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

Condition(s) studied

Investigational drug(s) / intervention(s)

LX1001: Gene therapy

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).

Eligibility

Primary outcome measure(s)

• Incidence of treatment emergent adverse events — 260 weeks
  All emergent adverse events will be collected
• Incidence of serious adverse events — 260 weeks
  All incidents of serious adverse events will be collected

Trial sites (2)

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