An Efficacy and Safety Study of ALZ-801 in APOE4/4 Early AD Subjects (APOLLOE4)

Alzheon

Status and phase: Completed; Phase 3

Conditions: Early Alzheimer's Disease

Treatments: Drug: Placebo Comparator: Placebo; Drug: Experimental: ALZ-801

Study type: Interventional

Funder types: Industry; NIH

Identifiers

NCT04770220

ALZ-801-AD301

2020-005755-20 (EudraCT Number)

R01AG065253 (U.S. NIH Grant/Contract)

Details and patient eligibility

About

This study is being conducted to evaluate the safety and efficacy of ALZ-801 in Early Alzheimer's disease (AD) subjects with the APOE4/4 genotype. This is a double-blind, randomized trial with one dose of ALZ-801 compared to placebo.

Full description

This is a multi-center, double-blind study that will evaluate 265 mg BID of ALZ-801, an oral tablet, over 78 weeks as a treatment for subjects (50-80 years old) with Early AD who are homozygous for the ε4 allele of the apolipoprotein gene (APOE4 homozygous or APOE4/4). 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 soluble biomarkers of AD and neurodegeneration will be measured and a sub-study to evaluate cerebrospinal fluid (CSF) biomarkers is also included.

Enrollment: 325 patients

Sex: All

Ages: 50 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Clinical diagnosis of MCI or Mild Dementia due to AD consistent with the National Institute on Aging-Alzheimer's Association (NIA-AA) Working Group Criteria.
• Homozygous for the ε4 allele of the apolipoprotein E gene (APOE4/4).
• MMSE score at Screening of 22 to 30 (inclusive).
• CDR - Global score of 0.5 or 1 and CDR Memory Box Score of ≥ 0.5.
• RBANS delayed memory index score ≤ 85.
• Evidence of progressive memory loss over the last 12 months per investigator assessment

Exclusion criteria

• Brain magnetic resonance imaging (MRI) indicative of significant abnormality per central reader, other than AD related atrophy. Computed tomography (CT) scan acceptable for subjects who cannot undergo MRI.
• Diagnosis of neurodegenerative disorder other than AD.
• Diagnosis of major depressive disorder (MDD) within one year prior to screening.
• Currently taking memantine or has taken memantine within 12 weeks prior to the Baseline Visit.
• History of suicidal behavior within one year prior to screening or has ongoing suicidal ideation.
• History of seizures, excluding febrile seizures of childhood or a single distant seizure (> 5 years).
• Medically confirmed history of recent cerebral infarct or transient ischemic attack within one year prior to screening.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

325 participants in 2 patient groups, including a placebo group

ALZ-801

Experimental group

Description:

ALZ-801 265 mg BID tablet orally. Subjects will receive placebo in the morning and one table of ALZ-801 265mg tablet in the evening during the first two weeks of the study; thereafter, they will receive a 265mg tablet BID.

Treatment:

Drug: Experimental: ALZ-801

Placebo

Placebo Comparator group

Description:

Subjects in the placebo treatment arm will receive placebo tablets BID throughout the study

Treatment:

Drug: Placebo Comparator: Placebo