Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
LX1001 · 2 trials · 2 indications
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...
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