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TerminatedPhase 2Results posted

Dose-Finding Study To Evaluate Safety, Tolerability, and Efficacy of E2609 in Participants With Mild Cognitive Impairment Due to Alzheimer's Disease (Prodromal Alzheimer's Disease) and Mild to Moderate Dementia Due to Alzheimer's Disease

A Placebo-Controlled, Double-Blind, Parallel-Group, Randomized, Proof-of-Concept, Dose-Finding Study To Evaluate Safety, Tolerability, and Efficacy of E2609 in Subjects With Mild Cognitive Impairment Due to Alzheimer's Disease (Prodromal Alzheimer's Disease) and Mild to Moderate Dementia Due to Alzheimer's Disease

Lead sponsor

Eisai Inc.

Asset

Elenbecestat

Listed sites

21

Recruiting sites

-

Enrollment

70

actual

Study population

Alzheimer’s disease, MCI / preclinical Alzheimer’s

Key I/E criteria

MCI due to AD / prodromal ADAmyloid biomarker required (PET)Study partner/caregiver requiredBackground AD symptomatic therapy, if used: stable ≥12 weeks

Primary endpoints

Core PhaseTEAEs and SAEsMarkedly Abnormal Vital Sign Values

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 number2014-002723-94
Org study IDE2609-G000-202
NCT IDNCT02322021

Timeline

Milestones

Study start2014-11-26actual
Study first posted2014-12-22estimated
Primary completion2019-12-20actual
Study completion2019-12-20actual
Last update posted2021-03-05actual
Results first posted2021-03-05actual

Assets

Drug assets

Study populations

Who this study enrolls

Alzheimer’s diseaseMCI / preclinical Alzheimer’s

Eligibility

Who can enroll

Minimum age50 Years
Maximum age85 Years
SexAll
Healthy volunteersNot accepted

Inclusion criteria

Participants must meet all of the following criteria to be included in this study:

1. Meets the core clinical research criteria of the National Institute on Aging-Alzheimer's Association (NIA-AA) for MCI due to AD (which is consistent with Prodromal AD) or AD dementia and is 'staged' or classified as either MCI or mild to moderate dementia.

2. Amyloid positive Positron Emission Tomography (PET) image based on centralized PET scan reading.

3. Male or female, age 50 to 85 years, inclusive at time of consent.

4. Must have an identified caregiver or informant who is willing and able to provide follow-up information on the participant throughout the course of the study.

5. If receiving an Acetylcholinesterase Inhibitor (AChEI) or memantine, must have been on a stable dose for at least 12 weeks before the Baseline cognitive assessments, with no plans for dose adjustment in the foreseeable future. Treatment-naive participants can be entered into the study but there should be no plans to initiate treatment with AChEIs or memantine at the time of entry into the study.

6. Must have been on stable doses of all other permitted chronically used concomitant medications (that is, not related to their cognitive decline) for at least 4 weeks before randomization

Exclusion criteria

Participants who meet any of the following criteria will be excluded from this study:

1. Any neurological condition that may be contributing to cognitive impairment above and beyond that caused by the participant's AD pathology, including any co-morbidities such as cerebrovascular disease, detected by medical history, neurological examination, or magnetic resonance imaging (MRI).

2. History of transient ischemic attacks or stroke within 12 months of Screening.

3. History of epilepsy.

4. Evidence of depression on a rating scale at Screening or any psychiatric diagnosis or symptoms, (example, hallucinations, major depression, etc.) that could confound the diagnosis or could interfere with study assessments or procedures. This includes suicidal ideation or suicidal behavior within 6 months prior to Screening, or hospitalization or treatment for suicidal behavior in the past 5 years.

5. Abnormally low serum vitamin B12.

6. Thyroid stimulating hormone above the normal range. This applies to all participants regardless of whether or not they are taking thyroid supplements.

7. Participants with liver disease (hepatic impairment), at Screening or Baseline. Participant with Gilbert's syndrome need not be excluded.

8. Not able to have a MRI, PET scanning, or cerebrospinal fluid (CSF) collection by Lumbar Puncture (LP).

9. Severe visual or hearing impairment that would prevent the participant from performing psychometric tests accurately.

10. History of immunodeficiency disorders.

11. Participants with chronic viral hepatitis.

12. History of Tuberculosis (TB). Participants with no history of TB will be tested for previous TB exposure and a positive test will be exclusionary.

13. History of ophthalmic shingles or ocular Herpes Simplex Virus (HSV) infection.

14. Any live vaccine in the 3 months or any active infection within the last 4 weeks before study drug administration (that is, randomization).

15. Any chronic inflammatory disease that is not adequately controlled or requires immunosuppressive or immunomodulatory therapy.

16. T helper cell, cytotoxic T cell, or B cell absolute counts below normal.

17. Immunoglobulin (Ig) IgG, IgA, or IgM levels below normal at Screening or Baseline, unless both the Investigator and the Medical Monitor agree that the finding is not clinically significant.

18. Clinically significant deviation from normal in physical examination, vital signs, or clinical laboratory tests at Screening or Baseline.

19. Exclusionary cardiac factors include: prolonged QT interval greater than 450 millisecond from Electrocardiograms (ECGs); history of risk factors for torsade de pointes or the use of concomitant medications that prolong the QT/corrected QT interval (QTc); left bundle branch block; persistent low or high heart rate; persistent low or high blood pressure; history of cardiac arrhythmias; other clinically significant ECG abnormalities.

20. Type 1 or Type 2 diabetes mellitus that is not well controlled.

21. Malignant neoplasms within 5 years before Screening (except for basal or squamous cell carcinoma in situ of the skin, or localized prostate cancer that did not require systemic therapy; these do not exclude the participant).

22. Medical conditions (example, cardiac, respiratory, gastrointestinal, renal disease) that are not stably controlled, or which, in the opinion of the investigator(s), could affect the participant's safety or interfere with the study assessments.

23. Hypopigmentation conditions (example, albinism and vitiligo).

24. Known or suspected history of drug or alcohol dependency or abuse within 2 years, current use of recreational drugs or a positive urine drug test.

25. Planned surgery that requires general, spinal, or epidural anesthesia that would take place during the study.

26. Participation in any other interventional clinical study related to cognitive impairment within 6 months before Screening unless it can be documented that the participant was in a placebo treatment arm.

27. Currently enrolled in another clinical study or used any investigational drug or device within 60 days or 5 half-lives of the investigational medication (whichever is longer) proceeding informed consent.

28. Hypersensitivity to the study drug or any of the excipients or to other Beta Secretase Cleaving Enzyme (BACE) inhibitors, or to the PET tracer, or components of its formulation.

29. Females who are lactating or pregnant. Females of childbearing potential who do not agree to adhere to the protocol specified methods for avoiding pregnancy.

30. Males who do not meet the protocol requirements for avoiding their partners becoming pregnant. Sperm donation is not permitted.

Endpoints (40)

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

Safety / tolerability / PK
14
Neuroimaging
12
Amyloid biomarkers
4
Other (unclassified)
4
Global cognition
2
Function / daily living
2
Fluid / digital biomarkers
2

Global cognition

2 endpoints
Secondary/protocol endpoint

Extension Phase: Change From Extension Phase Baseline in the Mini-Mental State Examination (MMSE) Scores

Time frame:Baseline, at Month 3, at Month 6, at Month 9, at Month 12, at Month 15, at Month 18, at Month 21, at Month 24, at Month 28 and at Month 32

Mini-Mental State Examination (MMSE)

ratio, descriptive

Secondary/registry result

Extension Phase: Change From Extension Phase Baseline in the Mini-Mental State Examination (MMSE) Scores

Time frame:Baseline, at Month 3, at Month 6, at Month 9, at Month 12, at Month 15, at Month 18, at Month 21, at Month 24, at Month 28 and at Month 32

Mini-Mental State Examination (MMSE)

ratio, descriptive

Posted result

GroupValue (mean), score on a scaleStandard deviation
Extension Phase: Elenbecestat 50 mgBaselinen=41 Participants21.55.67
Change at Month 3n=41 Participants-0.62.32
Change at Month 6n=39 Participants-0.62.39
Change at Month 9n=36 Participants-0.92.89
Change at Month 12n=36 Participants-1.42.43
Change at Month 15n=33 Participants-1.82.78
Change at Month 18n=32 Participants-1.73.01
Change at Month 21n=21 Participants-2.53.27
Change at Month 24n=10 Participants-3.94.46
Change at Month 28n=5 Participants-6.44.28
Change at Month 32n=1 Participants-2.0NA

Function / daily living

2 endpoints
Secondary/protocol endpoint

Extension Phase: Change From Extension Phase Baseline in the Functional Assessment Questionnaire (FAQ) Score

Time frame:Baseline, at Month 3, at Month 6, at Month 9, at Month 12, at Month 15, at Month 18, at Month 21, at Month 24, at Month 28 and at Month 32

descriptive

Secondary/registry result

Extension Phase: Change From Extension Phase Baseline in the Functional Assessment Questionnaire (FAQ) Score

Time frame:Baseline, at Month 3, at Month 6, at Month 9, at Month 12, at Month 15, at Month 18, at Month 21, at Month 24, at Month 28 and at Month 32

descriptive

Posted result

GroupValue (mean), score on a scaleStandard deviation
Extension Phase: Elenbecestat 50 mgBaselinen=41 Participants14.47.31
Change at Month 3n=41 Participants1.35.51
Change at Month 6n=39 Participants1.43.74
Change at Month 9n=35 Participants2.24.33
Change at Month 12n=36 Participants2.64.86
Change at Month 15n=34 Participants3.05.17
Change at Month 18n=32 Participants3.44.83
Change at Month 21n=22 Participants3.03.88
Change at Month 24n=10 Participants6.85.92
Change at Month 28n=6 Participants9.24.67
Change at Month 32n=1 Participants15.0NA

Amyloid biomarkers

4 endpoints
Secondary/protocol endpoint

Core Phase: Percent Change From Baseline in Cerebrospinal Fluid (CSF) Amyloid (A) Beta(1-x) and Abeta(1-42) After 1 Month and 18 Months of Treatment

Time frame:Month 1 (Week 5) and Month 18 (Week 79)

percent change from baseline, improvement

Secondary/protocol endpoint

Extension Phase: Percent Change From Extension Phase Baseline in Plasma Amyloid (A) Beta(1-x) Measurements at Months 12 and 24

Time frame:Month 12 and Month 24

percent change from baseline, improvement

Secondary/registry result

Core Phase: Percent Change From Baseline in Cerebrospinal Fluid (CSF) Amyloid (A) Beta(1-x) and Abeta(1-42) After 1 Month and 18 Months of Treatment

Time frame:Month 1 (Week 5) and Month 18 (Week 79)

percent change from baseline, improvement

Posted result

GroupValue (mean), percent changeStandard deviation
Core Phase: PlaceboPercent Change from Baseline in CSF Abeta(1-x) at Month 1 (Week 5)n=4 Participants6.6312.545
Percent Change from Baseline in CSF Abeta(1-x) at Month 18 (Week 79)n=1 Participants-1.81NA
Percent Change from Baseline in CSF Abeta(1-42) at Month 1 (Week 5)n=4 Participants-8.5316.818
Percent Change from Baseline in CSF Abeta(1-42) at Month 18 (Week 79)n=2 Participants-6.5024.809
Core Phase: Elenbecestat 5 mg Then 50 mgPercent Change from Baseline in CSF Abeta(1-x) at Month 1 (Week 5)n=7 Participants-18.1617.873
Percent Change from Baseline in CSF Abeta(1-x) at Month 18 (Week 79)n=2 Participants-33.8611.039
Percent Change from Baseline in CSF Abeta(1-42) at Month 1 (Week 5)n=7 Participants-9.4322.798
Percent Change from Baseline in CSF Abeta(1-42) at Month 18 (Week 79)n=2 Participants-27.1920.172
Core Phase: Elenbecestat 15 mg Then 50 mgPercent Change from Baseline in CSF Abeta(1-x) at Month 1 (Week 5)n=6 Participants-36.4316.026
Percent Change from Baseline in CSF Abeta(1-x) at Month 18 (Week 79)n=1 Participants-0.71NA
Percent Change from Baseline in CSF Abeta(1-42) at Month 1 (Week 5)n=6 Participants-20.6915.488
Percent Change from Baseline in CSF Abeta(1-42) at Month 18 (Week 79)n=3 Participants-24.0219.949
Core Phase: Elenbecestat 50 mgPercent Change from Baseline in CSF Abeta(1-x) at Month 1 (Week 5)n=6 Participants-56.6614.431
Percent Change from Baseline in CSF Abeta(1-x) at Month 18 (Week 79)n=2 Participants33.98160.017
Percent Change from Baseline in CSF Abeta(1-42) at Month 1 (Week 5)n=6 Participants-38.8916.378
Percent Change from Baseline in CSF Abeta(1-42) at Month 18 (Week 79)n=4 Participants-56.775.312
Secondary/registry result

Extension Phase: Percent Change From Extension Phase Baseline in Plasma Amyloid (A) Beta(1-x) Measurements at Months 12 and 24

Time frame:Month 12 and Month 24

percent change from baseline, improvement

Posted result

GroupValue (mean), percent changeStandard deviation
Extension Phase: Elenbecestat 50 mgPercent Change from Extension Phase Baseline in plasma Abeta(1-x) at Month 12n=27 Participants-71.823.30
Percent Change from Extension Phase Baseline in plasma Abeta(1-x) at Month 24n=5 Participants-71.131.20

Neuroimaging

12 endpoints
Primary/protocol endpoint

Extension Phase: Number of Participants With TEAEs and SAEs

Time frame:Up to 34 months

event count, event

Primary/registry result

Extension Phase: Number of Participants With TEAEs and SAEs

Time frame:Up to 34 months

event count, event

Posted result

GroupValue (count_of_participants), ParticipantsReported bounds
Extension Phase: Elenbecestat 50 mgTEAEsn=41 Participants30-
SAEsn=41 Participants6-
Primary/protocol endpoint

Extension Phase: Number of Participants With Abnormal Magnetic Resonance Imaging (MRI) Findings

Time frame:Up to 34 months

event count, event

Primary/registry result

Extension Phase: Number of Participants With Abnormal Magnetic Resonance Imaging (MRI) Findings

Time frame:Up to 34 months

event count, event

Posted result

GroupValue (count_of_participants), ParticipantsReported bounds
Extension Phase: Elenbecestat 50 mgBrain Vasogenic Edeman=27 Participants0-
Brain Microhemorrhagesn=23 Participants4-
Brain White Matter Disease: Focal Lesionsn=27 Participants25-
Brain White Matter Disease: No Lesionsn=27 Participants2-
Area of superficial siderosisn=41 Participants1-
Space occupying lesion (extra axial): Meningioman=41 Participants1-
Other pituitary lesionn=41 Participants1-
Secondary/protocol endpoint

Extension Phase: Percent Change From Extension Phase Baseline in Total Hippocampal Volume at Month 24

Time frame:Month 24

percent change from baseline, improvement

Secondary/protocol endpoint

Extension Phase: Percent Change From Extension Phase Baseline in Left and Right Hippocampal Volume at Month 24

Time frame:Month 24

percent change from baseline, improvement

Secondary/protocol endpoint

Extension Phase: Percent Change From Extension Phase Baseline in Whole Brain Volume at Month 24

Time frame:Month 24

percent change from baseline, improvement

Secondary/protocol endpoint

Extension Phase: Percent Change From Extension Phase Baseline in Total Ventricular Volume at Month 24

Time frame:Month 24

percent change from baseline, improvement

Secondary/registry result

Extension Phase: Percent Change From Extension Phase Baseline in Total Hippocampal Volume at Month 24

Time frame:Month 24

percent change from baseline, improvement

Posted result

GroupValue (mean), percent changeStandard deviation
Extension Phase: Elenbecestat 50 mgn=12 Participants-4.332.080
Secondary/registry result

Extension Phase: Percent Change From Extension Phase Baseline in Left and Right Hippocampal Volume at Month 24

Time frame:Month 24

percent change from baseline, improvement

Posted result

GroupValue (mean), percent changeStandard deviation
Extension Phase: Elenbecestat 50 mgPercent Change at Month 24 (Left Hippocampal Volume)n=12 Participants-4.312.065
Percent Change at Month 24 (Right Hippocampal Volume)n=12 Participants-4.372.667
Secondary/registry result

Extension Phase: Percent Change From Extension Phase Baseline in Whole Brain Volume at Month 24

Time frame:Month 24

percent change from baseline, improvement

Posted result

GroupValue (mean), percent changeStandard deviation
Extension Phase: Elenbecestat 50 mgn=12 Participants-3.261.555
Secondary/registry result

Extension Phase: Percent Change From Extension Phase Baseline in Total Ventricular Volume at Month 24

Time frame:Month 24

percent change from baseline, improvement

Posted result

GroupValue (mean), percent changeStandard deviation
Extension Phase: Elenbecestat 50 mgn=12 Participants15.188.544

Fluid / digital biomarkers

2 endpoints
Secondary/protocol endpoint

Core Phase: Mean Concentration of Elenbecestat in CSF

Time frame:Month 1 (Week 5) and Month 18 (Week 79)

concentration, descriptive

Secondary/registry result

Core Phase: Mean Concentration of Elenbecestat in CSF

Time frame:Month 1 (Week 5) and Month 18 (Week 79)

concentration, descriptive

Posted result

GroupValue (mean), nanogram per milliliter (ng/mL)Standard deviation
Core Phase: Elenbecestat 5 mg Then 50 mgMonth 1 (Week 5)n=7 Participants1.100.438
Month 18 (Week 79)n=2 Participants0.100
Core Phase: Elenbecestat 15 mg Then 50 mgMonth 1 (Week 5)n=6 Participants3.711.360
Month 18 (Week 79)n=2 Participants2.723.197
Core Phase: Elenbecestat 50 mgMonth 1 (Week 5)n=6 Participants9.933.568
Month 18 (Week 79)n=4 Participants14.463.411

Safety / tolerability / PK

14 endpoints
Primary/protocol endpoint

Core Phase: Number of Participants With Treatment Emergent Adverse Events (TEAEs)

Time frame:Up to 21 months

event count, event

Primary/registry result

Core Phase: Number of Participants With Treatment Emergent Adverse Events (TEAEs)

Time frame:Up to 21 months

event count, event

Posted result

GroupValue (count_of_participants), ParticipantsReported bounds
Core Phase: Placebon=17 Participants15-
Core Phase: Elenbecestat 5 mg Then 50 mgn=17 Participants15-
Core Phase: Elenbecestat 15 mg Then 50 mgn=19 Participants18-
Core Phase: Elenbecestat 50 mgn=17 Participants15-
Primary/protocol endpoint

Core Phase: Number of Participants With Serious Adverse Events (SAEs)

Time frame:Up to 21 months

event count, event

Primary/registry result

Core Phase: Number of Participants With Serious Adverse Events (SAEs)

Time frame:Up to 21 months

event count, event

Posted result

GroupValue (count_of_participants), ParticipantsReported bounds
Core Phase: Placebon=17 Participants2-
Core Phase: Elenbecestat 5 mg Then 50 mgn=17 Participants2-
Core Phase: Elenbecestat 15 mg Then 50 mgn=19 Participants5-
Core Phase: Elenbecestat 50 mgn=17 Participants1-
Primary/protocol endpoint

Core Phase: Number of Participants With Treatment Emergent Markedly Abnormal Laboratory Safety Test Values

Time frame:Up to 21 months

event count, event

Primary/registry result

Core Phase: Number of Participants With Treatment Emergent Markedly Abnormal Laboratory Safety Test Values

Time frame:Up to 21 months

event count, event

Posted result

GroupValue (count_of_participants), ParticipantsReported bounds
Core Phase: PlaceboMarkedly Abnormal Low: Lymphocytesn=17 Participants0-
Markedly Abnormal Low: Leukocytesn=17 Participants1-
Markedly Abnormal Low: Neutrophilsn=17 Participants1-
Markedly Abnormal Low: Hemoglobinn=17 Participants1-
Markedly Abnormal Low: Calciumn=17 Participants1-
Markedly Abnormal Low: Potassiumn=17 Participants0-
Markedly Abnormal High: Potassiumn=17 Participants2-
Markedly Abnormal High: Alkaline Phosphatasen=17 Participants0-
Markedly Abnormal High: Alanine Aminotransferasen=17 Participants1-
Markedly Abnormal High: Aspartate Aminotransferasen=17 Participants0-
Markedly Abnormal High: Gamma Glutamyl Transferasen=17 Participants1-
Markedly Abnormal Low: Glucosen=17 Participants0-
Markedly Abnormal High: Glucosen=17 Participants2-
Markedly Abnormal High: Cholesteroln=17 Participants1-
Markedly Abnormal High: Triglyceridesn=17 Participants1-
Markedly Abnormal High: Creatininen=17 Participants0-
Core Phase: Elenbecestat 5 mg Then 50 mgMarkedly Abnormal Low: Lymphocytesn=17 Participants2-
Markedly Abnormal Low: Leukocytesn=17 Participants0-
Markedly Abnormal Low: Neutrophilsn=17 Participants0-
Markedly Abnormal Low: Hemoglobinn=17 Participants0-
Markedly Abnormal Low: Calciumn=17 Participants0-
Markedly Abnormal Low: Potassiumn=17 Participants0-
Markedly Abnormal High: Potassiumn=17 Participants4-
Markedly Abnormal High: Alkaline Phosphatasen=17 Participants0-
Markedly Abnormal High: Alanine Aminotransferasen=17 Participants0-
Markedly Abnormal High: Aspartate Aminotransferasen=17 Participants0-
Markedly Abnormal High: Gamma Glutamyl Transferasen=17 Participants0-
Markedly Abnormal Low: Glucosen=17 Participants1-
Markedly Abnormal High: Glucosen=17 Participants4-
Markedly Abnormal High: Cholesteroln=17 Participants0-
Markedly Abnormal High: Triglyceridesn=17 Participants1-
Markedly Abnormal High: Creatininen=17 Participants2-
Core Phase: Elenbecestat 15 mg Then 50 mgMarkedly Abnormal Low: Lymphocytesn=19 Participants0-
Markedly Abnormal Low: Leukocytesn=19 Participants0-
Markedly Abnormal Low: Neutrophilsn=19 Participants0-
Markedly Abnormal Low: Hemoglobinn=19 Participants0-
Markedly Abnormal Low: Calciumn=19 Participants0-
Markedly Abnormal Low: Potassiumn=19 Participants1-
Markedly Abnormal High: Potassiumn=19 Participants2-
Markedly Abnormal High: Alkaline Phosphatasen=19 Participants0-
Markedly Abnormal High: Alanine Aminotransferasen=19 Participants0-
Markedly Abnormal High: Aspartate Aminotransferasen=19 Participants0-
Markedly Abnormal High: Gamma Glutamyl Transferasen=19 Participants0-
Markedly Abnormal Low: Glucosen=19 Participants0-
Markedly Abnormal High: Glucosen=19 Participants1-
Markedly Abnormal High: Cholesteroln=19 Participants0-
Markedly Abnormal High: Triglyceridesn=19 Participants3-
Markedly Abnormal High: Creatininen=19 Participants0-
Core Phase: Elenbecestat 50 mgMarkedly Abnormal Low: Lymphocytesn=17 Participants0-
Markedly Abnormal Low: Leukocytesn=17 Participants0-
Markedly Abnormal Low: Neutrophilsn=17 Participants1-
Markedly Abnormal Low: Hemoglobinn=17 Participants0-
Markedly Abnormal Low: Calciumn=17 Participants0-
Markedly Abnormal Low: Potassiumn=17 Participants0-
Markedly Abnormal High: Potassiumn=17 Participants0-
Markedly Abnormal High: Alkaline Phosphatasen=17 Participants1-
Markedly Abnormal High: Alanine Aminotransferasen=17 Participants1-
Markedly Abnormal High: Aspartate Aminotransferasen=17 Participants1-
Markedly Abnormal High: Gamma Glutamyl Transferasen=17 Participants1-
Markedly Abnormal Low: Glucosen=17 Participants0-
Markedly Abnormal High: Glucosen=17 Participants1-
Markedly Abnormal High: Cholesteroln=17 Participants0-
Markedly Abnormal High: Triglyceridesn=17 Participants2-
Markedly Abnormal High: Creatininen=17 Participants0-
Primary/protocol endpoint

Core Phase: Number of Participants With Markedly Abnormal Vital Sign Values

Time frame:Month 0(Baseline,Week 2,Week 3,Week 4);Month 1(Week 5,Week 7);Month 2(Week 9,Week 11);Month 3(Week 13);Month 4(Week 17);Month 5(Week 21);Month 6(Week 27);Month 9(Week 40);Month 12(Week 53);Month 15(Week 66);Month 18(Week 79) and Follow-up at Month 1 and 3

event count, event

Primary/registry result

Core Phase: Number of Participants With Markedly Abnormal Vital Sign Values

Time frame:Month 0(Baseline,Week 2,Week 3,Week 4);Month 1(Week 5,Week 7);Month 2(Week 9,Week 11);Month 3(Week 13);Month 4(Week 17);Month 5(Week 21);Month 6(Week 27);Month 9(Week 40);Month 12(Week 53);Month 15(Week 66);Month 18(Week 79) and Follow-up at Month 1 and 3

event count, event

Posted result

GroupValue (count_of_participants), ParticipantsReported bounds
Core Phase: PlaceboMarkedly Abnormal Low: Temperature (Month 0: Baseline)n=17 Participants0-
Markedly Abnormal Low: Temperature (Month 0: Week 2)n=16 Participants0-
Markedly Abnormal Low: Temperature (Month 0: Week 3)n=17 Participants2-
Markedly Abnormal Low: Temperature (Month 0: Week 4)n=17 Participants1-
Markedly Abnormal High: Temperature (Month 0: Week 4)n=17 Participants0-
Markedly Abnormal Low: Temperature (Month 1: Week 5)n=17 Participants1-
Markedly Abnormal Low: Temperature (Month 1: Week 7)n=17 Participants0-
Markedly Abnormal Low: Temperature (Month 2: Week 9)n=17 Participants0-
Markedly Abnormal Low: Temperature (Month 2: Week 11)n=16 Participants1-
Markedly Abnormal Low: Temperature (Month 3: Week 13)n=16 Participants1-
Markedly Abnormal Low: Temperature (Month 4: Week 17)n=15 Participants1-
Markedly Abnormal Low: Temperature (Month 5: Week 21)n=15 Participants1-
Markedly Abnormal Low: Temperature (Month 6: Week 27)n=15 Participants0-
Markedly Abnormal Low: Temperature (Month 9: Week 40)n=14 Participants1-
Markedly Abnormal Low: Temperature (Month 12: Week 53)n=13 Participants0-
Markedly Abnormal Low: Temperature (Month 15: Week 66)n=13 Participants0-
Markedly Abnormal Low: Temperature (Month 18: Week 79)n=12 Participants0-
Markedly Abnormal Low: Temperature (Follow-up: Month 1)n=13 Participants0-
Markedly Abnormal Low: Temperature (Follow-up: Month 3)n=12 Participants0-
Markedly Abnormal Low: Weight (Month 0: Baseline)n=17 Participants0-
Markedly Abnormal High: Weight (Month 0: Baseline)n=17 Participants1-
Markedly Abnormal High: Weight (Month 3: Week 13)n=16 Participants0-
Markedly Abnormal High: Weight (Month 6: Week 27)n=14 Participants0-
Markedly Abnormal High: Weight (Month 9: Week 40)n=13 Participants0-
Markedly Abnormal High: Weight (Follow-up: Month 3)n=10 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 0: Baseline)n=17 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 0: Week 2)n=16 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 0: Week 3)n=17 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 0: Week 4)n=17 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 1: Week 5)n=17 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 1: Week 7)n=17 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 3: Week 13)n=16 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 4: Week 17)n=15 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 6: Week 27)n=15 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 9: Week 40)n=14 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 12: Week 53)n=13 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 15: Week 66)n=13 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 18: Week 79)n=12 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Follow-up: Month 1)n=13 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Follow-up: Month 3)n=12 Participants0-
Markedly Abnormal High: Diastolic Blood Pressure (Month 0: Week 3)n=17 Participants1-
Markedly Abnormal Low: Diastolic Blood Pressure (Month 0: Week 4)n=17 Participants1-
Markedly Abnormal Low: Diastolic Blood Pressure (Month 1: Week 5)n=17 Participants0-
Markedly Abnormal Low: Diastolic Blood Pressure (Month 9: Week 40)n=14 Participants0-
Markedly Abnormal High: Diastolic Blood Pressure (Month 9: Week 40)n=14 Participants1-
Markedly Abnormal High: Diastolic Blood Pressure (Follow-up: Month 1)n=13 Participants1-
Markedly Abnormal High: Diastolic Blood Pressure (Follow-up: Month 3)n=12 Participants0-
Core Phase: Elenbecestat 5 mg Then 50 mgMarkedly Abnormal Low: Temperature (Month 0: Baseline)n=17 Participants2-
Markedly Abnormal Low: Temperature (Month 0: Week 2)n=17 Participants0-
Markedly Abnormal Low: Temperature (Month 0: Week 3)n=17 Participants1-
Markedly Abnormal Low: Temperature (Month 0: Week 4)n=17 Participants3-
Markedly Abnormal High: Temperature (Month 0: Week 4)n=17 Participants0-
Markedly Abnormal Low: Temperature (Month 1: Week 5)n=17 Participants0-
Markedly Abnormal Low: Temperature (Month 1: Week 7)n=17 Participants1-
Markedly Abnormal Low: Temperature (Month 2: Week 9)n=17 Participants1-
Markedly Abnormal Low: Temperature (Month 2: Week 11)n=16 Participants1-
Markedly Abnormal Low: Temperature (Month 3: Week 13)n=16 Participants2-
Markedly Abnormal Low: Temperature (Month 4: Week 17)n=16 Participants3-
Markedly Abnormal Low: Temperature (Month 5: Week 21)n=16 Participants3-
Markedly Abnormal Low: Temperature (Month 6: Week 27)n=15 Participants1-
Markedly Abnormal Low: Temperature (Month 9: Week 40)n=13 Participants2-
Markedly Abnormal Low: Temperature (Month 12: Week 53)n=12 Participants1-
Markedly Abnormal Low: Temperature (Month 15: Week 66)n=11 Participants3-
Markedly Abnormal Low: Temperature (Month 18: Week 79)n=11 Participants1-
Markedly Abnormal Low: Temperature (Follow-up: Month 1)n=12 Participants0-
Markedly Abnormal Low: Temperature (Follow-up: Month 3)n=11 Participants2-
Markedly Abnormal Low: Weight (Month 0: Baseline)n=17 Participants0-
Markedly Abnormal High: Weight (Month 0: Baseline)n=17 Participants3-
Markedly Abnormal High: Weight (Month 3: Week 13)n=16 Participants2-
Markedly Abnormal High: Weight (Month 6: Week 27)n=15 Participants1-
Markedly Abnormal High: Weight (Month 9: Week 40)n=13 Participants0-
Markedly Abnormal High: Weight (Follow-up: Month 3)n=12 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 0: Baseline)n=17 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 0: Week 2)n=17 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 0: Week 3)n=17 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 0: Week 4)n=17 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 1: Week 5)n=17 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 1: Week 7)n=17 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 3: Week 13)n=16 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 4: Week 17)n=16 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 6: Week 27)n=15 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 9: Week 40)n=13 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 12: Week 53)n=12 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 15: Week 66)n=11 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 18: Week 79)n=11 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Follow-up: Month 1)n=12 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Follow-up: Month 3)n=11 Participants0-
Markedly Abnormal High: Diastolic Blood Pressure (Month 0: Week 3)n=17 Participants0-
Markedly Abnormal Low: Diastolic Blood Pressure (Month 0: Week 4)n=17 Participants1-
Markedly Abnormal Low: Diastolic Blood Pressure (Month 1: Week 5)n=17 Participants0-
Markedly Abnormal Low: Diastolic Blood Pressure (Month 9: Week 40)n=13 Participants1-
Markedly Abnormal High: Diastolic Blood Pressure (Month 9: Week 40)n=13 Participants0-
Markedly Abnormal High: Diastolic Blood Pressure (Follow-up: Month 1)n=12 Participants0-
Markedly Abnormal High: Diastolic Blood Pressure (Follow-up: Month 3)n=11 Participants0-
Core Phase: Elenbecestat 15 mg Then 50 mgMarkedly Abnormal Low: Temperature (Month 0: Baseline)n=19 Participants0-
Markedly Abnormal Low: Temperature (Month 0: Week 2)n=19 Participants2-
Markedly Abnormal Low: Temperature (Month 0: Week 3)n=19 Participants4-
Markedly Abnormal Low: Temperature (Month 0: Week 4)n=19 Participants1-
Markedly Abnormal High: Temperature (Month 0: Week 4)n=19 Participants1-
Markedly Abnormal Low: Temperature (Month 1: Week 5)n=19 Participants2-
Markedly Abnormal Low: Temperature (Month 1: Week 7)n=18 Participants3-
Markedly Abnormal Low: Temperature (Month 2: Week 9)n=15 Participants1-
Markedly Abnormal Low: Temperature (Month 2: Week 11)n=15 Participants1-
Markedly Abnormal Low: Temperature (Month 3: Week 13)n=15 Participants3-
Markedly Abnormal Low: Temperature (Month 4: Week 17)n=13 Participants1-
Markedly Abnormal Low: Temperature (Month 5: Week 21)n=13 Participants0-
Markedly Abnormal Low: Temperature (Month 6: Week 27)n=12 Participants0-
Markedly Abnormal Low: Temperature (Month 9: Week 40)n=11 Participants0-
Markedly Abnormal Low: Temperature (Month 12: Week 53)n=11 Participants1-
Markedly Abnormal Low: Temperature (Month 15: Week 66)n=10 Participants2-
Markedly Abnormal Low: Temperature (Month 18: Week 79)n=10 Participants0-
Markedly Abnormal Low: Temperature (Follow-up: Month 1)n=15 Participants2-
Markedly Abnormal Low: Temperature (Follow-up: Month 3)n=12 Participants4-
Markedly Abnormal Low: Weight (Month 0: Baseline)n=19 Participants1-
Markedly Abnormal High: Weight (Month 0: Baseline)n=19 Participants2-
Markedly Abnormal High: Weight (Month 3: Week 13)n=15 Participants2-
Markedly Abnormal High: Weight (Month 6: Week 27)n=12 Participants1-
Markedly Abnormal High: Weight (Month 9: Week 40)n=11 Participants0-
Markedly Abnormal High: Weight (Follow-up: Month 3)n=14 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 0: Baseline)n=19 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 0: Week 2)n=19 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 0: Week 3)n=19 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 0: Week 4)n=19 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 1: Week 5)n=19 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 1: Week 7)n=18 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 3: Week 13)n=15 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 4: Week 17)n=13 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 6: Week 27)n=12 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 9: Week 40)n=11 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 12: Week 53)n=11 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 15: Week 66)n=10 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 18: Week 79)n=10 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Follow-up: Month 1)n=15 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Follow-up: Month 3)n=12 Participants0-
Markedly Abnormal High: Diastolic Blood Pressure (Month 0: Week 3)n=19 Participants0-
Markedly Abnormal Low: Diastolic Blood Pressure (Month 0: Week 4)n=19 Participants0-
Markedly Abnormal Low: Diastolic Blood Pressure (Month 1: Week 5)n=19 Participants0-
Markedly Abnormal Low: Diastolic Blood Pressure (Month 9: Week 40)n=11 Participants0-
Markedly Abnormal High: Diastolic Blood Pressure (Month 9: Week 40)n=11 Participants0-
Markedly Abnormal High: Diastolic Blood Pressure (Follow-up: Month 1)n=15 Participants0-
Markedly Abnormal High: Diastolic Blood Pressure (Follow-up: Month 3)n=12 Participants0-
Core Phase: Elenbecestat 50 mgMarkedly Abnormal Low: Temperature (Month 0: Baseline)n=17 Participants2-
Markedly Abnormal Low: Temperature (Month 0: Week 2)n=17 Participants2-
Markedly Abnormal Low: Temperature (Month 0: Week 3)n=16 Participants0-
Markedly Abnormal Low: Temperature (Month 0: Week 4)n=17 Participants1-
Markedly Abnormal High: Temperature (Month 0: Week 4)n=17 Participants0-
Markedly Abnormal Low: Temperature (Month 1: Week 5)n=17 Participants0-
Markedly Abnormal Low: Temperature (Month 1: Week 7)n=16 Participants0-
Markedly Abnormal Low: Temperature (Month 2: Week 9)n=16 Participants2-
Markedly Abnormal Low: Temperature (Month 2: Week 11)n=16 Participants2-
Markedly Abnormal Low: Temperature (Month 3: Week 13)n=16 Participants1-
Markedly Abnormal Low: Temperature (Month 4: Week 17)n=16 Participants1-
Markedly Abnormal Low: Temperature (Month 5: Week 21)n=16 Participants0-
Markedly Abnormal Low: Temperature (Month 6: Week 27)n=16 Participants1-
Markedly Abnormal Low: Temperature (Month 9: Week 40)n=15 Participants1-
Markedly Abnormal Low: Temperature (Month 12: Week 53)n=12 Participants0-
Markedly Abnormal Low: Temperature (Month 15: Week 66)n=12 Participants2-
Markedly Abnormal Low: Temperature (Month 18: Week 79)n=12 Participants1-
Markedly Abnormal Low: Temperature (Follow-up: Month 1)n=14 Participants0-
Markedly Abnormal Low: Temperature (Follow-up: Month 3)n=14 Participants0-
Markedly Abnormal Low: Weight (Month 0: Baseline)n=17 Participants0-
Markedly Abnormal High: Weight (Month 0: Baseline)n=17 Participants1-
Markedly Abnormal High: Weight (Month 3: Week 13)n=16 Participants2-
Markedly Abnormal High: Weight (Month 6: Week 27)n=15 Participants1-
Markedly Abnormal High: Weight (Month 9: Week 40)n=14 Participants1-
Markedly Abnormal High: Weight (Follow-up: Month 3)n=14 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 0: Baseline)n=17 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 0: Week 2)n=17 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 0: Week 3)n=16 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 0: Week 4)n=17 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 1: Week 5)n=17 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 1: Week 7)n=16 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 3: Week 13)n=16 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 4: Week 17)n=16 Participants0-
Markedly Abnormal High: Systolic Blood Pressure (Month 6: Week 27)n=16 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 9: Week 40)n=15 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 12: Week 53)n=12 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 15: Week 66)n=12 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Month 18: Week 79)n=12 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Follow-up: Month 1)n=14 Participants1-
Markedly Abnormal High: Systolic Blood Pressure (Follow-up: Month 3)n=14 Participants1-
Markedly Abnormal High: Diastolic Blood Pressure (Month 0: Week 3)n=16 Participants0-
Markedly Abnormal Low: Diastolic Blood Pressure (Month 0: Week 4)n=17 Participants0-
Markedly Abnormal Low: Diastolic Blood Pressure (Month 1: Week 5)n=17 Participants1-
Markedly Abnormal Low: Diastolic Blood Pressure (Month 9: Week 40)n=15 Participants0-
Markedly Abnormal High: Diastolic Blood Pressure (Month 9: Week 40)n=15 Participants0-
Markedly Abnormal High: Diastolic Blood Pressure (Follow-up: Month 1)n=14 Participants0-
Markedly Abnormal High: Diastolic Blood Pressure (Follow-up: Month 3)n=14 Participants1-
Primary/protocol endpoint

Core Phase: Number of Participants With Markedly Abnormal Electrocardiogram (ECG) Findings

Time frame:Up to 21 months

event count, event

Primary/registry result

Core Phase: Number of Participants With Markedly Abnormal Electrocardiogram (ECG) Findings

Time frame:Up to 21 months

event count, event

Posted result

GroupValue (count_of_participants), ParticipantsReported bounds
Core Phase: PlaceboAt least one post-baseline increase of >30 millisecond (msec) in QTcF intervaln=17 Participants2-
At least one post-baseline value of >450 msec in QTcF intervaln=17 Participants1-
At least one post-baseline value of >480 msec in QTcF intervaln=17 Participants0-
Core Phase: Elenbecestat 5 mg Then 50 mgAt least one post-baseline increase of >30 millisecond (msec) in QTcF intervaln=17 Participants4-
At least one post-baseline value of >450 msec in QTcF intervaln=17 Participants6-
At least one post-baseline value of >480 msec in QTcF intervaln=17 Participants0-
Core Phase: Elenbecestat 15 mg Then 50 mgAt least one post-baseline increase of >30 millisecond (msec) in QTcF intervaln=19 Participants4-
At least one post-baseline value of >450 msec in QTcF intervaln=19 Participants4-
At least one post-baseline value of >480 msec in QTcF intervaln=19 Participants1-
Core Phase: Elenbecestat 50 mgAt least one post-baseline increase of >30 millisecond (msec) in QTcF intervaln=17 Participants2-
At least one post-baseline value of >450 msec in QTcF intervaln=17 Participants3-
At least one post-baseline value of >480 msec in QTcF intervaln=17 Participants0-
Primary/protocol endpoint

Extension Phase: Number of Participants With Markedly Abnormal Vital Sign Values

Time frame:Up to 34 months

event count, event

Primary/registry result

Extension Phase: Number of Participants With Markedly Abnormal Vital Sign Values

Time frame:Up to 34 months

event count, event

Posted result

GroupValue (count_of_participants), ParticipantsReported bounds
Extension Phase: Elenbecestat 50 mgMarkedly Abnormal High: Diastolic Blood Pressuren=41 Participants1-
Markedly Abnormal Low: Diastolic Blood Pressuren=41 Participants1-
Markedly Abnormal High: Systolic Blood Pressuren=41 Participants5-
Markedly Abnormal High: Temperaturen=41 Participants1-
Markedly Abnormal Low: Temperaturen=41 Participants10-
Markedly Abnormal High: Weightn=41 Participants1-
Primary/protocol endpoint

Extension Phase: Number of Participants With Treatment Emergent Markedly Abnormal Laboratory Safety Test Values

Time frame:Up to 34 months

event count, event

Primary/registry result

Extension Phase: Number of Participants With Treatment Emergent Markedly Abnormal Laboratory Safety Test Values

Time frame:Up to 34 months

event count, event

Posted result

GroupValue (count_of_participants), ParticipantsReported bounds
Extension Phase: Elenbecestat 50 mgMarkedly Abnormal Low: Lymphocytesn=41 Participants4-
Markedly Abnormal Low: Neutrophilsn=41 Participants2-
Markedly Abnormal Low: Hemoglobinn=41 Participants1-
Markedly Abnormal High: Potassiumn=41 Participants4-
Markedly Abnormal High: Alanine Aminotransferasen=41 Participants1-
Markedly Abnormal High: Gamma Glutamyl Transferasen=41 Participants2-
Markedly Abnormal High: Glucosen=41 Participants3-
Markedly Abnormal High: Triglyceridesn=41 Participants2-
Markedly Abnormal High: Creatininen=41 Participants4-
Markedly Abnormal High: Uraten=41 Participants1-
Markedly Abnormal Low: Plateletsn=41 Participants1-

Other (unclassified)

4 endpoints
Primary/protocol endpoint/low confidence

Extension Phase: Number of Participants With Markedly Abnormal ECG Findings

Time frame:Up to 34 months

event count, event

Primary/registry result/low confidence

Extension Phase: Number of Participants With Markedly Abnormal ECG Findings

Time frame:Up to 34 months

event count, event

Posted result

GroupValue (count_of_participants), ParticipantsReported bounds
Extension Phase: Elenbecestat 50 mgAt least one post-baseline increase of >30 msec in QTcF intervaln=41 Participants2-
At least one post-baseline value of >450 msec in QTcF intervaln=41 Participants4-
Secondary/protocol endpoint/low confidence

Core Phase: Mean Concentration of Elenbecestat in Plasma

Time frame:Month 0 (Week 3), Month 3 (Week 13), Month 6 (Week 27), Month 12 (Week 53): Pre-dose, 1 to 6 hours Post-dose; Month 1 (Week 5), Month 18 (Week 79): Pre-dose, 4 to 8 hours Post-dose

concentration, descriptive

Secondary/registry result/low confidence

Core Phase: Mean Concentration of Elenbecestat in Plasma

Time frame:Month 0 (Week 3), Month 3 (Week 13), Month 6 (Week 27), Month 12 (Week 53): Pre-dose, 1 to 6 hours Post-dose; Month 1 (Week 5), Month 18 (Week 79): Pre-dose, 4 to 8 hours Post-dose

concentration, descriptive

Posted result

GroupValue (mean), ng/mLStandard deviation
Core Phase: Elenbecestat 5 mg Then 50 mgMonth 0 (Week 3): Pre-dosen=17 Participants1.431.105
Month 0 (Week 3): 1 to 6 hours Post-dosen=17 Participants4.283.185
Month 1 (Week 5): Pre-dosen=15 Participants1.470.870
Month 1 (Week 5): 4 to 8 hours Post-dosen=16 Participants4.802.529
Month 3 (Week 13): Pre-dosen=16 Participants1.911.703
Month 3 (Week 13): 1 to 6 hours Post-dosen=16 Participants6.062.456
Month 6 (Week 27): Pre-dosen=15 Participants2.342.196
Month 6 (Week 27): 1 to 6 hours Post-dosen=15 Participants11.1115.322
Month 12 (Week 53): Pre-dosen=11 Participants13.8418.482
Month 12 (Week 53): 1 to 6 hours Post-dosen=12 Participants49.7840.656
Month 18 (Week 79): Pre-dosen=10 Participants7.885.896
Month 18 (Week 79): 4 to 8 hours Post-dosen=9 Participants47.1628.651
Core Phase: Elenbecestat 15 mg Then 50 mgMonth 0 (Week 3): Pre-dosen=18 Participants5.813.785
Month 0 (Week 3): 1 to 6 hours Post-dosen=18 Participants18.4911.272
Month 1 (Week 5): Pre-dosen=19 Participants6.744.246
Month 1 (Week 5): 4 to 8 hours Post-dosen=19 Participants19.597.940
Month 3 (Week 13): Pre-dosen=15 Participants6.908.126
Month 3 (Week 13): 1 to 6 hours Post-dosen=14 Participants20.358.893
Month 6 (Week 27): Pre-dosen=11 Participants9.109.018
Month 6 (Week 27): 1 to 6 hours Post-dosen=12 Participants25.4922.247
Month 12 (Week 53): Pre-dosen=10 Participants21.0330.897
Month 12 (Week 53): 1 to 6 hours Post-dosen=10 Participants58.3865.148
Month 18 (Week 79): Pre-dosen=8 Participants7.136.990
Month 18 (Week 79): 4 to 8 hours Post-dosen=8 Participants51.9944.656
Core Phase: Elenbecestat 50 mgMonth 0 (Week 3): Pre-dosen=13 Participants15.7711.281
Month 0 (Week 3): 1 to 6 hours Post-dosen=13 Participants70.4137.681
Month 1 (Week 5): Pre-dosen=15 Participants15.4610.463
Month 1 (Week 5): 4 to 8 hours Post-dosen=16 Participants66.8321.370
Month 3 (Week 13): Pre-dosen=16 Participants12.317.007
Month 3 (Week 13): 1 to 6 hours Post-dosen=16 Participants75.1527.852
Month 6 (Week 27): Pre-dosen=15 Participants12.038.962
Month 6 (Week 27): 1 to 6 hours Post-dosen=15 Participants71.2839.114
Month 12 (Week 53): Pre-dosen=11 Participants16.7422.804
Month 12 (Week 53): 1 to 6 hours Post-dosen=11 Participants82.5556.084
Month 18 (Week 79): Pre-dosen=12 Participants19.2113.602
Month 18 (Week 79): 4 to 8 hours Post-dosen=11 Participants70.4322.500

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.