CompletedPhase 2Results posted

Efficacy and Safety Study of ELND005 as a Treatment for Agitation and Aggression in Alzheimer's Disease

A Prospective, Randomized, Double-Blind, Placebo-Controlled, Phase 2 Efficacy and Safety Study of Oral ELND005 for Treatment of Agitation and Aggression in Patients With Moderate to Severe Alzheimer's Disease

Lead sponsor

OPKO Health, Inc.

Asset

ELND005

Listed sites

Recruiting sites

Enrollment

350

actual

Study population

Alzheimer’s disease

Key I/E criteria

•Alzheimer's disease•MMSE 5-24

Primary endpoint

•Neuropsychiatric Inventory (NPI)

Footprint

Where this trial recruits

Site locations as reported to ClinicalTrials.gov. Site count is not enrollment count; per-site enrollment is not available from source.

Identifiers

Registered as

Eudract number2012-004299-20

Org study IDELND005-AG201

NCT IDNCT01735630

Timeline

Milestones

Study start2012-11 (month precision)

Study first posted2012-11-28estimated

Primary completion2015-05actual (month precision)

Study completion2015-05actual (month precision)

Results first posted2016-08-15estimated

Last update posted2019-10-21actual

Assets

Drug assets

ELND005

Study populations

Who this study enrolls

Alzheimer’s disease

Eligibility

Who can enroll

Minimum age50 Years

Maximum age95 Years

SexAll

Healthy volunteersNot accepted

Inclusion criteria

•Diagnosis of Probable AD according to the National Institute on Aging- Alzheimer's Association (NIA-AA) guidelines (McKhann et al 2011).

•MMSE score of 5 to 24 (inclusive) at the Screening Visit.

•Has clinically significant agitation/aggression defined as Neuropsychiatric Inventory (NPI)-agitation/aggression subscore of ≥4.

•No response or suboptimal response to standard nonpharmacological interventions

Exclusion criteria

•The agitation/aggression is attributable to concomitant medications, environmental conditions, or active medical or psychiatric condition.

•Current diagnosis of major depressive disorder according to the criteria of the Diagnostic and Statistical Manual of Mental Disorders Fourth Edition Text Revision (DSM IV TR).

•Has persistent and distressing psychotic symptoms (delusion and/or hallucinations) that require psychiatric hospitalization.

Endpoints (10)

What's being measured

Protocol endpoints and posted registry outcome measures, grouped into outcome categories. Composite endpoints show their component event types. Standard codes (LOINC, SNOMED CT) are shown where available.

Coverage by outcome category

Behavior / neuropsychiatric

Global cognition

Function / daily living

Global cognition

2 endpoints

Secondary/protocol endpoint

Change From Baseline in MMSE Scores

Time frame:Week 12

Mini-Mental State Examination (MMSE)

change from baseline, improvement

Secondary/registry result

Change From Baseline in MMSE Scores

Time frame:Week 12

Mini-Mental State Examination (MMSE)

change from baseline, improvement

Posted result

Group	Value (mean), units on a scale	Standard error
ELND005n=157 Participants	-0.3	0.2
Placebon=154 Participants	-0.4	0.2

Function / daily living

2 endpoints

Secondary/protocol endpoint

Change From Baseline in ADCS-ADL Scores

Time frame:Week 12

ADCS-Activities of Daily Living (ADCS-ADL)

change from baseline, improvement

Secondary/registry result

Change From Baseline in ADCS-ADL Scores

Time frame:Week 12

ADCS-Activities of Daily Living (ADCS-ADL)

change from baseline, improvement

Posted result

Group	Value (mean), units on a scale	Standard error
ELND005n=156 Participants	-1.458	0.7
Placebon=155 Participants	-2.229	0.7

Behavior / neuropsychiatric

6 endpoints

Primary/protocol endpoint

Change From Baseline in NPI-C Combined Agitation and Aggression Subscores (NPI-C A+A).

Time frame:Week 12

Neuropsychiatric Inventory (NPI)

change from baseline, improvement

Primary/registry result

Change From Baseline in NPI-C Combined Agitation and Aggression Subscores (NPI-C A+A).

Time frame:Week 12

Neuropsychiatric Inventory (NPI)

change from baseline, improvement

Posted result

Group	Value (mean), units on a scale	Standard error
ELND005n=157 Participants	-4.3	0.7
Placebon=155 Participants	-4.2	0.7

Secondary/protocol endpoint

Change From Baseline in Modified-ADCS-CGIC Agitation Scores

Time frame:Week 12

change from baseline, improvement

Secondary/protocol endpoint

Change From Baseline in NPI Total Scores

Time frame:Week 12

Neuropsychiatric Inventory (NPI)

change from baseline, improvement

Secondary/registry result

Change From Baseline in Modified-ADCS-CGIC Agitation Scores

Time frame:Week 12

change from baseline, improvement

Posted result

Group	Value (mean), units on a scale	Standard error
ELND005n=156 Participants	3.6	0.1
Placebon=157 Participants	3.4	0.1

Secondary/registry result

Change From Baseline in NPI Total Scores

Time frame:Week 12

Neuropsychiatric Inventory (NPI)

change from baseline, improvement

Posted result

Group	Value (mean), units on a scale	Standard error
ELND005n=157 Participants	-14.38	1.69
Placebon=154 Participants	-15.12	1.71

Provenance

Sources

Trial identity, design, statusClinicalTrials.gov API v2

Snapshot dateJuly 21, 2026

Endpoint classificationDelfa ADRD endpoint taxonomy

Results tableClinicalTrials.gov results section

Trial facts come from public ClinicalTrials.gov records. Endpoint categories are Delfa's classification of those records, not a ClinicalTrials.gov field. All figures reflect the July 21, 2026 snapshot.