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Long-term Extension of Phase 3 Study of ALZ- 801 in APOE4/4 Early AD Subjects

Phase 3
Active, not recruiting
Conditions
Early Alzheimer's Disease
Registration Number
NCT06304883
Lead Sponsor
Alzheon Inc.
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.

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
163
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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Primary Outcome Measures
NameTimeMethod
Primary cognitive efficacy endpoint 1Week 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 2Week 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 1Week 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 2Week 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 Outcome Measures
NameTimeMethod
Secondary cognitive efficacy endpoint 2Week 104

Change from baseline of the Phase 3 core study, and from baseline and from Week 52 of this study, in Neuropsychiatric Inventory.

Secondary functional efficacy endpointWeek 104

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.

Secondary global assessment efficacy endpointWeek 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 1Week 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 3Week 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 endpointWeek 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 endpointWeek 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.

Trial Locations

Locations (40)

Xenoscience, Inc.

🇺🇸

Phoenix, Arizona, United States

Banner Sun Health Research Institute

🇺🇸

Sun City, Arizona, United States

ATP Clinical Research

🇺🇸

Costa Mesa, California, United States

Torrance Clinical Research Institute

🇺🇸

Lomita, California, United States

Sutter Health

🇺🇸

Sacramento, California, United States

JEM Research Institute, Headlands Site

🇺🇸

Atlantis, Florida, United States

K2 Medical Research, LLC

🇺🇸

Maitland, Florida, United States

Mount Sinai Medical Center

🇺🇸

Miami Beach, Florida, United States

Aqualane Clinical Research

🇺🇸

Naples, Florida, United States

Charter Research

🇺🇸

Orlando, Florida, United States

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Xenoscience, Inc.
🇺🇸Phoenix, Arizona, United States

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