Hemophilia B is a bleeding disorder caused by missing or low levels of clotting factor IX. It affects about 1 in 25,000 male births. Treatment traditionally meant regular infusions of factor IX concentrate to prevent bleeding, but gene therapy approved in 2022 and newer long-acting products are changing what's possible.
What's actually going on in research
Trials are testing gene therapies that enable the body to produce its own factor IX, newer factor IX products that last longer between doses, and drugs that rebalance clotting without needing factor replacement. Several gene therapy approaches are in late-stage testing, aiming to reduce or eliminate the need for regular infusions.
Gene therapy
Etranacogene dezaparvovec was FDA-approved in 2022 as the first gene therapy for hemophilia B. Additional gene therapy candidates are in trials, testing whether one-time treatment can maintain protective factor IX levels for years.
Extended half-life products
Newer factor IX products stay active in the body longer, allowing people to infuse once weekly or less instead of multiple times per week. These products make prophylaxis more practical for many people.
Non-replacement therapies
Drugs like emicizumab and fitusiran work by different mechanisms than factor replacement. They're being tested in hemophilia B to see if they can prevent bleeding without needing factor IX infusions.
What to know before you search
Eligibility typically depends on baseline factor IX level, presence of inhibitors (antibodies against factor IX), age, liver health, and prior treatments.
What types of trials are currently open
- Gene therapy trials — Testing one-time treatments that give the body instructions to produce factor IX on its own, potentially reducing or eliminating the need for regular infusions.
- Extended half-life trials — Testing factor IX products engineered to last longer in the body, allowing less frequent dosing while maintaining protection.
- Non-replacement trials — Testing drugs that prevent bleeding through mechanisms other than replacing factor IX, such as rebalancing the clotting system.
- Pediatric studies — Testing treatments specifically in children with hemophilia B, including safety and dosing of gene therapy and long-acting products.
- Registry studies — Following people with hemophilia B over time to track bleeding patterns, joint health, treatment responses, and long-term outcomes with different therapies.
Recently added Hemophilia B trials
Take an injection of a new gene therapy for bleeding disorders
The purpose of this study is to evaluate the safety, tolerability, immunogenicity , PK, and PD of a single dose of SR604 in participants with Hemophilia A or Hemophilia B, with or without inhibitors （Part A）and to evaluate the safety, PK, PD, and efficacy of multiple doses of SR604 in participants with Hemophilia A or Hemophilia B, or Factor VII (FVII) deficiency, with or without inhibitors (Part B and Part C).
Take a new preventive therapy for hemophilia 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 with or without inhibitors, and to assess the pharmacokinetic characteristics of the new and old processes KN057. The participants in Part PK will be randomly assigned to Old process Group or New process Group in a 1:1 ratio. The participants in Old process Group will receive old process KN057 prophylaxis for the first 26 weeks and new process KN057 prophylaxis for the following 26 weeks. The participants in New process Group will receive new process KN057 prophylaxis for both the first 26 weeks and the last 26 weeks. The participants in Part non-PK will be non-randomized and treated with new process KN057 for 52 weeks prophylaxis after enrollment. Priority screening and enrollment of participants who have participated in the KN057-A-301 or KN057-A-302 study.
Find Hemophilia B trials matched specifically to you
Answer 3 quick questions and we'll show you trials that fit your situation.