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Biomarker Effects of ALZ-801 in APOE4 Carriers With Early Alzheimer's Disease

NCT ID: NCT04693520

Last Updated: 2025-12-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

84 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-09-30

Study Completion Date

2025-07-16

Brief Summary

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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.

Detailed Description

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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)

Conditions

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Early Alzheimer's Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Active treatment

ALZ-801 265 mg tablets once daily for two weeks and twice daily thereafter

Group Type EXPERIMENTAL

ALZ-801

Intervention Type DRUG

ALZ-801 265 mg twice daily (BID)

Interventions

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ALZ-801

ALZ-801 265 mg twice daily (BID)

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Age between 50 and 80 years, inclusive.
2. Early Alzheimer's Disease (AD): a diagnosis of Probable AD Dementia or Mild Cognitive Impairment (MCI) due to AD in accordance with the National Institute on Aging-Alzheimer's Association (NIA-AA) Working Group Criteria \[Albert et al, 2011; McKhann et al, 2011\].
3. One of the following apolipoprotein E (APOE) genotypes - either APOE4/4 (homozygous) or APOE3/4 (heterozygous).
4. MMSE score 22 to 30 inclusive; Clinical Dementia Rating (CDR)-Global Score of 0.5 or 1.0, and CDR Memory Box Score of ≥ 0.5.
5. Documented confirmation of AD diagnosis by either positive amyloid positron emission tomography (PET) or positive CSF AD signature. Subjects without documented positive AD biomarker status must have a positive CSF biomarker result from a sample provided at screening.
6. Stable doses of acetylcholinesterase for the duration of the study are allowed.

Exclusion Criteria

1. Brain MRI at screening indicative of significant abnormality
2. Diagnosis of neurodegenerative disorder other than AD
3. Current diagnosis of Major Depressive Disorder (MDD)
4. Concomitant treatment with memantine.
Minimum Eligible Age

50 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alzheon Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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John Hey, PhD

Role: STUDY_DIRECTOR

Alzheon Inc.

Locations

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St. Anne's University Hospital

Brno, , Czechia

Site Status

Motol University Hospital

Prague, , Czechia

Site Status

Vestra Clinics

Rychnov nad Kněžnou, , Czechia

Site Status

Brain Research Center

's-Hertogenbosch, , Netherlands

Site Status

Brain Research Center

Amsterdam, , Netherlands

Site Status

Brain Research Center

Zwolle, , Netherlands

Site Status

Countries

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Czechia Netherlands

References

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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.

Reference Type DERIVED
PMID: 39907966 (View on PubMed)

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.

Reference Type DERIVED
PMID: 38902572 (View on PubMed)

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.

Reference Type DERIVED
PMID: 38902571 (View on PubMed)

Other Identifiers

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ALZ-801-201ADBM

Identifier Type: -

Identifier Source: org_study_id