HEMOPHILIA

This study tests fitusiran, a monthly injection, in boys aged 1 to under 12 with severe hemophilia A or B who have developed inhibitors (antibodies that block standard treatment). The goal is to find the right dose to safely lower a protein called antithrombin, which helps preven…

This study tests whether a once-weekly medication (efanesoctocog alfa) can improve or prevent joint damage in 37 people with moderate or severe hemophilia A. Participants receive the drug for 12 months, and doctors use ultrasound and MRI to check joint health. The goal is to see …

This phase 3 trial tests fitusiran, an injectable drug, in 91 males aged 12 and older with severe hemophilia A or B, with or without inhibitors. Participants first receive standard care for 6 months, then switch to fitusiran for about 3 years. The goal is to see if fitusiran redu…

This study looks at the long-term safety and effectiveness of a drug called fitusiran in people with hemophilia A or B, including those with inhibitors that make standard treatments less effective. The main goal is to see if fitusiran is safe over time and if it reduces bleeding …

This study tests whether a training method that briefly restricts blood flow to the leg muscles can help reduce knee pain in people with hemophilia. Twelve participants will try both the real blood flow restriction and a sham version to compare effects. The goal is to see if this…

This study surveys 350 adults with hemophilia to better understand chronic pain—its type, location, and impact on daily life. Participants fill out questionnaires about their pain history, medication use, and emotional well-being. The goal is to help doctors recognize and treat p…

This study is observing 500 male patients aged 12 and older with severe hemophilia A or B across China. Researchers are collecting data on treatment patterns, costs, and quality of life to understand the true burden of the disease. The goal is to identify gaps in care and help im…