LX1001 for Early-Onset Alzheimer's Disease

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Early-Onset Alzheimer's Disease+1 MoreLX1001 - Biological
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

This trial is testing a gene therapy to treat patients with Alzheimer's who have two copies of the APOE4 gene. The trial will test different doses to see what is effective.

Eligible Conditions
  • Early-Onset Alzheimer's Disease
  • Alzheimer's Disease

Treatment Effectiveness

Effectiveness Progress

1 of 3

Similar Trials

Study Objectives

5 Primary · 14 Secondary · Reporting Duration: 1 year

1 year
Assess the levels of CSF biomarkers
Change from baseline in APOE2-APOE4 isoforms
Change from baseline in Clinical Dementia Rating Global and Sum of Boxes (CDR-SB)
Change from baseline in brain amyloid plaques as assessed by PET scan
Change from baseline in hippocampal and entorhinal cortex volumes as assessed by brain magnetic resonance imaging (MRI)
Change from baseline in levels of CSF biomarkers
Change from baseline in the Alzheimer's Disease Assessment Scale - Cognition 13 (ADAS-Cog 13)
Change from baseline in the Mini Mental State Examination (MMSE)
Clinical status as assessed using the Clinical Dementia Rating Global and Sum of Boxes (CDR-SB)
Alzheimer's Disease
Cognitive status as assessed using the Mini Mental State Examination (MMSE)
Evaluate brain amyloid plaques as assessed by PET scan
Hippocampal and entorhinal cortex volumes as assessed by brain magnetic resonance imaging (MRI)
Maximum tolerated dose of LX1001 gene therapy to APOE4 homozygotes with Alzheimer's disease
Proportion of participants with treatment-emergent adverse events and serious adverse events
Safety of LX1001 gene therapy, based on adverse events and serious adverse events
Shift from baseline in APOE2-APOE4 isoforms
The proportion of participants with treatment-emergent AEs and SAEs at each dosage
The proportion of participants with treatment-emergent adverse events and serious adverse events at each dosage

Trial Safety

Safety Progress

1 of 3

Similar Trials

Trial Design

6 Treatment Groups

Cohort II: 1.6 x 10^11 gc/mL CSF
1 of 6
Cohort 2: 4.4 x 10^10 gc/mL CSF
1 of 6
Cohort 1: 1.4 x 10^10 gc/mL CSF
1 of 6
Cohort 3: 1.4 x 10^11 gc/mL CSF
1 of 6
Cohort I: 5.0 x 10^10 gc/mL CSF
1 of 6
Cohort III: 5.0 x 10^11 gc/mL CSF
1 of 6

Experimental Treatment

15 Total Participants · 6 Treatment Groups

Primary Treatment: LX1001 · No Placebo Group · Phase 1 & 2

Cohort II: 1.6 x 10^11 gc/mL CSF
Biological
Experimental Group · 1 Intervention: LX1001 · Intervention Types: Biological
Cohort 2: 4.4 x 10^10 gc/mL CSF
Biological
Experimental Group · 1 Intervention: LX1001 · Intervention Types: Biological
Cohort 1: 1.4 x 10^10 gc/mL CSF
Biological
Experimental Group · 1 Intervention: LX1001 · Intervention Types: Biological
Cohort 3: 1.4 x 10^11 gc/mL CSF
Biological
Experimental Group · 1 Intervention: LX1001 · Intervention Types: Biological
Cohort I: 5.0 x 10^10 gc/mL CSF
Biological
Experimental Group · 1 Intervention: LX1001 · Intervention Types: Biological
Cohort III: 5.0 x 10^11 gc/mL CSF
Biological
Experimental Group · 1 Intervention: LX1001 · Intervention Types: Biological

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 1 year

Who is running the clinical trial?

Weill Medical College of Cornell UniversityOTHER
989 Previous Clinical Trials
1,338,915 Total Patients Enrolled
Lexeo TherapeuticsLead Sponsor
2 Previous Clinical Trials
25 Total Patients Enrolled
Alzheimer's Drug Discovery FoundationOTHER
19 Previous Clinical Trials
2,983 Total Patients Enrolled
Lexeo Clinical TrialsStudy DirectorLexeo Therapeutics
1 Previous Clinical Trials
15 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 9 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You are able to give informed consent.
You have a clinical diagnosis of mild cognitive impairment due to Alzheimer's disease or mild to moderate dementia due to Alzheimer's disease.
You have evidence of amyloid plaques by nuclear PET scan and CSF biomarkers consistent with Alzheimer's disease.
The titer of neutralizing anti-AAVrh10 antibody is less than 1:100.
Fertile individuals will be prescribed barrier birth control methods to prevent pregnancy for the duration of the study.
You agree not to post your personal data related to the study on social media.