Updated May 2026 · ClinicalTrials.gov
Phase 3 Hemophilia A Trials
16 Phase 3 trials for Hemophilia A, the final stage before a treatment can be submitted for FDA approval.
16 Phase 3 clinical trials for Hemophilia A are registered on ClinicalTrials.gov. Phase 3 is the final stage of testing before a treatment can be submitted for FDA approval, and the trials below come directly from the federal registry. Always talk to your doctor before contacting a study site.
What Phase 3 Means for Hemophilia A
Phase 3 trials are the largest and most expensive stage of clinical research before potential FDA approval. For Hemophilia A, a Phase 3 protocol typically enrolls several hundred to several thousand patients across many medical centers, randomizes participants between the investigational treatment and either a placebo or current standard of care (where ethically appropriate), and tracks them for months or years to confirm that the treatment is both effective and safe in a real-world patient population.
16 Phase 3 trials for Hemophilia A are currently registered. Conditions in this range often have a focused research agenda with a small number of late-stage candidates, frequently targeting a specific subpopulation or stage of the disease.
Pfizer (3), Hoffmann-La Roche (2), CSL Behring (2) lead the Phase 3 Hemophilia A sponsor list. The blend of industry, academic, and government sponsors on a condition's Phase 3 list is a useful signal of how broadly the research community is engaged with the disease.
Phase 3 Hemophilia A Trials on ClinicalTrials.gov
A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of NXT007 prophylaxis compared with emicizumab prophylaxis in people age 12...
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Study B7841007 is an open-label extension study to assess the long-term safety, tolerability, and efficacy of prophylaxis treatment with marstacimab in participants who did not...
Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa
This is a multinational, prospective, open-label, roll-over study in patients with haemophilia A, ≥6 years of age, who have completed participation in any of the parental studies...
Safety of KN057 Prophylaxis in Patients With Haemophilia A or B
The purposes of this open-label, multicenter III clinical trial are to evaluate the safety and efficacy of long-term preventive treatment with KN057 in Haemophilia A or B patients...
A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of NXT007 prophylaxis compared with Factor VIII (FVIII) prophylaxis in...
Efficacy and Safety Evaluation Study of SCT800 in Previously Untreated Hemophilia A Patients.
This is an Open Label, Uncontrolled Study to Evaluate the the Safety and Efficacy of SCT800 for Prophylaxis Treatment in Severe Previously Untreated Hemophilia Patients.
An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients...
The aim of the study is to demonstrate non-inferiority of ANB-002 compared with preventive use of coagulation factor IX (FIX) in adult subjects with hemophilia B with FIX activity...
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or...
This is a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIXco-Padua) in...
Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B...
A study to learn about the long-term safety and efficacy of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B respectively, who...
Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment...
The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B...
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric...
Phase 3 Clinical Project of Pegylated Recombinant Human Coagulation Factor VIII-Fc Fusion Protein
To evaluate the prophylactic efficacy of recombinant human coagulation factor Ⅷ-Fc fusion protein (FRSW117) for injection in patients with severe hemophilia A. To evaluate the...
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12...
This is a parallel, Phase 3, two-arm, open-label study to evaluate the efficacy and safety of treatment with fitusiran prophylaxis administered to male pediatric participants...
To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A
This study is a single-arm, open-label, multicenter study evaluating the efficacy and safety of GS1191-0445 injection as a single dose in Chinese subjects with hemophilia A....
Phase III Clinical Trial of STSP-0601 for Injection in Hemophilia Patients
This study will assess the efficacy of multiple-dose of STSP-0601 for the treatment of bleeding episodes in hemophilia A or B patients with inhibitor
SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis
The purpose of the aPCC-emicizumab safety study is to investigate the hemostatic efficacy as measured by thrombin generation, of a low personalized dose of aPCC (FEIBA) in...
What Participation Looks Like
Phase 3 trials for Hemophilia A typically enroll several hundred to several thousand participants across multiple sites. Participation involves a screening visit to confirm eligibility, randomization to either the investigational treatment or a comparator (often the current standard of care), regular study visits over months or years, and follow-up after the active treatment period. The protocols, time commitments, and visit schedules differ from trial to trial — read the per-trial page for the specifics before discussing participation with your doctor.
Each trial begins with informed consent and a screening visit, where the study team confirms eligibility against the inclusion and exclusion criteria. Randomization assigns participants to either the investigational treatment or a comparator. Standard-of-care portions of the protocol are typically billed to insurance; trial-specific procedures (extra imaging, biopsies, lab draws beyond standard care) are usually covered by the sponsor. Read each trial\'s detailed page for its specific time commitment and visit schedule.
Authoritative Resources for Hemophilia A Trials
Verify any individual trial directly on ClinicalTrials.gov. For the federal context on how Phase 3 results feed into approval decisions, see the FDA drug approval process. For oncology-specific trial resources, the National Cancer Institute publishes patient-oriented overviews. For trials registered outside the U.S., the WHO ICTRP aggregates registries from around the world.
Frequently Asked Questions
What is a Phase 3 Hemophilia A trial?
A Phase 3 trial is the final stage of clinical testing before a treatment can be submitted to the FDA for approval. For Hemophilia A, Phase 3 studies typically enroll hundreds to thousands of patients across multiple medical centers, comparing the new treatment to the current standard of care or a placebo (where ethically appropriate). The goal is to confirm efficacy, monitor side effects in a larger population, and generate the evidence the FDA needs to make an approval decision.
How many Phase 3 Hemophilia A trials are recruiting?
16 Phase 3 trials for Hemophilia A are currently registered on ClinicalTrials.gov. Recruitment status varies by trial — some are actively enrolling, some have closed enrollment but are still in the active treatment phase, and some are completing follow-up. Click any trial below to see its current status, eligibility criteria, and contact information.
Who can participate in a Phase 3 Hemophilia A trial?
Phase 3 eligibility depends entirely on the specific trial protocol. Each trial sets its own inclusion criteria (typically a confirmed diagnosis, certain disease stage or severity, age range) and exclusion criteria (often previous treatments, comorbidities, lab values that fall outside set ranges). The trial pages on this site translate the clinical eligibility criteria into plain English alongside the original text. Whether you fit any specific trial is a medical decision your doctor needs to confirm.
Is participating in a Phase 3 Hemophilia A trial safe?
Phase 3 trials use treatments that have already passed Phase 1 (safety in small groups) and Phase 2 (initial efficacy and side-effect monitoring), so the safety profile is better understood than in earlier-phase studies. That said, side effects can still emerge in larger populations, and the trial protocol may require additional procedures (lab draws, imaging, biopsies) beyond standard care. The informed consent document for any specific trial details the known risks. Discuss those risks with your physician before deciding whether to participate.
Where does this trial data come from?
All trial data is sourced from the ClinicalTrials.gov API v2, the federal registry maintained by the National Library of Medicine at NIH. Under FDAAA 801, most U.S. drug and device trials must register on ClinicalTrials.gov, making it the most comprehensive source of trial information. Sponsors are required to update trial status within 30 days of a change, but delays occur — always confirm the current status with the trial site before traveling for screening.
How This Page Is Built
The trial list is filtered to ClinicalTrials.gov registrations whose phase field includes Phase 3 and whose condition list includes Hemophilia A. Trial counts and the sponsor leaderboard are computed from the same record set. Plain-English eligibility translations on each linked trial page preserve the original clinical text alongside the accessible version. Read the full methodology for the data pipeline and known limitations.
Source: ClinicalTrials.gov API v2, maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData, Phase 3 Hemophilia A list, May 2026. Data: ClinicalTrials.gov."
Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.
Last updated 2026-05-08 · 16 Phase 3 trials tracked for Hemophilia A.
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