Gene Therapy for APOE4 Homozygote of Alzheimer's Disease

Alzheimer Disease Clinical Trial

Official title: A 52-Week, Multicenter, Phase 1/2 Open-label Study to Evaluate the Safety of LX1001 in Participants With APOE4 Homozygote Alzheimer's Disease

Status Active, not recruiting

Clinical Trial Summary

This clinical trial is an open label, dose-ranging study designed to evaluate gene therapy to treat patients who are APOE4 homozygotes with clinical diagnosis varying from mild cognitive impairment due to Alzheimer's, mild dementia due to Alzheimer's disease, and moderate dementia due to Alzheimer's disease.

Clinical Trial Description

The study will assess the safety and toxicity of intrathecal administration of AAVrh.10hAPOE2 (LX1001), serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the complementary deoxyribonucleic acid (cDNA) coding for human apolipoprotein E2 (APOE2), directly to the central nervous system (CNS)/ CSF of APOE4 homozygotes with Alzheimer's disease. All subjects will have evidence of cerebrospinal fluid (CSF) biomarkers consistent with Alzheimer's disease. The study will establish a maximum tolerable dose and generate preliminary evidence regarding whether direct administration of LX1001 to the CNS of those Alzheimer's patients will lead to conversion of the APOE protein isoforms in the CSF of APOE4 homozygotes from APOE4 to APOE2-APOE4. ;

Related Conditions & MeSH terms

• Alzheimer Disease
• Early Onset Alzheimer Disease

• NCT number: NCT03634007
• Study type: Interventional
• Source: Lexeo Therapeutics
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: November 6, 2019
• Completion date: November 2024