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Long-Term Follow-up of Gene Therapy for APOE4 Homozygote Alzheimer's Disease
NCT05400330 · View on ClinicalTrials.gov ↗
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 Studied
Interventions
- BIOLOGICAL LX1001
Study Locations (2)
Florida
- PPD- Orlando Research Unit — Orlando
North Carolina
- Duke University — Durham
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2023-05-08 |
| Est. Completion | 2028-11 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05400330
The ClinicalTrials.gov registry entry for NCT05400330 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 10 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Lexeo Therapeutics, which has 5 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Alzheimer Disease appearing as the primary indexed condition, and to 1 intervention — of which LX1001 is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05400330 reports 2 study locations spanning 2 distinct geographic areas — top geographies include Florida, North Carolina. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05400330 about?
NCT05400330 is a clinical study titled "Long-Term Follow-up of Gene Therapy for APOE4 Homozygote Alzheimer's Disease". 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 co...
What is the current status of trial NCT05400330?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2023-05-08. Estimated completion is 2028-11.
What conditions does trial NCT05400330 study?
This clinical trial studies the following conditions: Alzheimer Disease. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05400330?
The interventions under investigation include: LX1001 (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05400330?
This trial is sponsored by Lexeo Therapeutics, which has 5 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05400330 being conducted?
This trial has 2 study locations across Florida, North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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