6 completed, 2 recruiting, 1 active not recruiting. 7 Phase 3, 2 Phase 2, 1 Phase 1, 1 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 7 | Completed 5, Active not recruiting 1, Recruiting 1 |
| Phase 2 | 2 | Completed 2 |
| Phase 1 | 1 | Completed 1 |
| Phase 4 | 1 | Recruiting 1 |
Follow fitusiran: new trials by email Literature Review of fitusiran trials Research Report on fitusiran
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| 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 Fitusiran, Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC) - Hemophilia | Phase 3 | Recruiting | Sanofi | 85 | 2025-12-18 | 2026-10-27 |
| A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab Fitusiran (SAR439774), Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC) - Hemophilia A | Phase 4 | Recruiting | Sanofi | 20 | 2024-03-01 | 2026-09-23 |
| A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia Fitusiran, Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC) - Hemophilia | Phase 3 | Active not recruiting | Sanofi | 91 | 2023-02-01 | 2026-02-04 |
| Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B Fitusiran - Hemophilia | Phase 2 / Phase 3 | Completed | Genzyme, a Sanofi Company | 32 | 2020-01-28 | 2026-09-28 |
| 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 Fitusiran - Hemophilia | Phase 3 | Completed | Genzyme, a Sanofi Company | 281 | 2019-01-09 | 2026-09-30 |
| A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis Fitusiran, BPA prophylaxis, Factor (FVIII or FIX) prophylaxis - Hemophilia | Phase 3 | Completed | Genzyme, a Sanofi Company | 80 | 2018-07-25 | 2023-02-06 |
| A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors fitusiran, factor concentrates - Hemophilia A, Hemophilia B | Phase 3 | Completed | Genzyme, a Sanofi Company | 120 | 2018-03-01 | 2022-03-28 |
| A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors fitusiran, Bypassing agents - Hemophilia A, Hemophilia B | Phase 3 | Completed | Genzyme, a Sanofi Company | 60 | 2018-02-14 | 2022-03-28 |
| An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B Fitusiran (SAR439774) - Hemophilia A, Hemophilia B | Phase 1 / Phase 2 | Completed | Genzyme, a Sanofi Company | 34 | 2015-09-18 | 2024-06-05 |
Hemophilia A (9) · Hemophilia B (3) · Blood Coagulation Disorders (2) · Blood Coagulation Disorders, Inherited (2) · Coagulation Protein Disorders (2) · Genetic Diseases, Inborn (2) · Genetic Diseases, X-Linked (2) · Hematologic Diseases (2) · Hemorrhagic Disorders (2)
Genzyme, a Sanofi Company (6) · Sanofi (3)
United States (9) · Italy (7) · Turkey (Türkiye) (7) · China (6) · India (6) · Taiwan (6) · Canada (5) · France (5) · Japan (5) · South Korea (5) · Spain (5) · United Kingdom (5) · Australia (4) · Germany (4) · Malaysia (4)
PMID 34922648, PMID 35114155, PMID 37003278, PMID 37003287, PMID 38452197, PMID 39642315, PMID 40053895
2016 (3), 2015 (1), 2018 (1), 2019 (1), 2023 (1), 2025 (1)