Hemophilia A is a bleeding disorder caused by low or missing Factor VIII, a clotting protein. About 1 in 5,000 males are born with it. Treatment has shifted from on-demand infusions when bleeding occurs to regular prophylaxis that prevents most bleeds, and newer therapies now last weeks instead of days.
What's actually going on in research
Trials are testing gene therapy that could provide lasting Factor VIII production from a single infusion, extended half-life Factor VIII products requiring fewer infusions, bispecific antibodies that mimic Factor VIII function, and small interfering RNA drugs that rebalance clotting. The goal is reducing treatment burden while preventing joint damage and spontaneous bleeds.
Gene therapy
Several gene therapies deliver a working Factor VIII gene using a viral vector. Some people in trials have maintained near-normal Factor VIII levels for years after a single infusion, though durability varies.
Bispecific antibodies
Emicizumab, approved in 2017, bridges Factor IXa and Factor X to restore clotting without replacing Factor VIII. It's given weekly or monthly by injection under the skin, avoiding IV infusions.
RNA interference
Fitusiran lowers antithrombin, a natural brake on clotting, to rebalance hemostasis. It's given monthly by injection and works regardless of inhibitor status.
What to know before you search
Eligibility typically depends on baseline Factor VIII level, presence of inhibitors, number of recent bleeds, joint damage, and prior treatments.
What types of trials are currently open
- Gene therapy trials — Testing one-time infusions of viral vectors carrying the Factor VIII gene. Studies track how long Factor VIII levels stay elevated and whether bleeds decrease.
- Factor replacement trials — Testing new Factor VIII products with longer half-lives, requiring fewer infusions per week. Many use fusion proteins or modifications that slow breakdown.
- Non-factor therapy trials — Testing drugs like emicizumab and fitusiran that restore clotting balance without replacing Factor VIII. Often studied in people with inhibitors who don't respond to Factor VIII.
- Inhibitor trials — Testing treatments for people who've developed antibodies against Factor VIII. This includes immune tolerance induction and alternative clotting agents.
- Joint health studies — Following people with hemophilia to understand how prophylaxis prevents joint damage and whether physical therapy or other interventions improve outcomes.
Recently added Hemophilia A trials
Study of Surgical Practices in Hemophilia A Patients Treated With Efanesoctocog Alfa (Altuvoct®)
Hemophilia A is an inherited bleeding disorder caused by the absence or deficiency of coagulation factor VIII. The perioperative management of individuals with hemophilia A involves replacement therapies, typically through bolus or continuous infusions of Factor VIII, to ensure effective hemostatic control during surgery. Efanesoctocog alfa represents a significant advance in treatment. It is a highly engineered, VWF-independent, recombinant FVIII fusion molecule with an ultra-long half-life of 47 hours in adults and 40 hours in children. Efanesoctocog alfa is approved in the U.S. and Germany for adults and children with hemophilia A for multiple purposes: routine prophylaxis to reduce bleeding episodes, on-demand treatment of bleeding episodes, and perioperative management. Despite its approval, the precise optimal use of efanesoctocog alfa in the surgical setting remains underexplored. Further research is essential to define its specific benefits in surgery, thereby enhancing its clinical utility and informing treatment protocols. The objective of this cohort study is to collect clinical data on the surgical management of patients with hemophilia A treated with Altuvoct® in a real-world setting. Data collected will include surgical context (outpatient or inpatient), number of FVIII infusions during the perioperative period, length of hospital stay, postoperative date of return to usual prophylaxis, and factor VIII use. The results will be compared with those obtained using efmoroctocog (Elocta) in the ongoing CHALE study in France. The multicenter design is critical due to the rarity of hemophilia A, the diversity of surgical procedures, and the need to enroll a sufficient number of patients. The management of patients with hemophilia A during and after surgery is inherently multidisciplinary and requires careful coordination and adherence to numerous requirements. Given the variability in practice among centers, this study aims to support secondary harmonization of protocols and minimize intercenter variability. Such efforts are in line with the missions assigned to the National Reference Center for Hemophilia in France, coordinated by Pr Dargaud, and emphasize the importance of optimizing and standardizing care practices. A similar study is currently underway in France with efmoroctocog alfa (Elocta), which has already included over 155 procedures under real-world conditions. Using a similar case report form (CRF) for the present study will enable a direct comparison of surgical outcomes between extended half-life and ultra-extended half-life FVIII treatments, providing deeper insight into their respective roles in perioperative care. Additionally, this approach will highlight the added value of efanesoctocog alfa compared to existing therapies. Another key advantage of this research is the opportunity to compare outcomes in patients receiving combined therapy with efanesoctocog alfa and emicizumab. Since the ongoing CHALE study has already included patients treated with both efmoroctocog and emicizumab, this comparison will further enhance our understanding of combination treatment strategies.
Study of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein (FRSW107) as Prophylactic Treatment.
The indication for this product is to control and prophylaxis in patients with Hemophilia A (congenital Factor VIII deficiency): The Primary Objective： To evaluate the efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) for prophylactic treatment in previously treated patients with severe Hemophilia A. Secondary Objectives： To evaluate the health-related quality of life, pharmacokinetic (PK) profiles, safety and immunogenicity of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) for prophylactic treatment in previously treated subjects with severe Hemophilia A.
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