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Home › Conditions › Hemophilia B

Hemophilia B

Part of Blood Coagulation Disorders, Inherited

2 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Hemophilia B

42 trials tracked for Hemophilia B: 24 in phase 3 or 4 and 11 with published results. The most-cited published study has 345 citations.

42Trials tracked
24Phase 3 & 4
0Recruiting
11With published results
Phase distribution
Phase 4 4 Phase 3 20 Phase 2 7 Phase 1 3 Other / NA 8
  1. Phase 3 HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients Completed · 345 cited
  2. Phase 3 A Safety and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B Completed · 211 cited
  3. Phase 3 A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors Completed · 123 cited
  4. Phase 3 A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors Completed · 92 cited
  5. Phase 3 A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B Completed · 63 cited
  6. Phase 3 BAX 326 Surgery Study in Hemophilia B Patients Completed · 26 cited
Show 36 more trials
  1. Phase 3 Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B Completed · 26 cited
  2. Phase 3 Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B Completed · 24 cited
  3. Phase 3 BAX 326 Pediatric Study Completed · 9 cited
  4. Phase 4 Safety and Efficacy of Benefix in Patients With Hemophilia B in Usual Care Settings in China Completed · 4 cited
  5. Phase 3 BAX 326 (rFIX) Continuation Study Completed · 4 cited
  6. Phase 4 Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophilia B Completed
  7. Phase 4 RIXUBIS PMS India (RIXUBIS PMS) Completed
  8. Phase 4 Study Evaluating BeneFIX in Patients With Haemophilia B, Previously Treated With Plasma Derived Factor IX Completed
  9. Phase 3 Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen Completed
  10. Phase 3 A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B Completed
  11. Phase 3 Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B Completed
  12. Phase 3 A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%) Completed
  13. Phase 3 Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects Completed
  14. Phase 3 Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B Completed
  15. Phase 3 Pivotal Study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B Patients Completed
  16. Phase 3 Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B Completed
  17. Phase 3 Efficacy and Safety Study of Prophylactic Versus On-Demand Treatment With Feiba NF in Subjects With Hemophilia A or B and a High Titer Inhibitor Completed
  18. Phase 3 Study Comparing On-Demand Treatment With Two Prophylaxis Regimens Of BeneFIX In Patients With Severe Hemophilia B Completed
  19. Phase 2 A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B Completed
  20. Phase 2 Study of Next-Generation Recombinant Factor IX Variant in Adult Subjects With Hemophilia B Completed
  21. Phase 2 Dose Confirmation Trial of AAV5-hFIXco-Padua Completed
  22. Phase 2 An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B Completed
  23. Phase 2 A Gene Therapy Study for Hemophilia B Completed
  24. Phase 2 Efficacy and Safety of AlphaNine Versus BeneFIX in Patients With Severe Hereditary Haemophilia B Completed
  25. Phase 2 Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B Completed
  26. Phase 1 An Open-Label, Single Dose Pharmacokinetic Study of Benefix (Recombinant Factor IX) in Male Chinese Subjects With Hemophilia B Completed
  27. Phase 1 Dose-escalation Study to Investigate the Safety, PK, and PD of ISU304/CB2679d in Hemophilia B Patients Completed
  28. Phase 1 Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia Completed
  29. N/A Post Marketing Surveillance To Observe Safety and Efficacy Of BeneFIX In Patients With Hemophilia B Completed
  30. N/A Treatment Patterns And Outcomes In Patients Treated With Benefix Or Refacto/Refacto AF - A Swedish Cohort Study Completed
  31. N/A RIXUBIS Drug Use-Result Survey (Japan) Completed
  32. N/A Post Marketing Observational Study of Reformulated BeneFIX Completed
  33. N/A An Institutional Pilot Study to Investigate Physical Activity Patterns in Boys With Hemophilia Completed
  34. N/A BeneFIX Drug Use Results Survey [All-Case Surveillance] Completed
  35. N/A Study Evaluating Liver Transplantation in Haemophilia Patients in Spain Completed
  36. N/A Registry For Patients Treated With BeneFix In Usual Care Setting In Germany Completed

Showing the 42 most-cited and recently-updated of 42 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Hemophilia B: what the trials found

Several recombinant factor IX and gene therapy interventions have demonstrated efficacy in managing Hemophilia B. Prophylactic treatment with rIX-FP significantly reduced the frequency of spontaneous bleeding events compared to on-demand treatment (p<0.0001) 6. Additionally, a specific Factor VIII inhibitor bypassing activity showed a significant reduction in Annualized Bleeding Rate (ABR) when used for prophylaxis compared to on-demand treatment (p=0.0003) 21.

Gene therapy and long-acting agents have shown clinical promise; one study reported that 96.3% of participants remained free of routine FIX prophylaxis after AMT-061 dosing, with a significant portion achieving zero bleeding episodes over 52 weeks 5. Fitusiran demonstrated a statistically significant reduction in both total ABR and spontaneous ABR compared to the control group across two different trials (p<0.0001) [7, 8].

Standard recombinant factor IX products like BeneFIX and Nonacog alfa are established for treatment; Nonacog alfa showed a significant reduction in annualized bleeding episodes compared to on-demand therapy 20. Other studies confirmed the use of rIX-FP 9, rFIXFc [10, 19], and BAX326 [13, 14] in various clinical contexts. Recombinant Factor VIIa BI also showed high rates of treatment success and sustained bleeding control for acute episodes 18.

For the clinician treating this condition

  • Prophylactic regimens with rIX-FP or specific Factor VIII inhibitors significantly reduce the annual rate of spontaneous bleeding compared to on-demand treatments [6, 21].
  • fitusiran demonstrated a significant reduction in both total and spontaneous annualized bleeding rates (ABR) [7, 8].
  • Gene therapy (AMT-061) showed high rates of patients remaining off routine prophylaxis with sustained periods of zero bleeding episodes 5.

AI synthesis of 6 cited trials, updated Jun 27, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Meta-analysis finds non-clotting factor therapies reduce bleeding in congenital hemophilia
Hematology Meta-analysis
Meta-analysis finds non-clotting factor therapies reduce bleeding in congenital hemophilia Can new therapies help people with hemophilia bleed less and live better?
This meta-analysis of 6 RCTs (397 participants) synthesizes evidence on non-clotting factor therapies for bleed prevention in congenital hem…
For people with hemophilia, frequent bleeding can disrupt daily life and cause pain.
Apr 16, 2026
Concizumab Reduces Bleeding in Hemophilia with Inhibitors: Median ABR 0.8 at 56 Weeks
Hematology RCT
Concizumab Reduces Bleeding in Hemophilia with Inhibitors: Median ABR 0.8 at 56 Weeks Could a New Treatment Change the Lives of People with Hemophilia?
In the phase 3 explorer7 RCT, concizumab prophylaxis significantly reduced median annualized bleeding rate (ABR) to 0.8 in patients with hem…
A new treatment for hemophilia helped patients with inhibitors experience just one bleeding episode a year, offering hope for fewer interrup…
Mar 27, 2026

Research across Blood Coagulation Disorders, Inherited

Related studies from across the Blood Coagulation Disorders, Inherited family.

Gene therapy reduces bleeding rates and factor use in adults with severe hemophilia A
Hematology Meta-analysis
Gene therapy reduces bleeding rates and factor use in adults with severe hemophilia A Can gene therapy reduce bleeding and treatment burden for adults with severe hemophilia A?
A systematic review and meta-analysis of adults with severe/moderately severe hemophilia A found gene therapy reduced annualized bleeding ra…
For adults with severe hemophilia A, gene therapy may offer a way to bleed less and rely far less on regular infusions.
Apr 4, 2026
Phase 3 trial of damoctocog alfa pegol in previously treated children with severe haemophilia A
Hematology Phase III
Phase 3 trial of damoctocog alfa pegol in previously treated children with severe haemophilia A Phase 3 trial studies new treatment for severe haemophilia A in children
A phase 3 clinical trial evaluated damoctocog alfa pegol in previously treated children aged 7 to 12 years with severe haemophilia A.
A new treatment for severe haemophilia A in children aged 7 to 12 is being tested in a Phase 3 trial to check its safety and effectiveness.
Apr 2, 2026
Damoctocog alfa pegol shows safety and maintained bleed protection in children with severe haemophilia A
Hematology Phase III
Damoctocog alfa pegol shows safety and maintained bleed protection in children with severe haemophilia A Study finds haemophilia drug safe and effective in children over six months
A phase 3, open-label, single-arm study of 35 previously treated children aged 7 to <12 years with severe haemophilia A found no adverse eve…
A haemophilia drug prevented bleeds in children aged 7 to under 12, with no severe side effects and no one stopping treatment due to bad rea…
Apr 2, 2026