NCT05400330Active, not recruitingPhase 1
Long-Term Follow-up of Gene Therapy for APOE4 Homozygote Alzheimer's Disease
Long-Term Follow-Up to Evaluate the Safety of LX1001 in Participants With APOE4 Homozygote Alzheimer's Disease
Source: ClinicalTrials.gov · Last updated 1 Jul 2025
Brief summary (registry, in English)
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).
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 10 (actual)
- First submitted
- 27 May 2022
- Start date
- 8 May 2023
- Primary completion date
- Nov 2028
- Lead sponsor
- Lexeo Therapeutics
- Diseases
- Alzheimer disease
- NCT number
- NCT05400330
What you commit to
- Study type: Interventional · Phase 1
- Participants enrolled: 10
- From 8 May 2023 to the primary completion date, Nov 2028
- 2 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- Participants with any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to participant safety
Other
- 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.
Who the study is looking for
- Participants who received LX1001 in study LX1001-01
Age in the registry: 50 years to - · Sex: All · Does not accept healthy volunteers
Sites (2)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Duke University Coordinator of this site? Confirm its status | Durham | United States | Unknown status |
| PPD- Orlando Research Unit Coordinator of this site? Confirm its status | Orlando | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.