Early Alzheimer's Disease
Conditions
Keywords
Dementia, Central Nervous System Diseases, Nervous System Diseases, Tauopathies, Neurodegenerative Diseases, Neurocognitive Disorders, Mental Disorders, Alzheimer Disease
Brief summary
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.
Detailed description
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.
Interventions
ALZ-801 265 mg BID tablet orally.
Sponsors
Alzheon Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
This is a multi-center, open-label, single group, long-term extension study.
Eligibility
Sex/Gender
ALL
Age
50 Years to 85 Years
Healthy volunteers
No
Inclusion criteria
* Subject has completed the Week 78 of the Phase 3 core study (ALZ-801-AD301) while on study drug. * Subject has a reliable study partner who has sufficient contact with the subject to be able to provide accurate information about the subject's cognitive and functional abilities.
Exclusion criteria
* Significant worsening of medical conditions that may preclude completion of this study. * Evidence of symptomatic or new moderate-severe radiologic ARIA at baseline. * Has received (or plans to receive) amyloid antibodies since completing Phase 3 core study (ALZ-801-AD301). * Subject taking any prohibited medications per protocol.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Primary cognitive efficacy endpoint 1 | Week 104 | 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). |
| Primary cognitive efficacy endpoint 2 | Week 104 | 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. |
| Incidence, Nature, and Severity of Treatment Emergent Adverse events (TEAEs) | Week 104 | Safety and tolerability as measured by incidence, nature and severity of treatment emergent adverse events (TEAE), serious TEAE, and TEAE leading to withdrawal. |
| Primary imaging biomarker endpoint 1 | Week 104 | 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). |
| Primary imaging biomarker endpoint 2 | Week 104 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Secondary functional efficacy endpoint | Week 104 | 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. |
| Secondary global assessment efficacy endpoint | Week 104 | 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. |
| Secondary cognitive efficacy endpoint 1 | Week 104 | 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. |
| Secondary cognitive efficacy endpoint 2 | Week 104 | Change from baseline of the Phase 3 core study, and from baseline and from Week 52 of this study, in Neuropsychiatric Inventory. |
| Secondary cognitive efficacy endpoint 3 | Week 104 | Change from baseline of the Phase 3 core study, and from baseline and from Week 52 of this study, in Mini-Mental State Examination. |
| Secondary imaging biomarker endpoint | Week 104 | 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. |
| Secondary fluid biomarker endpoint | Week 104 | 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. |
Countries
Canada, United Kingdom, United States
Contacts
PRINCIPAL_INVESTIGATORSusan Abushakra, MD
Alzheon Inc.
Outcome results
None listed