Fitusiran clinical trials in HA and HB with and without inhibitors
| Fitusiran trials . | Subjects . | No. . | Dose, frequency . | Results . |
|---|---|---|---|---|
| Phase 1 | ||||
| NCT0203560522 | HA and HB | N = 25 | 80 mg mo | AT reduction, 70%-89% |
| Phase 2 | ||||
| NCT0255477324 | HA and HB HA-I and HB-I | N = 14 N = 13 | 50 or 80 mg mo | Haem-A-QoL total score = −8.33 Haem-A-QoL total score = −9.16 |
| Phase 3 | ||||
| NCT0341724525,26 | HA and HB | N = 120 | 80 mg SQ mo | ABR reduced 89.9% vs OD-factor Factor use reduced 94.7%-95.9% |
| NCT0341710225,26 | HA-I and HB-I | N = 60 | 80 mg SQ mo | ABR reduced 90.8% vs OD-BPA Factor use reduced 97.5%-98.2% |
| NCT0354987127 | HA-I and HB-I | N = 65 | 80 mg SQ mo | HRQoL score = −4.6, SAE = 13.4% |
| Surgical28 | ||||
| NCT0203560522 NCT0255477324 | HA and HB HA-I and HB-I | N = 13 | 50 or 80 mg mo Lower dose CFC, BPA, tapering early | In 17 surgeries (11 major and 6 minor), blood loss was minimal or similar to that of individuals who were nonhemophilic |
| Fitusiran trials . | Subjects . | No. . | Dose, frequency . | Results . |
|---|---|---|---|---|
| Phase 1 | ||||
| NCT0203560522 | HA and HB | N = 25 | 80 mg mo | AT reduction, 70%-89% |
| Phase 2 | ||||
| NCT0255477324 | HA and HB HA-I and HB-I | N = 14 N = 13 | 50 or 80 mg mo | Haem-A-QoL total score = −8.33 Haem-A-QoL total score = −9.16 |
| Phase 3 | ||||
| NCT0341724525,26 | HA and HB | N = 120 | 80 mg SQ mo | ABR reduced 89.9% vs OD-factor Factor use reduced 94.7%-95.9% |
| NCT0341710225,26 | HA-I and HB-I | N = 60 | 80 mg SQ mo | ABR reduced 90.8% vs OD-BPA Factor use reduced 97.5%-98.2% |
| NCT0354987127 | HA-I and HB-I | N = 65 | 80 mg SQ mo | HRQoL score = −4.6, SAE = 13.4% |
| Surgical28 | ||||
| NCT0203560522 NCT0255477324 | HA and HB HA-I and HB-I | N = 13 | 50 or 80 mg mo Lower dose CFC, BPA, tapering early | In 17 surgeries (11 major and 6 minor), blood loss was minimal or similar to that of individuals who were nonhemophilic |
ABR, annualized bleed rate; BPA, bypassing agent; CFC, clotting factor concentrate; Haem-A-QoL, hemophilia A quality of life; HA-I, HA with inhibitor; HB-I, HB with inhibitor; HRQoL, health-related quality of life; OD, on-demand; SAE, serious adverse events; SQ, subcutaneous.