Hemophilia A is a hereditary bleeding disorder caused by deficiency of clotting Factor VIII, leading to painful joint bleeds, muscle bleeds, and potentially life-threatening bleeding with injuries or surgery. Gene therapy trials are bringing the possibility of a one-time cure closer to reality.
What's actually going on in research
Emicizumab, a bispecific antibody that mimics Factor VIII function without being a Factor VIII protein, has transformed prophylaxis by allowing subcutaneous weekly or monthly injections instead of IV infusions. Gene therapy delivering functional Factor VIII genes via viral vectors to liver cells has produced multi-year Factor VIII production in early trials, and one therapy is now approved. Non-factor therapy approaches including fitusiran (an anti-antithrombin agent) are being tested to reduce bleeding independent of the missing factor.
Emicizumab prophylaxis
Emicizumab bridges Factor IX and Factor X to substitute for Factor VIII function, dramatically reducing bleeds with subcutaneous dosing. Trials are testing it in infants, those with inhibitors, and new dosing schedules.
Gene therapy
Viral vector-delivered Factor VIII gene therapy has produced sustained factor levels for years in some patients. Trials are working on durability, consistency across patients, and use in younger individuals.
Non-factor hemostasis therapies
Fitusiran reduces anticoagulant activity to rebalance clotting without replacing the missing factor. Trials are testing it alongside emicizumab and in patients with inhibitors.
What to know before you search
Eligibility depends on hemophilia A severity (Factor VIII level), presence or absence of inhibitors, age, and prior treatment history.
What types of trials are currently open
- Prophylaxis trials — Testing new factor concentrates, extended half-life products, and bispecific antibodies for bleed prevention.
- Gene therapy trials — Evaluating viral vector gene delivery for long-term Factor VIII production and potential cure.
- Inhibitor management trials — Testing immune tolerance induction and non-factor therapies in patients who develop inhibitors.
- Joint health trials — Evaluating physical therapy, early prophylaxis, and surgical options for hemophilic arthropathy.
- Pediatric trials — Testing prophylaxis approaches and gene therapy in infants and young children.
Recently added Hemophilia A trials
Safety of KN057 Prophylaxis in Patients With Haemophilia 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.
Joint Health, Balance and Quality of Life in Adults With Hemophilia A
Hemophilia is a hereditary bleeding disorder characterized by recurrent bleeding episodes, particularly into joints and muscles, leading to chronic musculoskeletal complications. Repeated joint bleeding may result in hemophilic arthropathy, which is associated with progressive joint damage, chronic pain, reduced mobility, and functional limitations. Advances in prophylactic treatment have significantly improved life expectancy in individuals with hemophilia; however, long-term musculoskeletal complications continue to affect daily functioning and overall well-being. Therefore, the evaluation of health-related quality of life (QoL) has become increasingly important in adults with hemophilia, as it reflects the broader impact of the disease beyond clinical severity. Joint health is considered one of the major determinants of physical functioning in people with hemophilia. Hemophilic arthropathy may lead to chronic pain, muscle weakness, reduced range of motion, and impaired physical performance. In addition to structural joint changes, impairments in neuromuscular function, including reduced proprioception, muscle strength, and postural control, may also contribute to activity limitations. Balance impairments may increase the risk of falls, which may be particularly relevant for individuals with hemophilia because fall-related trauma can lead to bleeding episodes and further joint deterioration. Although previous studies have investigated the relationship between joint health, pain, and functional limitations, the combined contribution of joint health, pain severity, dynamic balance performance, and fall history to QoL in adults with hemophilia has not been sufficiently clarified. This prospective cross-sectional study aims to evaluate QoL in adults with hemophilia and to investigate the associations of joint health, pain severity, dynamic balance performance, and fall history with QoL outcomes. The study includes 36 adults diagnosed with severe hemophilia A who are followed at the Adult Hematology Unit of Van Yuzuncu Yil University. Participants aged 18 years and older who met the inclusion criteria were recruited consecutively. Individuals using walking aids, having neurological or psychiatric disorders, chronic inflammatory diseases, recent lower extremity surgery, or regular medication use that could affect balance were excluded. Joint health is assessed using the Hemophilia Joint Health Score (HJHS), a clinician-administered tool evaluating joint impairment in the knees and ankles. Dynamic balance is assessed using a computerized stabilometric platform (Pro-kin Tecnobody Stabilometric Analysis System) with the Limits of Stability (LoS) test, which evaluates the ability to voluntarily control the center of pressure within the base of support. Pain intensity is measured using the Numeric Pain Rating Scale (NPRS), an 11-point scale ranging from 0 to 10. Fall history is determined based on the self-reported number of falls within the previous 12 months. QoL is assessed using the Turkish version of the Haemophilia-Specific Quality of Life Questionnaire for adults (Haem-A-QoL), a validated patient-reported outcome measure evaluating multiple domains of physical, emotional, and social well-being. Higher scores indicate poorer QoL. The findings of this study are expected to improve the understanding of factors associated with QoL in adults with hemophilia and may help identify potentially modifiable clinical targets for rehabilitation interventions.
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