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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07285460 | A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B | PHASE3 | RECRUITING | 85 | — | — | Dec 18, 2025 | Dec 30, 2031 | May 26, 2026 | 27 | United States, Belgium +10 |
| NCT05662319 | A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia | PHASE3 | ACTIVE NOT_RECRUITING | 91 | — | — | Feb 1, 2023 | Jan 25, 2029 | Feb 4, 2026 | 50 | United States, Canada +15 |
| NCT03974113 | Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B | PHASE3 | ACTIVE NOT_RECRUITING | 32 | — | — | Jan 28, 2020 | Dec 15, 2026 | Aug 8, 2025 | 16 | United States, Canada +4 |
| NCT03754790 | Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX | PHASE3 | ACTIVE NOT_RECRUITING | 281 | — | — | Jan 9, 2019 | Nov 5, 2026 | Dec 17, 2025 | 79 | United States, Australia +18 |
| NCT03549871 | A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis | PHASE3 | COMPLETED | 80 | — | — | Jul 25, 2018 | Mar 25, 2022 | Feb 6, 2023 | 35 | United States, Australia +13 |
A bleeding episode is defined as any occurrence of hemorrhage that requires administration of CFCs or BPAs
A bleeding episode is defined as any occurrence of hemorrhage that requires administration of CFCs or BPAs, e.g., hemarthrosis, muscle, or mucosal bleeding.
Characterize the AT activity at the optimal therapeutic dose
The number of participants experiencing any TEAEs, serious TEAEs, discontinuation due to TEAEs and death will be reported
Bleeding episodes (BE): any occurrence of hemorrhage might require administration of factor/BPA. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location occurred within 72 hours of last injection used to treat BE at that location was considered part of original BE and counted as 1 BE towards ABR. Bleeding began after 72 hours of last injection at that location was considered as a new event. ABR = total number of qualifying BE/total number of days in the respective period\*365.25. Estimated data were derived by using repeated measures negative binomial (NB) regression model.
A bleeding episode (BE): any occurrence of hemorrhage might require administration of factor/BPA. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection used to treat BE at that location was considered a part of original BE and counted as 1 BE towards ABR. Any bleeding that began after 72 hours of last injection at that location was considered as a new event. ABR = total number of qualifying BE/number of days in the respective period \*365.25.
| Arm | Type | Description |
|---|---|---|
| Fitusiran-naïve arm | EXPERIMENTAL | Participants will go through a 24-week standard of care (SOC) period before receiving a selected dose of fitusiran at regular interval. If a fitusiran dose adjustment is needed during the study, participants will follow a specific dosing regimen as per study protocol. |
| EFC15647 roll-over arm | EXPERIMENTAL | Participants will continue receiving their current fitusiran dose from EFC15467. If a fitusiran dose adjustment is needed during the study, participants will follow a specific dosing regimen as per study protocol. |
| All participants | EXPERIMENTAL | In standard of care (SOC) period, participants will receive on-demand or prophylactic treatment with clotting factor concentrates (CFCs) or bypassing agents (BPAs) for 6 months (from Day -168 to Day -1). In fitusiran treatment period, participants will receive subcutaneous fitusiran prophylaxis once every other month (Q2M) or once monthly (QM) for 36 months (from Day 1 to Day 1009). In case of bleeding events participants will receive IV CFCs or BPAs. Participants may receive Antithrombin concentrate (ATIIIC) as rescue medicine. . |
| Fitusiran | EXPERIMENTAL | Participants will receive a selected dose of fitusiran at regular intervals, as per study protocol |
| Name | Type | Description |
|---|---|---|
| Fitusiran | DRUG | Pharmaceutical form: solution for injection in PBS Route of administration: subcutaneous |
| Clotting factor concentrates (CFC) or bypassing agents (BPA) | BIOLOGICAL | Pharmaceutical form: solution for injection Route of administration: intravenous injection |
| Antithrombin concentrate (ATIIIC) | BIOLOGICAL | Pharmaceutical form: solution for injection Route of administration: intravenous injection |
| BPA prophylaxis | DRUG | Pharmaceutical form: solution for injection Route of administration: Intravenous |
| Factor (FVIII or FIX) prophylaxis | DRUG | Pharmaceutical form: solution for injection Route of administration: Intravenous |
Inclusion Criteria: Participants not previously exposed to fitusiran are eligible to be included in the study only if all of the following criteria apply: * Participant must be 1 to \<12 years of age at the time of enrollment. * Participants must have severe hemophilia A or B (FVIII \<1% or FIX ≤2...