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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01983241 | Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD) | PHASE3 | ACTIVE NOT_RECRUITING | 345 | — | — | Nov 1, 2013 | Jan 1, 2027 | Mar 4, 2026 | 52 | United States, Argentina +15 |
Whole lung PD15 measured by CT scan
| Arm | Type | Description |
|---|---|---|
| Alpha-1 MP 60 mg/kg | EXPERIMENTAL | Alpha-1 MP 60 mg/kg administered weekly by IV infusion for 156 weeks |
| Alpha-1 MP 120 mg/kg | EXPERIMENTAL | Alpha-1 MP 120 mg/kg administered weekly by IV infusion for 156 weeks |
| Placebo | PLACEBO_COMPARATOR | 0.9% Sodium Chloride for Injection, USP, administered weekly by IV infusion for 156 weeks |
| Name | Type | Description |
|---|---|---|
| Alpha-1 MP | BIOLOGICAL | - |
| 0.9% Sodium Chloride for Injection, USP | OTHER | - |
Inclusion Criteria: * Have a documented total alpha1-PI serum level \< 11 µM. * Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or "at-risk" alleles. * At the Screening (Week -3) Visit, have a post-bronchodilator forced expiratory volume in...