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Long-Term Follow-up of Gene Therapy for APOE4 Homozygote Alzheimer's Disease

NCT05400330 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2025-07-01

No results posted yet for this study

Summary

The primary purpose of this long-term follow-up study is to assess the long-term safety profile of APOE4 homozygote participants who were administered gene therapy (LX1001) for the treatment of Alzheimer's disease in Study LX100101. A secondary objective is to assess the biomarker as shown by the conversion of CSF APOE isoforms from APOE4 to APOE2-APOE4. Additional secondary outcomes include amyloid PET scan, CSF markers (including Aβ42, Aβ42/Aβ40 ratio T--tau, and P-tau), and quantitative MRI (and other biomarkers that may be informative for this therapeutic approach). Other secondary objectives include instruments to assess cognitive and clinical AD and to evaluate if treatment with AAVrh.10hAPOE2 improves brain tau pathology with tau PET scan (LX1001-01 Cohorts 3 and 4 only).

Conditions

  • Alzheimer Disease

Interventions

BIOLOGICAL

LX1001

Gene therapy

Sponsors & Collaborators

  • Lexeo Therapeutics

    lead INDUSTRY

Principal Investigators

  • Lexeo Clinical Trials · Lexeo Therapeutics

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-05-08
Primary Completion
2028-11-30
Completion
2028-11-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05400330 on ClinicalTrials.gov