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 evolved from plasma infusions to engineered factor VIII products, and recently to drugs that work through entirely different mechanisms.
What's actually going on in research
Trials are testing gene therapy to provide lasting factor VIII production from a single treatment, non-factor drugs like emicizumab that bypass the need for factor VIII, and next-generation factor products with longer half-lives. Research also focuses on preventing joint damage, improving bleed prevention in people with inhibitors, and developing oral medications.
Gene therapy
Several gene therapies deliver working factor VIII genes into liver cells, aiming to free people from regular infusions. Some participants maintain near-normal clotting for years after a single treatment.
Non-factor therapies
Emicizumab, a biweekly or monthly injection, mimics factor VIII function through a different pathway. It works in people with inhibitors and is being tested as primary prevention in those without inhibitors.
Inhibitor management
About 30% of people with severe hemophilia A develop antibodies against factor VIII. New drugs aim to control bleeding in these patients without triggering immune reactions.
What to know before you search
Eligibility typically depends on hemophilia severity, presence of inhibitors, prior treatments, age, and liver health for gene therapy trials.
What types of trials are currently open
- Gene therapy trials — Testing one-time infusions that deliver a working factor VIII gene. Participants are monitored for years to track clotting factor levels and bleeding rates.
- Non-factor treatment trials — Testing drugs like emicizumab and other approaches that improve clotting without replacing factor VIII directly.
- Extended half-life trials — Testing modified factor VIII products that last longer in the bloodstream, reducing infusion frequency.
- Inhibitor trials — Testing treatments for people who develop antibodies against factor VIII, including immune tolerance therapy and alternative clotting agents.
- Joint health studies — Following people over time to understand how different treatments prevent joint damage from repeated bleeding.
Recently added Hemophilia A trials
A First-in Human Study of ARD001 in Healthy Volunteers and People With Hemophilia A
This study is evaluating ARD001, an investigational medicine being developed for the treatment of hemophilia A. In Phase 1, healthy adult participants will receive a single dose of ARD001 or placebo to assess its safety and tolerability, how it moves through the body, its effects on blood coagulation, and whether the body develops antibodies against it. If the Phase 1 results support further study and the required regulatory and ethics approvals are obtained, Phase 2 will evaluate repeated doses of ARD001 in adults with hemophilia A. Phase 2 is not currently open for enrollment. The study is designed to determine whether ARD001 can be administered with acceptable safety and to identify doses suitable for further clinical development.
Share your surgical experience with a new hemophilia A treatment
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.
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