HEMOPHILIA A

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 medicine called NXT007 in people with severe or moderate hemophilia A, a bleeding disorder. The goal is to see if it is safe and helps control bleeding. The trial includes adults, teens, and children, both with and without inhibitors that make standard trea…

This study follows about 100 people with moderate to severe hemophilia A in Taiwan who are starting a preventive treatment called efanesoctocog alfa. Researchers will track joint health, bleeding episodes, and safety over up to 5 years using routine clinic visits and medical reco…

This study tests a one-time gene therapy (BBM-H803) for adults with severe hemophilia A. The treatment uses a harmless virus to deliver a working gene so the body can produce its own clotting factor VIII, potentially reducing or stopping the need for regular factor infusions. The…

This study follows 250 people with hemophilia A who are taking efanesoctocog alfa to prevent bleeding. The goal is to see how well the drug protects joints from bleeds over the long term in everyday life. Participants will have their joint bleed rates tracked for several years.

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 looks at whether it is safe for males aged 12 and older with severe hemophilia A to switch from their current medicine (emicizumab) to a new one called fitusiran. About 20 participants will be followed for up to 28 months to monitor side effects and blood clotting. The…

This study tests a new gene therapy called SPK-8011QQ in adult men with severe or moderately severe hemophilia A. The main goal is to check if the treatment is safe and tolerable. Only 5 participants will be enrolled, and the study is currently recruiting.

This study tests a new medicine called NXT007 to see if it works better than the standard factor VIII treatment at preventing bleeding episodes in people with hemophilia A who do not have inhibitors. About 126 participants aged 12 and older with severe or moderate hemophilia A wi…

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 looks at whether a low, personalized dose of aPCC (FEIBA) can safely help blood clot in children and adults with hemophilia A who have inhibitors and are already taking emicizumab. Researchers will measure how well the blood clots after the dose and track any serious s…

This study tests a single-dose gene therapy called GS1191-0445 for adults with hemophilia A, a bleeding disorder. The therapy uses a harmless virus to deliver a working gene to the liver, helping the body produce its own clotting factor. Researchers will measure how well it reduc…

This early-stage trial tests a gene therapy that delivers a working copy of the clotting factor VIII gene into the blood cells of people with hemophilia A. The goal is to see if it is safe and can reduce bleeding episodes. The study involves 10 male participants aged 2 and older …

This study tests a new medicine called NXT007 against an existing drug, emicizumab, to see which better prevents bleeding episodes in people with hemophilia A. The trial includes 360 participants aged 12 and older with severe or moderate hemophilia A, with or without inhibitors. …

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 study tests a medicine called Nuwiq to prevent bleeding during major surgery in women and girls with hemophilia A. About 28 participants will receive the treatment, and doctors will check if it works well. The goal is to manage bleeding, not to cure the condition.

This study looks at whether a newer medication called Emicizumab works better than the standard Factor VIII treatment to prevent bleeding in children and teens with hemophilia A who play sports. Participants must be between 6 and 19 years old, have moderate or severe hemophilia A…

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 follows about 20 Korean people aged 12 and older with hemophilia A who are already taking Jivi as prescribed by their doctor. Researchers will collect information from routine clinic visits to see how safe the drug is in real-world use. No extra tests or visits are req…

This study is a large registry that will follow up to 2,300 people in China with various blood diseases, such as leukemia, multiple myeloma, and hemophilia. Researchers will collect information from medical records to understand how common these diseases are, what treatments peop…

This study follows pregnant women with a severe hemophilia A gene and their babies after birth. The goal is to learn why some mothers have heavy bleeding after delivery and why some children develop antibodies that make hemophilia treatment less effective. By observing 500 mother…

This study looks at one adult with severe hemophilia A who needs long-term blood thinners (anticoagulation). Hemophilia A is a bleeding disorder, so taking blood thinners is risky. The report will share how new clotting factor treatments help balance bleeding and clotting risks. …

This study compares a new clotting factor medicine (efanesoctocog alfa) with two existing ones in adults with severe hemophilia A. About 24 men aged 18-65 will receive a single dose of their current treatment and then a single dose of the new medicine. The main goal is to see how…

This study looks at how joint health, pain, balance, and falls affect the quality of life in adults with severe hemophilia A. Researchers will measure these factors in 36 adults to find which ones most impact daily well-being. The goal is to identify areas that could be improved …

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 watches how doctors treat men with hemophilia A whose bodies have developed antibodies (inhibitors) that block standard clotting factor. Researchers will track different treatment plans, including immune tolerance therapy and newer drugs like emicizumab, to see which w…

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…