HEMOPHILIA B

This early-stage study tests a new drug called SR604 in healthy volunteers and people with hemophilia A, B, or factor VII deficiency. The goal is to check safety and how the drug works in the body. About 31 participants will receive the drug as a shot under the skin.

This study tests a single-dose gene therapy called CSL222 in 20 adolescent boys (ages 12-17) with severe hemophilia B. The treatment aims to help the body produce its own clotting factor, reducing or stopping the need for regular infusions. Researchers will track bleeding rates a…

This study tests a single infusion of BE-101, made from a person's own blood cells that are gene-edited to produce clotting factor IX. It aims to safely raise factor levels and reduce bleeding episodes in 24 adults with moderately severe to severe hemophilia B. Participants must …

This study follows 500 people with hemophilia B who received the gene therapy HEMGENIX to see how well it controls bleeding over time and if any safety issues arise. It also includes a group on standard factor IX treatment for comparison. The goal is to understand the real-world …

This study looks at the long-term safety and effectiveness of a medicine called marstacimab for people with severe hemophilia A or B. It is given as a shot under the skin to help prevent bleeding episodes. The study includes about 245 participants who have already completed earli…

This study tests a new gene therapy called REGV131-LNP1265 for people with severe or moderately severe hemophilia B. The therapy uses CRISPR to insert a working gene so the body can produce its own clotting factor, potentially reducing or eliminating the need for regular factor r…

This study tests a new medicine called marstacimab in children aged 1 to 17 with severe hemophilia A or B. The goal is to see if a weekly shot under the skin can prevent bleeding episodes better than their past standard treatments. Participants receive the drug for 12 months, and…

This study tests a single dose of a gene therapy called ANB-002 in adults with hemophilia B, a bleeding disorder. The goal is to see if it can reduce the number of bleeding episodes compared to standard preventive treatment with clotting factor. About 24 men with severe hemophili…

This study tests a one-time gene therapy called CSL222 for adults with severe or moderately severe hemophilia B who have detectable antibodies that might block treatment. The goal is to see if it reduces bleeding episodes. About 35 participants will receive a single dose and be m…

This study follows people with hemophilia A or B who already received a one-time gene therapy (giroctocogene fitelparvovec or fidanacogene elaparvovec) in earlier Pfizer trials. Researchers will monitor long-term safety, including blood clots, liver issues, and inhibitor developm…

This early-stage trial tests a gene therapy for hemophilia B, a bleeding disorder. Researchers use a modified virus to deliver a working gene into patients' own stem cells, aiming to help their bodies produce the missing clotting factor. The study enrolls 10 males aged 2 and olde…

This study aims to see if a blood test from the mother can accurately diagnose genetic disorders in the fetus, such as sickle cell disease or cystic fibrosis, as early as 9 weeks into pregnancy. The test uses fetal DNA found in the mother's blood, avoiding the miscarriage risk of…

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 looks at 46 men with severe hemophilia who have joint swelling that isn't causing obvious symptoms. Researchers want to find out if this swelling is active (meaning there's ongoing bleeding or inflammation) or inactive. They will use ultrasound, MRI, and physical exams…

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…