Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05400330 | Long-Term Follow-up of Gene Therapy for APOE4 Homozygote Alzheimer's Disease | PHASE1 | ACTIVE NOT_RECRUITING | 10 | — | — | May 8, 2023 | Nov 1, 2028 | Jul 1, 2025 | 2 | United States |
| NCT03634007 | Gene Therapy for APOE4 Homozygote of Alzheimer's Disease | PHASE1 | COMPLETED | 15 | — | — | Nov 6, 2019 | Nov 7, 2024 | Oct 20, 2025 | 4 | United States |
All emergent adverse events will be collected
All incidents of serious adverse events will be collected
Adverse events categorized and graded
Adverse events categorized and graded per study drug dose
| Arm | Type | Description |
|---|---|---|
| Previously administered LX1001 | EXPERIMENTAL | 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 |
| Cohort 1: 1.4 x 10^10 gc/mL CSF | EXPERIMENTAL | Participants will receive 1.4 x 10\^10 gc/mL CSF of LX1001. |
| Cohort 2: 4.4 x 10^10 gc/mL CSF | EXPERIMENTAL | Participants will receive 4.4 x 10\^10 gc/mL CSF of LX1001. |
| Cohort 3: 1.4 x 10^11 gc/mL CSF | EXPERIMENTAL | Participants will receive 1.4 x 10\^11 gc/mL CSF of LX1001. |
| Cohort 4: 1.4 x 10^14 gc (fixed dose) | EXPERIMENTAL | Participants will receive 1.4 x 10\^14 gc (fixed dose; approximately 3.4 × 10\^11 gc/mL CSF based on an average CSF volume of 409 mL) of LX1001. |
| Name | Type | Description |
|---|---|---|
| LX1001 | BIOLOGICAL | Gene therapy |
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 medi...