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LX1001 for Early-Onset Alzheimer's Disease

Phase-Based Progress Estimates

Effectiveness

Safety

Early-Onset Alzheimer's Disease+1 MoreLX1001 - Biological

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Eligibility

18+

All Sexes

What conditions do you have?

Select

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

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ALN-APP

Group 1

3TC

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

ALN-APP

Group 1

3TC

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