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 resistance to standard treatments. The goal is to see if regular shots of fitusiran can reduce the number of bleeding episodes compar…

This study tests a new drug called STSP-0601 in 40 people with hemophilia A or B who have developed inhibitors (antibodies that block standard treatment). The goal is to see if the drug can effectively stop bleeding episodes within 12 hours. Participants receive multiple doses du…

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 looks at whether a non-invasive light treatment called photobiomodulation can help children with hemophilia who have bleeding in the knee joint. The goal is to see if it reduces pain and swelling and improves how well the knee can move. About 24 boys and girls aged 9 t…

This study is creating a large 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, improve treatments, and guide public health dec…

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). Researchers will collect blood samples from 20 patients before and after the…

This study is a registry that will follow about 100 adults and teens with hemophilia who are already taking marstacimab, a monthly shot under the skin. The main goal is to learn how patients feel about this treatment compared to the standard IV infusions, and to measure how much …

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 over time and what side effects may occur. Researchers collect safety data on approved therapies to understand long-term ris…

This study is a long-term observation of 1,000 patients receiving stem cell transplants for blood diseases at a hospital on the Qinghai-Tibet Plateau. Researchers will collect information on patient health, treatment results, and costs to understand how high altitude affects reco…

This study is creating a large database of people with various blood diseases in China, 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, out…

This study aims to create a reliable lab test to monitor anti-TFPI antibodies, a new type of treatment for hemophilia. Researchers will test blood samples from 11 men with severe hemophilia who are already taking the anti-TFPI drug Marstacimab. The goal is to better measure how w…

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 receiving these drugs as part of their normal care. No new treatments are…