Long-Term Follow-up of Gene Therapy for APOE4 Homozygote Alzheimer's Disease (NCT05400330)
๐ฉบ Plain-English Summary (8th-Grade Level)
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 Aฮฒ42, Aฮฒ42/Aฮฒ40 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).
Who can join: To join this study, participants generally need to meet the listed inclusion criteria (1 items) and avoid the listed exclusions (3 items).
Location: PPD- Orlando Research Unit โ Orlando, Florida
๐ "Do I Qualify?" โ 1-Minute Self Screener
Instant ChecklistCheck every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).
Usually NOT eligible if any of the following apply:
- Exclusion Criteria:
- Participants with any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to participant safety
- Participants who agree not to post their personal medical data in relation to this study or any study information online, including social media sites, until all participants have completed all LX1001 clinical studies, including long-term follow-up.
๐ Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.
๐ All Participating Trial Locations (2 Sites)
- PPD- Orlando Research Unit โ Orlando, Florida
- Duke University โ Durham, North Carolina
๐ฐ Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA ยง 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.