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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03748823 | Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab | PHASE3 | COMPLETED | 139 | — | — | Feb 19, 2019 | Aug 31, 2023 | Sep 19, 2024 | 51 | United States, Australia +12 |
| Arm | Type | Description |
|---|---|---|
| Ravulizumab SC Treatment Group | EXPERIMENTAL | In the Randomized Treatment Period, participants will receive an IV loading dose of ravulizumab on Day 1 followed by SC maintenance doses of ravulizumab administered via the ravulizumab OBDS on Day 15 and every week (qw) thereafter for a total of 10 weeks of study treatment. In the Extension Period, ravulizumab SC will be administered via the ravulizumab OBDS from Day 71 qw through Day 1274. |
| Ravulizumab IV Treatment Group | ACTIVE_COMPARATOR | In the Randomized Treatment Period, participants will receive an IV loading dose of ravulizumab on Day 1 followed by IV maintenance doses of ravulizumab on Day 15. In the Extension Period, ravulizumab SC will be administered via the ravulizumab OBDS from Day 71 qw through Day 1274. |
| Name | Type | Description |
|---|---|---|
| Ravulizumab OBDS | COMBINATION_PRODUCT | The ravulizumab OBDS is a biological-device combination product consisting of a prefilled cartridge containing ravulizumab SC and an on-body injector. |
| Ravulizumab | BIOLOGICAL | Administered by IV infusion. Ravulizumab IV doses will be based on participant body weight. |
Inclusion Criteria: * Male or female ≥18 years of age * Treated with eculizumab for PNH for at least 3 months prior to Day 1 * LDH level ≤1.5 × upper limit of normal (ULN) at screening * PNH diagnosis confirmed by documented high-sensitivity flow cytometry. * Documented meningococcal vaccination no...