Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Hemophilia B
Open-data reference.
21 US clinical trials · 9 currently recruiting
Active & Recent Trials
An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B
CSL Behring
NCT06008938
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Pfizer
NCT05145127
Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
Pfizer
NCT05568719
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
Regeneron Pharmaceuticals
NCT06379789
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
Pfizer
NCT05611801
Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)
CSL Behring
NCT06003387
A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)
Equilibra Bioscience
NCT06349473
BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B
Be Biopharma
NCT06611436
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B
CSL Behring
NCT07080905
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Pfizer
NCT03861273
Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B
Baxalta now part of Shire
NCT01687608
Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B
Pfizer
NCT03307980
Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B
St. Jude Children's Research Hospital
NCT00979238
Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
Sangamo Therapeutics
NCT04628871
A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy
Regeneron Pharmaceuticals
NCT05568459
Females With Severe or Moderate Hemophilia A or B: an International Multi-center Study
Weill Medical College of Cornell University
NCT00936312
Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy
St. Jude Children's Research Hospital
NCT05044845
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
Genzyme, a Sanofi Company
NCT03417245
AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)
Spur Therapeutics
NCT04272554
Correlation Between Vitamin D Status and Bone Mineral Density in Patients With Hemophilia
Virginia Commonwealth University
NCT01090206
Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
Ultragenyx Pharmaceutical
NCT02971969
Phase Distribution
| Phase | Trial count |
|---|---|
| Early Phase 1 | 5 |
| Phase 2 | 1 |
| Phase 3 | 7 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Hemophilia B Trial Landscape
ClinicalTrials.gov lists 21 US studies indexed under Hemophilia B, and 9 of those are currently open to recruitment — roughly 43% of the total volume on the registry. That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.
The phase distribution for Hemophilia B shows 7 late-stage studies (Phase 3 and Phase 4 combined) alongside 6 earlier-phase entries (Phase 1 through Phase 2). Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.
Top sponsor activity for Hemophilia B is led by Pfizer with 5 indexed trials, alongside 9 other organizations in the top contributor list. The list on this page surfaces up to 21 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation — eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.
Frequently Asked Questions
How many clinical trials are there for Hemophilia B?
PlainTrial tracks 21 US clinical trials for Hemophilia B, of which 9 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Hemophilia B?
Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.
Is this data current?
Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.
Related
Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.
Read our methodology — how this data is sourced, computed, and verified.
Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2024 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.