HEMOPHILIA

This study tests a medicine called fitusiran in boys aged 1 to under 12 with severe hemophilia A or B, including those whose bodies have developed antibodies that block standard treatments. The goal is to see if regular shots of fitusiran can reduce the number of bleeding episode…

This study tests whether injecting bevacizumab directly into a joint can prevent repeated bleeding episodes in people with hemophilia who have chronic joint inflammation. About 25 participants will receive one injection, and researchers will track bleeding rates, joint health, an…

This study tests a clotting medicine called prothrombin complex concentrate (PCC) to stop bleeding in people with hemophilia A who have developed inhibitors (antibodies that block standard treatment). About 30 participants aged 12 to 65 will receive PCC on-demand during bleeding …

This study tests a new medicine called STSP-0601 to see how well it stops bleeding in people with hemophilia A or B who have developed inhibitors (antibodies that block standard treatment). About 40 participants aged 12 to 70 will receive the drug during a bleeding episode. The m…

This study tests whether a non-invasive light treatment called photobiomodulation can reduce pain, swelling, and improve knee movement in children aged 9-14 with hemophilia who have bleeding in the knee joint. The treatment is given alongside standard care. The study will measure…

This study looks at new blood tests to see if they can better measure how well bleeding treatments work in people with hemophilia who have developed inhibitors (antibodies that block standard treatment). About 20 adults will give blood samples before and after receiving bypassing…

This study is a registry that collects information from 1,000 patients with blood diseases who receive stem cell transplants at a hospital on the Qinghai-Tibet Plateau. Researchers will track their health, treatments, and costs over time to learn how high altitude affects recover…

This study is working on a lab test to measure how well a new type of hemophilia medicine (anti-TFPI antibodies) works. Researchers will take blood samples from 11 men with severe hemophilia who are already taking this medicine. The goal is to create a reliable test that doctors …

This study is creating a large database of people in China with various blood diseases, such as leukemia, multiple myeloma, and hemophilia. Researchers will collect information from medical records and follow participants over time to learn about disease patterns, treatments, and…

This study is a registry for 100 adults and adolescents with hemophilia who are already prescribed marstacimab. The main goal is to learn whether patients prefer a simple shot under the skin (subcutaneous) over the usual intravenous injections, and to measure how much the treatme…

This study is creating a huge database of health information from up to 200,000 people with blood disorders like hemophilia, sickle cell disease, and thrombosis. Researchers will use this data to better understand these conditions and improve treatments. Anyone seen at a particip…

This study looks at how certain medicines act in the bodies of children and young adults under 21. Researchers want to find the safest and most effective doses for these age groups. Participants are already taking these drugs as part of their regular care, or have had a recent CO…

This study follows 3,000 people with non-cancer blood disorders (like hemophilia, sickle cell disease, and clotting issues) to see how well treatments work and what side effects occur over time. Researchers will collect safety data, including allergic reactions, infections, and o…