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).
Protocol Amendment History
10 amendments
This ClinicalTrials.gov record has been amended 10 times since 2022-05-27; most recent amendment 2025-06-30.
Status change: Enrolling by Invitation → Active, Not Recruiting2025-06-30
Status change: Active, Not Recruiting → Enrolling by Invitation2025-03-25
Status change: Recruiting → Active, Not Recruiting2025-02-18
Status change: Not Yet Recruiting → Recruiting2023-06-28