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The study will investigate the effects of oral ALZ-801, in subjects with Early AD who have the APOE4/4 or APOE3/4 genotype, on the biomarkers of core AD pathology. The objectives of this study include determining the efficacy and safety/tolerability of ALZ-801. In addition, the study will evaluate the extended PK profile over 8 hours in 16 subjects after 65 weeks of treatment.
The LTE year 1 & 2 study will investigate the effects of oral ALZ-801, in subjects with Early AD who have the APOE4/4 or APOE3/4 genotype, on the biomarkers of core AD pathology. The objectives of LTE study are to continue longitudinal assessment of the efficacy and safety/tolerability of ALZ-801 over a total period of 4 years (2-year Core study plus 2 years of LTE).
Core study: ALZ-801 265 mg twice daily (BID) in the Core Study, Weeks 0-104
LTE year 1: ALZ-801 265 mg BID in the Core Study and the Long-Term Extension (LTE, Weeks 104-208)
LTE year 2: ALZ-801 265mg BID in the Core Study and LTE Year 1 (Weeks 104-156), and LTE Year 2 (Weeks 156-208)
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Active treatment | Experimental | ALZ-801 265 mg tablets once daily for two weeks and twice daily thereafter |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ALZ-801 | Drug | ALZ-801 265 mg twice daily (BID) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Plasma Biomarker of Core AD Pathology | Percent change from baseline in p-tau181 | Week 104 |
| Incidence, Nature, and Severity of Treatment Emergent Adverse events (TEAE) | Safety and tolerability as measured by incidence, nature and severity of treatment emergent adverse events (TEAE), serious TEAE, and TEAE leading to withdrawal. | Week 108 |
| Volumetric Magnetic Resonance Imaging (vMRI) Biomarker - Hippocampal Volume | Change from baseline in hippocampal volume measured in mm3 | Weeks 104 |
| Measure | Description | Time Frame |
|---|---|---|
| Plasma Biomarkers of AD and Neurodegeneration | Percent changes from baseline in: Aβ-40, Aβ-42,p-tau217 and plasma glial fibrillary acidic protein (GFAP),NfL | Weeks 104 |
| vMRI Biomarker - Ventricular volume and Cortical Thickness |
| Measure | Description | Time Frame |
|---|---|---|
| Cognitive assessment - Rey Auditory Verbal Learning Test (RAVLT) | Change from baseline in RAVLT score | Weeks 104, week 156 and week 208 |
| Cognitive Assessment - Digit Symbol Substitution Test (DSST) | Change from baseline in DSST score |
Inclusion Criteria
Exclusion Criteria
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| Name | Affiliation | Role |
|---|---|---|
| John Hey, PhD | Alzheon Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| St. Anne's University Hospital | Brno | Czechia | ||||
| Motol University Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39907966 | Derived | Hey JA, Yu JY, Abushakra S, Schaefer JF, Power A, Kesslak P, Paul J, Tolar M. Clinical Pharmacokinetics of Oral ALZ-801/Valiltramiprosate in a 2-Year Phase 2 Trial of APOE4 Carriers with Early Alzheimer's Disease. Clin Pharmacokinet. 2025 Mar;64(3):407-424. doi: 10.1007/s40262-025-01482-8. Epub 2025 Feb 5. | |
| 38902572 | Derived |
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| ID | Term |
|---|---|
| D000544 | Alzheimer Disease |
| ID | Term |
|---|---|
| D003704 | Dementia |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| C000631566 | ALZ-801 |
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Change from baseline in cortical thickness measured in mm3
| Weeks 104, 156 and 208 |
| Additional CSF Biomarkers of AD Pathology and Neurodegeneration | Percent changes from baseline for: p-tau217,Aβ-40, Aβ-42, NfL, t-tau, sTREM2, YKL-40 and neurogranin | Weeks 104 |
| Incidence, Nature, and Severity of Treatment Emergent Adverse events (TEAE) | Safety and tolerability as measured by incidence, nature and severity of treatment emergent adverse events (TEAE), serious TEAE, and TEAE leading to withdrawal. | Week 160 and week 212 |
| Volumetric Magnetic Resonance Imaging (vMRI) Biomarker - Hippocampal Volume | Change from baseline in hippocampal volume measured in mm3 | Week 156 and week 208 |
| Weeks 104, Week 156 and Week 208 |
| Functional Assessment - Amsterdam Instrumental Activities of Daily Living (A-IADL) | Change from baseline in A-IADL score | Weeks 104, Week 156 and Week 208 |
| Cognitive Assessment - Mini Mental State Examination (MMSE) | Change from baseline in MMSE score | Weeks 104, Week 156 and Week 208 |
| Global Assessment - Clinical Dementia Rating - Sum of Boxes (CDR-SB) | Change from baseline in CDR-SB score | Weeks 104, Week 156 and Week 208 |
| Prague |
| Czechia |
| Vestra Clinics | Rychnov nad Kněžnou | Czechia |
| Brain Research Center | 's-Hertogenbosch | Netherlands |
| Brain Research Center | Amsterdam | Netherlands |
| Brain Research Center | Zwolle | Netherlands |
| Hey JA, Yu JY, Abushakra S, Schaefer JF, Power A, Kesslak P, Tolar M. Analysis of Cerebrospinal Fluid, Plasma beta-Amyloid Biomarkers, and Cognition from a 2-Year Phase 2 Trial Evaluating Oral ALZ-801/Valiltramiprosate in APOE4 Carriers with Early Alzheimer's Disease Using Quantitative Systems Pharmacology Model. Drugs. 2024 Jul;84(7):825-839. doi: 10.1007/s40265-024-02068-7. Epub 2024 Jun 20. |
| 38902571 | Derived | Hey JA, Abushakra S, Blennow K, Reiman EM, Hort J, Prins ND, Sheardova K, Kesslak P, Shen L, Zhu X, Albayrak A, Paul J, Schaefer JF, Power A, Tolar M. Effects of Oral ALZ-801/Valiltramiprosate on Plasma Biomarkers, Brain Hippocampal Volume, and Cognition: Results of 2-Year Single-Arm, Open-Label, Phase 2 Trial in APOE4 Carriers with Early Alzheimer's Disease. Drugs. 2024 Jul;84(7):811-823. doi: 10.1007/s40265-024-02067-8. Epub 2024 Jun 20. |
| D024801 |
| Tauopathies |
| D019636 | Neurodegenerative Diseases |
| D019965 | Neurocognitive Disorders |
| D001523 | Mental Disorders |