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NCT05400330 · ClinicalTrials.gov registry record · Phase 1

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

A Phase 1 study of Alzheimer Disease, sponsored by Lexeo Therapeutics.

Active
Registry status
Phase 1
Development phase
10
Enrollment target
2
Study locations

NCT05400330: Active Phase 1 study of Alzheimer Disease, sponsored by Lexeo Therapeutics.

NCT05400330 is a Phase 1 study of Alzheimer Disease that is active but no longer recruiting, run by Lexeo Therapeutics. The registered enrollment target is 10 participants, below the 2,751-participant average among 306 other Alzheimer Disease trials with a reported enrollment target (100% lower). The trial reports 2 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT05400330, a Phase 1 study of Alzheimer Disease, is active but no longer recruiting, sponsored by Lexeo Therapeutics.

ACTIVE NOT RECRUITING
Registry status
Phase 1
Development phase
10 participants
Enrollment target
2
Study locations

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).

Primary Outcome

All emergent adverse events will be collected

Conditions Studied

Interventions

  • BIOLOGICAL LX1001

Study Locations (2)

Florida

  • PPD- Orlando Research Unit - Orlando

North Carolina

  • Duke University - Durham

Trial Details

FieldValue
Enrollment Target 10 participants
Start Date 2023-05-08
Est. Completion 2028-11
Phase Phase 1
Lexeo Therapeutics

6 total trials

What the registry record for NCT05400330 still lists

NCT05400330 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 2,751-participant average among 306 other Alzheimer Disease trials with a reported enrollment target (100% lower).

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.

NCT05400330 reports a single indexed study location in Florida, North Carolina.

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.

What interventions are being tested in trial NCT05400330?

The interventions under investigation include: LX1001 (BIOLOGICAL).

Who is sponsoring clinical trial NCT05400330?

This trial is sponsored by Lexeo Therapeutics, which has 6 total clinical trials registered on ClinicalTrials.gov.

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.

Similar trials for Alzheimer Disease

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT05400330's enrollment target sits among peer trials

10 291st of 306 higher than 13 of 306 other Alzheimer Disease trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Alzheimer Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT05400330, the US trial registry maintained by the National Library of Medicine. NCT05400330 (small enrollment · single site footprint · active not recruiting) retrieved and formatted by PlainTrial, see methodology.