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

Alzheon

Status and phase: Completed; Phase 2

Conditions: Early Alzheimer's Disease

Treatments: Drug: ALZ-801

Study type: Interventional

Funder types: Industry

Identifiers

NCT04693520

ALZ-801-201ADBM

Details and patient eligibility

About

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.

Full description

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)

Enrollment: 84 patients

Sex: All

Ages: 50 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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84 participants in 1 patient group

Active treatment

Experimental group

Description:

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

Treatment:

Drug: ALZ-801