HEMOPHILIA B

This early-stage study tests a new drug called SR604 in healthy people and those with hemophilia A, B, or factor VII deficiency. The main goal is to check safety and how the body processes the drug. About 31 participants will receive injections to find the right dose and see if i…

This study tests a single-dose gene therapy called CSL222 in 20 teenage boys (ages 12 to 17) with severe hemophilia B, a bleeding disorder. The treatment aims to help their bodies produce a missing clotting factor, potentially reducing or stopping the need for regular infusions. …

This study tests an experimental gene therapy called REGV131-LNP1265 for people with severe or moderately severe hemophilia B. The therapy uses CRISPR to insert a working gene into DNA, aiming to teach the body to produce its own clotting factor and reduce the need for regular fa…

This study looks at the long-term safety of a drug called marstacimab for people with severe hemophilia A or B. It is for those who already completed an earlier study. The goal is to see if the drug is safe and helps prevent bleeding episodes over time. About 245 participants wil…

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

This early-stage trial tests a gene therapy for hemophilia B, a bleeding disorder. The treatment uses a modified virus to deliver a working gene for clotting factor IX into the patient's own stem cells. The goal is to help the body produce the missing clotting protein, potentiall…

This study tests a single dose of a new gene therapy called ANB-002 in adults with hemophilia B, a bleeding disorder. The goal is to see if it can prevent bleeding episodes as well as or better than regular factor IX injections. The trial involves 24 men aged 18 or older with sev…

This study follows people with hemophilia A or B who already received a one-time gene therapy (giroctocogene fitelparvovec or fidanacogene elaparvovec) in a previous Pfizer trial. Researchers will monitor their health over time to check for side effects and how well the treatment…

This study tests a medicine called marstacimab, given as a weekly shot under the skin, to prevent bleeding in children aged 1 to 17 with severe hemophilia A or B. Participants receive the drug for 12 months, and their bleeding rates are compared to their own history from the year…

This study tracks 500 people with hemophilia B who received HEMGENIX gene therapy to see how well it controls bleeding over time and if any safety issues arise. It also compares them to patients on standard factor IX shots. The goal is to understand if the treatment keeps working…

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 is testing a blood test that can check for genetic disorders in unborn babies without the risks of traditional invasive tests. The test looks at fetal DNA found in the mother's blood as early as 9 weeks into pregnancy. Researchers aim to make this safe, accurate diagno…

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 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 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…