A Phase 3 interventional study of Experimental: ALZ-801 in Early Alzheimer's Disease, sponsored by Alzheon Inc.. Terminated at 41 sites in 3 countries. Open to participants aged 50 Years to 85 Years. Per ClinicalTrials.gov, last updated 2026-06-25.
Sponsored by Alzheon Inc. · Phase 3, Interventional, and Treatment
This study is being conducted to evaluate the long-term safety and efficacy of ALZ-801 in Early Alzheimer's disease (AD) subjects with the APOE4/4 genotype. This is an open-label trial of treatment with ALZ-801.
This is a long-term extension study of the Phase 3, multicenter, randomized, double-blind, placebo-controlled study of the efficacy, safety, and imaging biomarker effects of ALZ-801 in subjects with Early Alzheimer's Disease and APOE4/4 genotype. Subjects who at initial screening for the Phase 3 study were 50-80 years old, had a clinical diagnosis of AD, carried the APOE4/4 genotype, and were at the early stage of disease (Early AD], who complete at least 78 weeks of the Phase 3 study while on study medication, were eligible for enrollment. Subjects will be treated for 104 weeks with ALZ-801, followed by a 4-week safety follow-up visit after the last dose of ALZ-801. Clinical trial sites, subjects and their study partner will remain blinded to the treatment (ALZ-801 or placebo) that they received in the core Phase 3 study. The primary efficacy outcome assessment is a measure of cognition (ADAS-Cog 13). Additional measures of global and functional impairments will also be assessed. Imaging and biomarkers of AD and neurodegeneration will be measured.
3,965 studies on the registry are indexed under Alzheimer Disease; 926 are open to participants now.
This study's enrollment of 163 is above the median of 70 across 3,019 interventional studies indexed under Alzheimer Disease.
Browse Alzheimer Disease studies →Alzheon Inc. is the lead sponsor of 6 studies on the registry; 1 is open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 1 (20%) have results posted.
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Exclusion Criteria:
ALZ-801 265 mg BID tablet orally. Subjects will take one 265 mg tablet of ALZ-801 in the evening during the first 4 weeks of the study; thereafter, they will take one 265 mg tablet BID.
Drug: Experimental: ALZ-801
ALZ-801 265 mg BID tablet orally.
Primary cognitive efficacy endpoint 1
Change from baseline in Alzheimer's Disease Assessment Scale - Cognitive Subscale 13 (ADAS-Cog 13), from baseline of Phase 3 core study (ALZ-801-AD301) to Week 52 and Week 104 of this long-term extension study (ALZ-801-AD351).
Time frame: Week 104
Primary cognitive efficacy endpoint 2
Change from baseline in Alzheimer's Disease Assessment Scale - Cognitive Subscale 13 (ADAS-Cog 13), from baseline of this study to Week 52 and Week 104.
Time frame: Week 104
Incidence, Nature, and Severity of Treatment Emergent Adverse events (TEAEs)
Safety and tolerability as measured by incidence, nature and severity of treatment emergent adverse events (TEAE), serious TEAE, and TEAE leading to withdrawal.
Time frame: Week 104
Primary imaging biomarker endpoint 1
Change from baseline in total hippocampal volume (mm3) as measured by Magnetic Resonance Imaging (MRI), from baseline of the Phase 3 core study (ALZ-801-AD301) to Week 52 and Week 104 of this long-term extension study (ALZ-801-AD351).
Time frame: Week 104
Primary imaging biomarker endpoint 2
Change from baseline in total hippocampal volume (mm3) as measured by Magnetic Resonance Imaging (MRI), from baseline of this study to Week 52 and Week 104.
Time frame: Week 104
Secondary functional efficacy endpoint
Change from baseline of the Phase 3 core study, and from baseline and from Week 52 of this study, in Disability Assessment for Dementia scores.
Time frame: Week 104
Secondary functional efficacy endpoint
Change from baseline of the Phase 3 core study, and from baseline and from Week 52 of this study, in Amsterdam - Instrumental Activities of Daily Living scores.
Time frame: Week 104
Secondary global assessment efficacy endpoint
Change from baseline of the Phase 3 core study, and from baseline and from Week 52 of this study, in Clinical Dementia Rating - Sum of Boxes (CDR-SB) scores.
Time frame: Week 104
Secondary cognitive efficacy endpoint 1
Change from baseline of the Phase 3 core study, and from baseline and from Week 52 of this study, in Alzheimer's Disease Assessment Scale - Cognitive Subscale 11.
Time frame: Week 104
Secondary cognitive efficacy endpoint 2
Change from baseline of the Phase 3 core study, and from baseline and from Week 52 of this study, in Neuropsychiatric Inventory.
Time frame: Week 104
Secondary cognitive efficacy endpoint 3
Change from baseline of the Phase 3 core study, and from baseline and from Week 52 of this study, in Mini-Mental State Examination.
Time frame: Week 104
Secondary imaging biomarker endpoint
Change from baseline of the Phase 3 core study (ALZ-801-AD301) and from baseline of this long-term extension study (ALZ-801-AD351) in cortical thickness, whole brain volume and ventricular volume (mm3) as measured by Magnetic Resonance Imaging (MRI) to Weeks 26, 52, 78 and 104.
Time frame: Week 104
Secondary fluid biomarker endpoint
Change from baseline of the Phase 3 core study, and from baseline and from Week 52 of this study in plasma p-tau181, Aβ 42, Aβ 40, GFAP, and NfL levels.
Time frame: Week 104
Ran from Apr 2024 to primary completion in Mar 2026, 23 months.
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This study is terminated, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: ALZ-801
Alzheon Inc.