HEMOPHILIA B

This study looks at the long-term safety and effectiveness of a medicine called marstacimab for people with severe hemophilia A or B. Participants receive regular injections to help prevent bleeding episodes. The goal is to see if this treatment is safe and reduces bleeds over ti…

This study tests a single-dose gene therapy called CSL222 (Etranacogene dezaparvovec) in 35 adults with severe or moderately severe hemophilia B who have detectable AAV5 antibodies—a group often excluded from gene therapy. The goal is to see if it reduces bleeding episodes and is…

This study tests an experimental injection called SR604 in 76 adults with hemophilia A, B, or congenital factor VII deficiency. The goal is to see if it is safe and can reduce bleeding episodes. Participants will receive either a single dose or multiple doses, and researchers wil…

This study tests a single-dose gene therapy called CSL222 in 20 teenage boys with severe hemophilia B, a bleeding disorder. The goal is to see if the treatment reduces bleeding episodes and improves quality of life. Participants must already be on regular factor IX replacement th…

This study tests an experimental gene insertion therapy called REGV131-LNP1265 for people with severe or moderately severe hemophilia B. The therapy uses CRISPR/Cas9 to add a working copy of the clotting factor IX gene into the body's DNA, aiming to enable long-term production of…

This study tests a new medicine called marstacimab in children aged 1 to 17 with severe hemophilia A or B. The medicine is given as a weekly shot under the skin to prevent bleeding episodes. Researchers will compare how well it works to the children's own past year of standard tr…

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 1 trial tests a gene therapy that uses a lentivirus to deliver a working clotting factor IX gene into patients' own stem cells. Ten males with severe hemophilia B will receive the modified cells. The goal is to see if it's safe and can raise clotting factor levels, pot…

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 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 drug moves through the body. About 31 participants will receive single or multiple doses to find the right…

This study follows 500 people with hemophilia B who received HEMGENIX gene therapy, plus a comparison group on standard factor IX treatment. Researchers will track bleeding rates and safety over several years to see if the gene therapy provides lasting control. The goal is to und…

This phase 3 trial is testing a single dose of ANB-002, a gene therapy that delivers a working copy of the FIX gene, in 24 adults with hemophilia B. The goal is to see if it can reduce bleeding episodes compared to regular preventive factor IX infusions. Participants will be foll…

This study is testing a new blood test that can diagnose single-gene disorders in unborn babies using a sample from the mother. The test looks at fetal DNA found in the mother's blood, which is safer than traditional invasive methods that carry a small risk of miscarriage. Resear…

This study follows 173 people with hemophilia A or B who previously received a single-dose gene therapy in a Pfizer trial. Researchers will track bleeding episodes, clotting factor levels, and any long-term side effects like blood clots or liver issues. The goal is to understand …

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 looks at how well hip, knee, shoulder, and other joint replacement surgeries work in adults with inherited bleeding disorders like hemophilia. Researchers will collect information from medical records and follow patients over time to understand risks, complications, an…

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…