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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05133531 | A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment | PHASE3 | ACTIVE NOT_RECRUITING | 202 | — | — | Aug 1, 2022 | Oct 26, 2026 | Apr 30, 2026 | 68 | United States, Brazil +22 |
Cohort A
Cohort B Not requiring a red blood cell (RBC) transfusion per the protocol
Cohort B LDH ≤1.5 × ULN at each visit
| Arm | Type | Description |
|---|---|---|
| Cohort A | EXPERIMENTAL | Randomized 1:1 |
| Cohort B | EXPERIMENTAL | Randomized 1:1 |
| Name | Type | Description |
|---|---|---|
| Ravulizumab | DRUG | Administered Intravenous (IV) per the protocol |
| Pozelimab | DRUG | Administered IV and subcutaneous (SC) per the protocol |
| Cemdisiran | DRUG | Administered SC per the protocol |
| Eculizumab | DRUG | Administered IV per the protocol |
Key Inclusion Criteria: 1. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes as described in the protocol 2. Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms as described in the protocol 3. LDH level ≥2 × UL...