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CompletedPhase 2Results posted

Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease

Phase II Study to Assess the Safety, Tolerability, and Target Engagement of AMX0035, a Fixed Combination of Sodium Phenylbutyrate and Tauroursodeoxycholic Acid for the Treatment of Alzheimer's Disease

Asset

AMX0035

Listed sites

11

Recruiting sites

-

Enrollment

95

actual

Study population

Alzheimer’s disease

Key I/E criteria

Alzheimer's diseaseMoCA 8MRI contraindications excluded

Primary endpoints

Treatment-Emergent Adverse Event (TEAEs)Effect of Treatment on a Global Composite Statistical Test of Cognition

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

Org study IDAMX-8000
NCT IDNCT03533257

Timeline

Milestones

Study first posted2018-05-23actual
Study start2018-09-14actual
Primary completion2020-10-22actual
Study completion2020-11-06actual
Last update posted2025-03-07actual
Results first posted2025-03-07actual

Assets

Drug assets

Study populations

Who this study enrolls

Alzheimer’s disease

Eligibility

Who can enroll

Minimum age55 Years
Maximum age89 Years
SexAll
Healthy volunteersNot accepted

Inclusion criteria

1. Ages 55-89, inclusive, male or female

2. Diagnosis of "Probable Alzheimer's Disease" or Mild Cognitive Impairment (amnestic or amnestic plus other) with biomarkers that suggest intermediate or high likelihood that the syndrome is due to AD, according to 2011 NIA-AA Workgroup criteria

3. MoCA score >/=8

4. Able to read and write in English sufficiently to complete all study procedures

5. Geriatric Depression Scale <7

6. Willing and able to complete all assessments and study procedures

7. Not pregnant, lactating or of child-bearing potential (women must be >2 years post-menopausal or surgically sterile)

8. Study partner with at least two days per week with contact with patient willing to accompany patient to visits and complete partner study forms

9. No known hypersensitivity to TURSO or Phenylbutyrate

10. If on cholinesterase inhibitor and/or memantine, treatment must have started for no less than 3 months (84 days) prior to baseline and the dosing regimen must have remained stable for 6 weeks (42 days) prior to baseline. The Investigator anticipated that the dosing regimen at baseline would remain unchanged throughout participation in the study

Exclusion criteria

1. Any CNS disease other than suspected AD, such as clinical stroke, brain tumor, normal pressure hydrocephalus, multiple sclerosis, significant head trauma with persistent neurological cognitive deficits or complaints, Parkinson's disease, frontotemporal dementia, or other neurodegenerative diseases

2. Abnormal liver function defined as AST and/or ALT > 3 times the upper limit of normal

3. Renal insufficiency as defined by a serum creatinine > 1.5 times the upper limit of normal

4. Recent (less than 1 year) cholecystectomy or the presence of post-cholecystectomy syndrome or biliary obstruction

5. Clinically significant unstable medical condition (other than AD) that in the Site Investigator opinion would pose a risk to the participant if they were to participate in the study

6. Any contraindication to undergo MRI studies such as:

1. History of a cardiac pacemaker or pacemaker wires

2. Metallic particles in the body

3. Vascular clips in the head

4. Prosthetic heart valves

5. Severe claustrophobia impeding ability to participate in an imaging study, or

MRI findings that show one or more of the following:

1. More than 4 incidental microhemorrhages

2. Incidental lacunar infarct with attributable signs or symptoms and with history of stroke

3. Incidental meningiomas with attributable signs or symptoms

4. Newly recognized meningioma

7. Major active or chronic psychiatric illness (e.g. depression, bipolar disorder, obsessive compulsive disorder, schizophrenia) that is not stable or well controlled within the previous year prior to baseline

8. Any significant neurodevelopmental disability

9. Current suicidal ideation or history of suicide attempt within 5 years of baseline or significant change from the screening and baseline C-SSRS at the discretion of the Site Investigator

10. History of alcohol or other substance abuse or dependence within the past 2 years

11. Any significant systemic illness or medical condition that could affect safety or compliance with study at the discretion of the Site Investigator

12. Laboratory abnormalities in B12, TSH, or other common laboratory parameters that might contribute to cognitive dysfunction

13. Current use of medications with psychoactive properties that may deleteriously affect cognition (e.g., anticholinergics, centrally-acting antihistamines, antipsychotics, sedative hypnotics, anxiolytics)

14. Use of any investigational therapy being used or evaluated for the treatment of AD is prohibited beginning 3 months (90 days) prior to the Baseline Visit and throughout the study.

15. Use of other investigational agents 1 month (28 days) prior to the Baseline Visit and for the duration of the trial.

Endpoints (16)

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

Global cognition
4
Function / daily living
4
Behavior / neuropsychiatric
2
Neuroimaging
2
Safety / tolerability / PK
2
Other (unclassified)
2

Global cognition

4 endpoints
Secondary/protocol endpoint

Effect of Treatment on Cognition

Time frame:24 weeks

ADAS-Cog

descriptive

Secondary/protocol endpoint

Effect of Treatment on Cognitive Impairment

Time frame:24 weeks

Montreal Cognitive Assessment (MoCA)

descriptive

Secondary/registry result

Effect of Treatment on Cognition

Time frame:24 weeks

ADAS-Cog

descriptive

Posted result

GroupValue (least_squares_mean), score on a scaleStandard error
Active (AMX0035)n=51 Participants2.261.402
Placebon=44 Participants1.521.496
p0.6698Mixed Models Analysis
Secondary/registry result

Effect of Treatment on Cognitive Impairment

Time frame:24 weeks

Montreal Cognitive Assessment (MoCA)

descriptive

Posted result

GroupValue (least_squares_mean), score on a scaleStandard error
Active (AMX0035)n=51 Participants-2.180.483
Placebon=44 Participants-0.700.512
p0.0361Mixed Models Analysis

Function / daily living

4 endpoints
Primary/protocol endpoint

Effect of Treatment on a Global Composite Statistical Test of Cognition, Function, and Neuroanatomy (GST)

Time frame:24 weeks

change from baseline, improvement

Primary/registry result

Effect of Treatment on a Global Composite Statistical Test of Cognition, Function, and Neuroanatomy (GST)

Time frame:24 weeks

change from baseline, improvement

Posted result

GroupValue (least_squares_mean), score on a scaleStandard error
Active (AMX0035)n=51 Participants0.240.049
Placebon=44 Participants0.170.052
p0.3654Mixed Models Analysis
Secondary/protocol endpoint

Effect of Treatment on Functioning

Time frame:24 weeks

descriptive

Secondary/registry result

Effect of Treatment on Functioning

Time frame:24 weeks

descriptive

Posted result

GroupValue (least_squares_mean), score on a scaleStandard error
Active (AMX0035)n=51 Participants2.610.790
Placebon=44 Participants1.590.845
p0.3086Mixed Models Analysis

Behavior / neuropsychiatric

2 endpoints
Secondary/protocol endpoint

Effect of Treatment on Neuropsychiatric Symptoms

Time frame:24 weeks

Neuropsychiatric Inventory (NPI)

descriptive

Secondary/registry result

Effect of Treatment on Neuropsychiatric Symptoms

Time frame:24 weeks

Neuropsychiatric Inventory (NPI)

descriptive

Posted result

GroupValue (least_squares_mean), score on a scaleStandard error
Active (AMX0035)n=51 Participants0.150.490
Placebon=44 Participants-0.460.526
p0.3585Mixed Models Analysis

Neuroimaging

2 endpoints
Secondary/protocol endpoint

Regional Brain Volume

Time frame:24 weeks

descriptive

Secondary/registry result

Regional Brain Volume

Time frame:24 weeks

descriptive

Posted result

GroupValue (least_squares_mean), mm^3Standard error
Active (AMX0035)n=51 Participants-75.1825.429
Placebon=44 Participants-70.7823.308
p0.8996Mixed Models Analysis

Safety / tolerability / PK

2 endpoints
Primary/protocol endpoint

Number of Participants With Treatment-Emergent Adverse Event (TEAEs)

Time frame:From first dose to 24 weeks

event count, event

Primary/registry result

Number of Participants With Treatment-Emergent Adverse Event (TEAEs)

Time frame:From first dose to 24 weeks

event count, event

Posted result

GroupValue (count_of_participants), ParticipantsReported bounds
Active (AMX0035)n=51 Participants34-
Placebon=44 Participants26-

Other (unclassified)

2 endpoints
Secondary/protocol endpoint/low confidence

Effect of Treatment on Dementia Severity

Time frame:24 weeks

descriptive

Secondary/registry result/low confidence

Effect of Treatment on Dementia Severity

Time frame:24 weeks

descriptive

Posted result

GroupValue (least_squares_mean), score on a scaleStandard error
Active (AMX0035)n=51 Participants2.340.595
Placebon=44 Participants1.810.636
p0.5552Mixed Models Analysis

Publications (2)

Bibliography

Records linked to this trial through ClinicalTrials.gov references, PubMed NCT search, and curated study seeds. 'Canonical' marks design/result papers; others are registry references or candidates.

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.