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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06449001 | Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis | PHASE3 | RECRUITING | 6 | — | — | Aug 11, 2025 | Mar 10, 2028 | Dec 23, 2025 | 4 | Canada, France +1 |
| NCT05389449 | A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH | PHASE3 | ACTIVE NOT_RECRUITING | 80 | — | — | Oct 28, 2022 | Jul 31, 2026 | Apr 16, 2026 | 45 | United States, Brazil +13 |
| NCT04469465 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA) | PHASE3 | COMPLETED | 86 | — | — | Dec 16, 2020 | Jan 16, 2024 | May 4, 2025 | 62 | United States, Brazil +16 |
Baseline was defined as the lowest Hgb value observed between and including Screening and Day 1. The least square (LS) mean and standard error (SE) were produced using mixed-effect model for repeated measures (MMRM). Hgb values collected within 4 weeks after transfusion were not included in the MMRM.
| Arm | Type | Description |
|---|---|---|
| Danicopan | EXPERIMENTAL | Participants will receive a 12-week weight-based open-label treatment period and up to 1 year open-label long term extension period. |
| Danicopan + C5 Inhibitor | EXPERIMENTAL | Participants will receive danicopan, in addition to their C5 inhibitor therapy, for 24 weeks (12 weeks in Treatment Period 1, followed by 12 weeks in Treatment Period 2). |
| Placebo + C5 Inhibitor | PLACEBO_COMPARATOR | Participants will receive placebo, in addition to their C5 inhibitor therapy, for 12 weeks during Treatment Period 1. At Week 12, participants randomized to receive placebo will be switched to danicopan for an additional 12 weeks (Treatment Period 2). |
| Name | Type | Description |
|---|---|---|
| Danicopan | DRUG | Participants will receive danicopan on a weight-based dosing regimen. |
| Placebo | DRUG | Oral tablet |
| C5 Inhibitor | DRUG | Participants will continue to receive their ongoing C5 inhibitor (eculizumab or ravulizumab) therapy according to their usual dose and schedule. |
Inclusion Criteria: * Confirmed diagnosis of PNH. * CS-EVH defined by: Anemia: Hgb ≤ 11.0 g/dL, and absolute reticulocyte count ≥ 100 × 109/L * Treated with ravulizumab or eculizumab for at least 12 weeks immediately preceding Day 1, the dose received should be stable during this period, and there ...