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

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ClinicalTrials.gov Identifier: NCT05400330
Recruitment Status : Recruiting
First Posted : June 1, 2022
Last Update Posted : November 15, 2023
Sponsor:
Information provided by (Responsible Party):
Lexeo Therapeutics

Study Description
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Brief 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 Cohort 3 only).

Condition or disease Intervention/treatment Phase
Alzheimer Disease Biological: LX1001 Phase 1

Detailed Description:

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 3 ascending doses (1.4 × 1010, 4.4 × 1010, 1.4 × 1011gene copy [gc]/mL CSF) as per droplet digital polymerase chain reaction methodology, with each group consisting of approximately n=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)

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Long-Term Follow-Up to Evaluate the Safety of LX1001 in Participants With APOE4 Homozygote Alzheimer's Disease
Actual Study Start Date : May 8, 2023
Estimated Primary Completion Date : November 2028
Estimated Study Completion Date : November 2028

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Previously administered LX1001

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 3 ascending doses (1.4 × 1010, 4.4 × 1010, 1.4 × 1011 gene copy [gc]/mL CSF) as per droplet digital polymerase chain reaction methodology, with each group consisting of approximately n=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

 Biological: LX1001
Gene therapy
Other Name: AAVrh.10hAPOE2


Outcome Measures
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Primary Outcome Measures :
  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


Eligibility Criteria
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Ages Eligible for Study:   50 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05400330


Contacts
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Contact: Lexeo Clinical Trials +1 212-547-9879 clinicaltrials@lexeotx.com
Contact: Lexeo Clinical Trials

Locations
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United States, Florida
PPD- Orlando Research Unit Recruiting
Orlando, Florida, United States, 32806
Contact: Shannon Killingsworth    689-216-3100    PPDOrlandoRecruitment.sm@ppd.com   
United States, North Carolina
Duke University Not yet recruiting
Durham, North Carolina, United States, 27708
Contact: Sidney Wright    919-681-9249    sidney.fitz@duke.edu   
Sponsors and Collaborators
Lexeo Therapeutics
Investigators
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Study Director: Lexeo Clinical Trials Lexeo Therapeutics
More Information
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Responsible Party: Lexeo Therapeutics
ClinicalTrials.gov Identifier: NCT05400330    
Other Study ID Numbers: LX1001-02
First Posted: June 1, 2022    Key Record Dates
Last Update Posted: November 15, 2023
Last Verified: November 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Alzheimer Disease
Dementia
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Tauopathies
Neurodegenerative Diseases
Neurocognitive Disorders
Mental Disorders