- active not recruitingPHASE3
A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa
This study is looking at how a new medicine, efanesoctocog alfa, affects physical activity and joint health in people aged 12 and over with severe haemophilia A. Participants will use a special activity tracker and diary.
Austria · Croatia · Czechia - recruitingNA
Liver Biopsy In Haemophilia Gene Therapy
This study looks at how gene therapy works in the liver for people with severe haemophilia A or B. Researchers will take a small liver sample (biopsy) to understand how the treatment affects liver cells and stays effective over time. This helps improve future gene therapies.
United Kingdom