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Home › Conditions › Hemophilia A

Hemophilia A

Part of Blood Coagulation Disorders, Inherited

5 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Hemophilia A

106 trials tracked for Hemophilia A: 78 in phase 3 or 4 and 31 with published results. The most-cited published study has 1086 citations.

106Trials tracked
78Phase 3 & 4
0Recruiting
31With published results
Phase distribution
Phase 4 21 Phase 3 57 Phase 2 11 Phase 1 7 Other / NA 10
  1. Phase 3 A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With Inhibitors Completed · 1,086 cited
  2. Phase 3 A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors Completed · 747 cited
  3. Phase 4 Survey of Inhibitors in Plasma-Product Exposed Toddlers Completed · 491 cited
  4. Phase 3 A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors Completed · 389 cited
  5. Phase 3 A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks in Participants With Hemophilia A Completed · 386 cited
  6. Phase 3 Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study) Completed · 257 cited
Show 44 more trials
  1. Phase 3 Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) Completed · 194 cited
  2. Phase 3 A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors Completed · 123 cited
  3. Phase 3 Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A Completed · 115 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 An Open-label Safety, Efficacy and Pharmacokinetic Study of a Recombinant FVIII Compared to Recombinant Human Antihemophilic FVIII in Patients With Severe Hemophilia A Completed · 87 cited
  6. Phase 3 A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors Completed · 84 cited
  7. Phase 3 BAX 855 Pediatric Study Completed · 80 cited
  8. Phase 3 BAX 855 PK-guided Dosing Completed · 78 cited
  9. Phase 3 Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A Completed · 74 cited
  10. Phase 3 BAY81-8973 Pediatric Safety and Efficacy Trial Completed · 40 cited
  11. Phase 3 Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A Completed · 39 cited
  12. Phase 4 Canadian Hemophilia Prophylaxis Study Completed · 38 cited
  13. Phase 3 An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia A Completed · 30 cited
  14. Phase 3 Phase 3 Efficacy and Safety Study of BAX 855 in Severe Hemophilia A Patients Undergoing Surgical Procedures Completed · 28 cited
  15. Phase 3 A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A Completed · 25 cited
  16. Phase 3 Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A Completed · 22 cited
  17. Phase 3 Evaluating the Haemostatic Effect of NNC 0129-0000-1003 During Surgical Procedures in Subjects With Haemophilia A. Completed · 14 cited
  18. Phase 3 A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors Completed · 13 cited
  19. Phase 3 Safety and Efficacy of Turoctocog Alfa (N8) in Prevention and On-demand Treatment of Bleeding Episodes in Subjects With Haemophilia A: An Extension to Trials NN7008-3543, NN7008-3545, NN7008-3600, NN7008-3893 and NN7008-4015 Completed · 9 cited
  20. Phase 3 A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A Completed · 9 cited
  21. Phase 4 PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Hemophilia A Completed · 3 cited
  22. Phase 3 A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A Completed · 3 cited
  23. Phase 4 Study of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies Completed · 1 cited
  24. Phase 4 Prophylaxis Regimen for Hemophilia A Patients Completed
  25. Phase 3 A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood Completed
  26. Phase 4 Study Evaluating Safety And Efficacy Of Moroctocog Alfa (AF-CC) In Previously Treated Hemophilia A Patients Completed
  27. Phase 4 Phase 3/4 Study of a Recombinant Protein-Free Factor VIII (rAHF-PFM): Comparison of Continuous Infusion Versus Intermittent Bolus Infusion in Hemophilia A Subjects Undergoing Major Orthopedic Surgery Completed
  28. Phase 4 Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting Completed
  29. Phase 4 Research Study to Look at Side Effects During Regular Injection With Factor VIII Medicine Named Turoctocog Alfa for a 8 Weeks Period Completed
  30. Phase 4 Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A Patients Completed
  31. Phase 4 Safety Study of Alphanate in Previously Treated Patients With Severe Hemophilia A Completed
  32. Phase 4 Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophilia A (Post-marketing Investigation) Completed
  33. Phase 4 A Study of ADVATE in People With Hemophilia A in India Completed
  34. Phase 4 A Post Approval Commitment Study to Gain More Information on How Safe and Effective KOVALTRY is in Chinese Children, Adolescents /Adults With Severe Hemophilia A Completed
  35. Phase 4 Dose-Response Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A Completed
  36. Phase 4 Pharmacokinetic Study of ADVATE 3000 IU in Previously Treated Patients With Severe Hemophilia A Completed
  37. Phase 4 BAY14-2222 Prophylaxis and Joint Function Improvement (Adults) Completed
  38. Phase 4 Prophylaxis Versus on Demand Treatment for Children With Hemophilia A Completed
  39. Phase 4 Pharmacokinetic Comparison of Advate rAHF-PFM With Recombinate rAHF in Patients With Severe Hemophilia A Completed
  40. Phase 4 Prophylaxis Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A Completed
  41. Phase 4 Russian Kogenate Pediatric Study Completed
  42. Phase 3 Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen Completed
  43. Phase 3 Pharmacokinetics and Safety of rFVIIIFc Manufactured at 15,000 L (15K) Scale Completed
  44. Phase 3 Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A Completed

Showing the 50 most-cited and recently-updated of 106 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 A: what the trials found

Several recombinant Factor VIII (rFVIII) and modified products demonstrate efficacy in managing Hemophilia A. Specifically, rFVIII-FS showed significant reductions in joint bleeds and total bleeds compared to on-demand treatment 21, alongside improvements in physical assessment scores 21. Other recombinant options like Moroctocog-alfa reported 0% inhibitor development in clinical trials [10, 14], while Xyntha demonstrated a very low rate of less than expected therapeutic effect (LETE) in on-demand settings (0.06%) 3.

Prophylaxis regimens with recombinant factors have shown significant impact on bleeding rates and joint health. A study comparing standard prophylaxis to on-demand treatment using an rFVIII product showed a statistically significant reduction in the mean transformed annualized bleed rate (ABR) 18. Additionally, Emicizumab demonstrated significantly lower model-based ABRs for both treated bleeds and all bleeds compared to no prophylaxis or previous episodic bypassing agents 22.

Surgical outcomes were improved with recombinant protein-free Factor VIII; specifically, a study showed significant differences in the volume of packed red blood cells (PRBC) in drainage fluid during surgery when using ADVATE 13. Furthermore, certain recombinant factors like rFVIIIFc were associated with zero days missed from school or work following successful treatment 4.

Immunogenicity and safety profiles vary across products. While some studies noted high rates of clinically significant Factor VIII inhibitors in specific trials 12, others reported 0% inhibitor development for Moroctocog-alfa [10, 14]. One study on plasma-derived VWF/FVIII and rFVIII concentrates identified a frequency of inhibitor development ranging from 29 to 47 across the first 50 episodes or three years 1.

For the clinician treating this condition

  • Prophylactic regimens with recombinant Factor VIII (e.g., rFVIII-FS, Moroctocog-alfa) are associated with significantly lower annualized bleed rates and improved physical assessment scores compared to on-demand therapy.
  • Emicizumab showed a significant reduction in both treated and total annualized bleeding rates compared to no prophylaxis or previous bypassing agents.
  • Recombinant protein-free Factor VIII (rAHF-PFM) demonstrated statistically significant differences in surgical outcomes regarding PRBC volume in drainage fluid 13.
  • Immunogenicity varies by product; while some trials reported high inhibitor rates, others showed 0% development for specific recombinant agents.

AI synthesis of 8 cited trials, updated Jun 25, 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
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
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

Questions about Hemophilia A

Do non-clotting factor therapies work for both Hemophilia A and Hemophilia B?

Non-clotting factor therapies like emicizumab and concizumab are effective for preventing bleeding in both Hemophilia A and Hemophilia B.

Full answer →
How much does gene therapy reduce bleeding rates for adults with severe Hemophilia A?

Gene therapy reduces annualized bleeding rates in adults with severe Hemophilia A by a standardized mean difference of -0.72 compared to standard factor treatment, though bleeding rates can rise again if the treatment wears off.

Full answer →
Do non-clotting factor therapies actually reduce bleeding in congenital Hemophilia A?

Yes, non-clotting factor therapies like emicizumab and concizumab significantly reduce bleeding rates in people with congenital Hemophilia A compared to no treatment or standard clotting factor prophylaxis.

Full answer →