grossberg GT. Efficacy of Brexpiprazole in Participants with Agitation Associated with Dementia Due to Alzheimer's Disease: Pooled Analysis of Randomized Controlled Trials. Clin Drug Investig. 2026 Jan 27. doi: 10.1007/s40261-025-01517-9. Online ahead of print.

  • Shah A, Kalu U, Chen D, Slomkowski M, Hobart M, Such P, Grossberg GT. Brexpiprazole side-effect profile in people with agitation in Alzheimer's dementia: a plain language summary. Curr Med Res Opin. 2026 Jan 14:1-3. doi: 10.1080/03007995.2025.2608578. Online ahead of print.

  • Grossberg GT, Sabbagh MN, Chumki SR, Wang D, Such P, Zhang Z, Palma AM, Cummings JL. Efficacy of Brexpiprazole on Neuropsychiatric Symptoms and Impact on Caregivers: Pooled Neuropsychiatric Inventory (NPI) Analysis in Patients With Agitation Associated With Dementia due to Alzheimer's Disease. J Geriatr Psychiatry Neurol. 2025 Dec 24:8919887251407124. doi: 10.1177/08919887251407124. Online ahead of print.

  • Stroud J, Cummings JL, Chumki SR, Such P, Wang D, Palma AM, Zhang Z, Brubaker M, Grossberg GT. Brexpiprazole for agitation in clinically relevant patient subgroups: a post hoc analysis of efficacy and safety in patients with agitation associated with dementia due to Alzheimer's disease. Curr Med Res Opin. 2025 Aug;41(8):1523-1534. doi: 10.1080/03007995.2025.2552278. Epub 2025 Sep 5.

  • Shah A, Estilo A, Sheridan PL, Kalu U, Chen D, Chang D, Slomkowski M, Lee D, Hefting N, Hobart M, Behl S, Such P, Brubaker M, Grossberg GT. Safety and Tolerability of Brexpiprazole in Participants with Agitation Associated with Dementia due to Alzheimer's Disease: Pooled Analysis of Three Randomized Trials and an Extension Trial. CNS Drugs. 2025 Oct;39(10):1011-1023. doi: 10.1007/s40263-025-01200-9. Epub 2025 Jul 19.

  • Meunier J, Creel K, Loubert A, Larsen KG, Aggarwal J, Hefting N, Oberdhan D. Defining a clinically meaningful within-patient change threshold for the Cohen-Mansfield Agitation Inventory in Alzheimer's dementia. Front Neurol. 2024 Jul 23;15:1379062. doi: 10.3389/fneur.2024.1379062. eCollection 2024.

  • Grossberg GT, Kohegyi E, Mergel V, Josiassen MK, Meulien D, Hobart M, Slomkowski M, Baker RA, McQuade RD, Cummings JL. Efficacy and Safety of Brexpiprazole for the Treatment of Agitation in Alzheimer's Dementia: Two 12-Week, Randomized, Double-Blind, Placebo-Controlled Trials. Am J Geriatr Psychiatry. 2020 Apr;28(4):383-400. doi: 10.1016/j.jagp.2019.09.009. Epub 2019 Oct 1.

MeSH Terms

Conditions

Alzheimer DiseaseMental DisordersNervous System DiseasesDementiaCognitive DysfunctionPsychomotor Agitation

Interventions

brexpiprazole

Condition Hierarchy (Ancestors)

Brain DiseasesCentral Nervous System DiseasesTauopathiesNeurodegenerative DiseasesNeurocognitive DisordersCognition DisordersDyskinesiasNeurologic ManifestationsPsychomotor DisordersNeurobehavioral ManifestationsSigns and SymptomsPathological Conditions, Signs and SymptomsAberrant Motor Behavior in DementiaBehavioral SymptomsBehavior

Limitations and Caveats

None reported

Results Point of Contact

Title
Global Clinical Development
Organization
Otsuka Pharmaceutical Development & Commercialization, Inc.

Study Officials

  • Eva Kohegyi, MD

    Otsuka Pharmaceutical Development & Commercialization, Inc.

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 22, 2013

First Posted

May 24, 2013

Study Start

July 11, 2013

Primary Completion

March 15, 2017

Study Completion

March 15, 2017

Last Updated

December 31, 2020

Results First Posted

June 16, 2020

Record last verified: 2020-12

Data Sharing

IPD Sharing
Will share

Anonymized Individual participant data (IPD) that underlie the results of this study will be shared with researchers to achieve aims pre-specified in a methodologically sound research proposal. Small studies with less than 25 participants are excluded from data sharing.

Shared Documents
STUDY PROTOCOL, SAP, CSR
Time Frame
Data will be available after marketing approval in global markets, or beginning 1-3 years following article publication. There is no end date to the availability of the data.
Access Criteria
Otsuka will share data on an Otsuka-owned remotely accessible data sharing platform with Python and R analytical software. Research requests should be directed to clinicaltransparency@Otsuka-us.com

Locations