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PEGASUS
CompletedPhase 2Results postedStudy 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
Lead sponsor
Asset
AMX0035
Listed sites
11
Recruiting sites
-
Enrollment
95
actual
Study population
Alzheimer’s disease
Key I/E criteria
•Alzheimer's disease•MoCA 8•MRI 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
Timeline
Milestones
Assets
Drug assets
Study populations
Who this study enrolls
Eligibility
Who can enroll
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 endpointsEffect of Treatment on Cognition
Time frame:24 weeks
ADAS-Cog
descriptive
Effect of Treatment on Cognitive Impairment
Time frame:24 weeks
Montreal Cognitive Assessment (MoCA)
descriptive
Effect of Treatment on Cognition
Time frame:24 weeks
ADAS-Cog
descriptive
Posted result
| Group | Value (least_squares_mean), score on a scale | Standard error |
|---|---|---|
| Active (AMX0035)n=51 Participants | 2.26 | 1.402 |
| Placebon=44 Participants | 1.52 | 1.496 |
Effect of Treatment on Cognitive Impairment
Time frame:24 weeks
Montreal Cognitive Assessment (MoCA)
descriptive
Posted result
| Group | Value (least_squares_mean), score on a scale | Standard error |
|---|---|---|
| Active (AMX0035)n=51 Participants | -2.18 | 0.483 |
| Placebon=44 Participants | -0.70 | 0.512 |
Function / daily living
4 endpointsEffect of Treatment on a Global Composite Statistical Test of Cognition, Function, and Neuroanatomy (GST)
Time frame:24 weeks
change from baseline, improvement
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
| Group | Value (least_squares_mean), score on a scale | Standard error |
|---|---|---|
| Active (AMX0035)n=51 Participants | 0.24 | 0.049 |
| Placebon=44 Participants | 0.17 | 0.052 |
Effect of Treatment on Functioning
Time frame:24 weeks
descriptive
Effect of Treatment on Functioning
Time frame:24 weeks
descriptive
Posted result
| Group | Value (least_squares_mean), score on a scale | Standard error |
|---|---|---|
| Active (AMX0035)n=51 Participants | 2.61 | 0.790 |
| Placebon=44 Participants | 1.59 | 0.845 |
Behavior / neuropsychiatric
2 endpointsEffect of Treatment on Neuropsychiatric Symptoms
Time frame:24 weeks
Neuropsychiatric Inventory (NPI)
descriptive
Effect of Treatment on Neuropsychiatric Symptoms
Time frame:24 weeks
Neuropsychiatric Inventory (NPI)
descriptive
Posted result
| Group | Value (least_squares_mean), score on a scale | Standard error |
|---|---|---|
| Active (AMX0035)n=51 Participants | 0.15 | 0.490 |
| Placebon=44 Participants | -0.46 | 0.526 |
Neuroimaging
2 endpointsRegional Brain Volume
Time frame:24 weeks
descriptive
Regional Brain Volume
Time frame:24 weeks
descriptive
Posted result
| Group | Value (least_squares_mean), mm^3 | Standard error |
|---|---|---|
| Active (AMX0035)n=51 Participants | -75.18 | 25.429 |
| Placebon=44 Participants | -70.78 | 23.308 |
Safety / tolerability / PK
2 endpointsNumber of Participants With Treatment-Emergent Adverse Event (TEAEs)
Time frame:From first dose to 24 weeks
event count, event
Number of Participants With Treatment-Emergent Adverse Event (TEAEs)
Time frame:From first dose to 24 weeks
event count, event
Posted result
| Group | Value (count_of_participants), Participants | Reported bounds |
|---|---|---|
| Active (AMX0035)n=51 Participants | 34 | - |
| Placebon=44 Participants | 26 | - |
Other (unclassified)
2 endpointsEffect of Treatment on Dementia Severity
Time frame:24 weeks
descriptive
Effect of Treatment on Dementia Severity
Time frame:24 weeks
descriptive
Posted result
| Group | Value (least_squares_mean), score on a scale | Standard error |
|---|---|---|
| Active (AMX0035)n=51 Participants | 2.34 | 0.595 |
| Placebon=44 Participants | 1.81 | 0.636 |
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.
Registry references + supporting bibliography
- PMID37941072via DERIVED
- PMID39131742via RESULT
Provenance
Sources
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.