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 Study to Assess the Safety of Marstacimab and How Well it Works in People With Mild to Moderate Hemophilia A or B Without Inhibitors
This is an open-label, multicenter study evaluating the efficacy and safety of marstacimab prophylaxis in approximately 95 male and female adolescents and adults aged 12 to \<75 years with mild to moderate hemophilia A or B without inhibitors who have a severe bleeding phenotype. Eligible participants must have baseline factor VIII or factor IX activity levels \>1% to ≤15% and a history of clinically significant bleeding despite prior treatment with either on-demand factor replacement therapy or routine factor prophylaxis. The study will compare bleeding outcomes and other efficacy measures during a historical 12-month period of prescribed factor replacement therapy prior to study entry with outcomes observed during a 12-month treatment phase in which participants receive marstacimab 150 mg administered subcutaneously once weekly. Participants will be enrolled into 2 cohorts based on their treatment regimen before study entry: a prior on-demand treatment cohort and a prior routine prophylaxis treatment cohort. Female participants with severe hemophilia A or B without inhibitors may also be enrolled on a case-by-case basis. Data from these participants will contribute to safety assessments, and separate descriptive analyses of efficacy outcomes will be performed for all female participants. The study will also evaluate the long-term safety and efficacy of marstacimab during a long-term extension (LTE) treatment phase of up to 36 months. Participants who complete the 12-month treatment phase may continue into the LTE phase. Participants may be in the study for approximately 50 months, including a 12-month treatment phase and a long-term extension phase of up to 36 months.
Donate blood and tissue samples to identify hemophilia arthritis markers
Chronic arthropathy is one of the most common complications of haemophilia, affecting up to 90 per cent of patients aged 4 and over in at least one joint. the study aims to assess gene expression in biological samples collected via synovectomy and joint replacement surgery, as well as in peripheral blood, in patients with severe haemophilia A or B; to compare these data with those from patients with osteoarthritis and rheumatoid arthritis; and to correlate the gene expression data with information relating to the clinical and laboratory phenotype, the history of inhibitors, and ultrasound and histological findings.
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