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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06672237 | A Phase 3 Study of NTLA-2001 in ATTRv-PN | PHASE3 | RECRUITING | 60 | — | — | Nov 22, 2024 | Aug 1, 2028 | Apr 16, 2026 | 14 | Argentina, Brazil +4 |
| Arm | Type | Description |
|---|---|---|
| nexiguran ziclumeran | EXPERIMENTAL | nexiguran ziclumeran 55 mg by single IV infusion |
| Normal Saline | PLACEBO_COMPARATOR | Placebo; Normal saline (0.9% NaCl) by single IV infusion |
| Name | Type | Description |
|---|---|---|
| nexiguran ziclumeran | BIOLOGICAL | nexiguran ziclumeran 55 mg by single IV infusion |
| Normal Saline as Placebo | DRUG | Normal saline (0.9% NaCl) by single IV infusion |
Inclusion Criteria: * Diagnosis of ATTRv-PN * Karnofsky Performance Status (KPS) ≥ 60 Exclusion Criteria: * Other causes of amyloidosis (amyloidosis caused by non-TTR protein) * Other known causes of sensorimotor or autonomic neuropathy * Diabetes mellitus * New York Heart Association Class III o...