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NCT Number: NCT05400330

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 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).

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Alzheimer Disease Brain Diseases Central Nervous System Diseases Dementia Mental Disorders Nervous System Diseases Neurocognitive Disorders Neurodegenerative Diseases Tauopathies

Age range

50 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

PPD- Orlando Research Unit, Orlando, Florida, United States

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About this study

This is a long-term follow-up study to evaluate the safety following LX1001, a gene therapy, for participants who are APOE4 homozygotes with clinical diagnoses varying from MCI or dementia due to AD who have previously received LX1001. Study LX1001-01 was designed to assess the safety of LX1001 at 4 ascending doses (1.4 × 1010, 4.4 × 1010, 1.4 × 1011 gene copy [gc]/mL CSF and 1.4 x 1014 [fixed dose]) as per droplet digital polymerase chain reaction methodology, with each group consisting of approximately n=3-5 individuals for a total of approximately 15 participants for the entire study.

In this study, participants who have received LX1001 in the parent protocol (LX1001-01) will be followed for up to 260 weeks post gene therapy administration to assess the safety and efficacy parameters (~208 weeks within this study)

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Participants who received LX1001 in study LX1001-01

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.

Treatment and study plan

LX1001

Biological

Gene therapy

Other names: AAVrh.10hAPOE2

Primary outcomes

  1. Incidence of treatment emergent adverse events

    Time frame: 260 weeks

    All emergent adverse events will be collected

  2. Incidence of serious adverse events

    Time frame: 260 weeks

    All incidents of serious adverse events will be collected

Sponsors and collaborators

Lead sponsor

Lexeo Therapeutics

Industry

Registry information

Official study title

Long-Term Follow-Up to Evaluate the Safety of LX1001 in Participants With APOE4 Homozygote Alzheimer's Disease

Acronym: LEADLTFU

Important dates

Study start
2023
Primary completion
2028
Study completion
2028
First posted
Jun 1, 2022
Registry last updated
Jul 1, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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