Hemophilia

This program provides concizumab, a daily injection under the skin, for free to people with congenital hemophilia who need better treatment. Participants will be monitored for safety and how well the medicine works over several years. The program is for those with severe hemophil…

This phase 3 trial is testing a drug called fitusiran in about 85 boys aged 1 to under 12 with severe hemophilia A or B, including those with inhibitors that make standard treatments less effective. The drug is given as a regular shot under the skin to help prevent bleeding episo…

This study tests a drug called foscenvivint in people with liver cirrhosis caused by HIV and hepatitis C co-infection who also have hemophilia. Participants receive the drug once a week for 24 weeks. Researchers will check if the drug improves liver function and is safe to use.

This registry follows 100 adults and teens with hemophilia A or B who are prescribed marstacimab, a once-weekly shot under the skin. The main goals are to see if patients prefer this shot over IV treatments and to measure treatment burden after 6 months. Researchers will also tra…

This Phase 3 study is testing a drug called KN057, given as a weekly injection under the skin, to prevent bleeding in people with hemophilia A or B (with or without inhibitors). The trial will enroll 70 males aged 12 to 65 and will monitor safety and how well the drug works over …

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

This study tests whether a 6-week online rehabilitation program—combining pain education and exercise—works as well as in-person sessions for adults with hemophilia who have chronic joint pain. Participants are randomly assigned to either telerehabilitation or face-to-face care. …

This study tests whether a non-invasive light therapy called photobiomodulation can reduce pain and swelling in the knees of children with hemophilia who have joint bleeding. Twenty-four boys and girls aged 9 to 14 will receive the light treatment alongside standard care. Researc…

This study is collecting blood and other samples from 5,000 children and young adults (under 21) who are already taking certain medications as part of their regular care. The goal is to understand how these drugs move through and affect children's bodies, so doctors can prescribe…

This study is collecting long-term safety and effectiveness data on treatments for various blood disorders, including hemophilia, sickle cell disease, and clotting disorders. It aims to enroll 3000 people of any age with these conditions. Researchers will monitor side effects, co…

This study looks at whether ultrasound and other imaging can find hidden joint changes in people with hemophilia who have no obvious symptoms. Researchers will follow 46 patients for up to 12 weeks, using scans and physical exams to see if these hidden changes are active or inact…

This study is a large registry that collects health information from up to 200,000 people with blood disorders such as hemophilia, thrombosis, and sickle cell disease. Participants are seen at Hemophilia Treatment Centers across the U.S. The goal is to gather real-world data to h…

This study aims to create a reliable lab test to monitor how well anti-TFPI antibodies work in people with severe hemophilia. Researchers will take a single blood sample from 11 patients already on anti-TFPI therapy and test it in the lab. The goal is to better predict and manage…

This study looks at why people with hemophilia (a bleeding disorder) are less likely to die from heart disease. Researchers will use a new, detailed CT scanner to compare heart artery health in 80 men aged 45+ with hemophilia to similar men without it. The goal is to understand h…

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 that tracks patients with blood diseases who receive stem cell transplants at a hospital on the Qinghai-Tibet Plateau. Researchers will collect information on recovery, complications, and costs over time. The goal is to understand how high altitude affect…

This study is testing new blood tests to see if they can better measure how well bleeding treatments work in people with haemophilia who have developed inhibitors (antibodies that block standard treatment). The researchers will take blood samples from 20 patients before and after…