HEMOPHILIA A

This study is testing a one-time gene therapy injection for men with hemophilia A, a serious bleeding disorder. The therapy aims to help the liver produce the missing clotting factor, which could significantly reduce or prevent bleeding episodes. The trial will follow 50 Chinese …

This study aims to find a safer, personalized low dose of a clotting medication called FEIBA for children and adults with severe hemophilia A who are already taking emicizumab. Researchers want to see if this approach can effectively control bleeding while minimizing risks like b…

This study is comparing how long a new hemophilia A treatment (efanesoctocog alfa) stays active in the bloodstream versus two existing treatments. It involves 24 adult men with severe hemophilia A who are already using one of the comparison treatments. Each participant will recei…

This study aims to evaluate the long-term safety and effectiveness of marstacimab, a preventive injection treatment, for people with severe hemophilia A or B. It includes about 245 participants, both children and adults, who have already been in previous marstacimab studies. The …

This study aims to understand how well a preventive treatment called efanesoctocog alfa protects joint health over time in people with hemophilia A. It will follow about 250 people already prescribed this medication in real-world settings for 2-3 years, tracking their joint bleed…

This study is observing people with moderate to severe hemophilia A in Taiwan who are starting a long-acting preventative clotting factor treatment called efanesoctocog alfa. Researchers will follow up to 100 participants for five years to see how well the treatment protects thei…

This study aims to understand the best ways to treat people with hemophilia A whose immune systems have developed 'inhibitors' that block standard treatments. Researchers will observe 120 male participants for up to 5 years as they receive different combinations of immune toleran…

This study is checking if it's safe for people with severe hemophilia A to switch from their current preventive treatment (emicizumab) to a newer one (fitusiran). It will involve about 20 male participants aged 12 and older who are already on emicizumab. Researchers will closely …

This study is testing a single injection of an experimental gene therapy called BBM-H803 for adults with severe Hemophilia A. The goal is to see if the treatment is safe and if it can help the body produce its own missing clotting factor, which could reduce or prevent bleeding ep…

This study aims to find out which treatment works better to prevent bleeding in children and teens with Hemophilia A who play sports. It compares a newer, less frequent injection (Emicizumab) against the standard intravenous Factor VIII treatment. Researchers will track bleeding …

This is the first human study testing a new drug called SR604. Researchers want to see if it's safe and how it behaves in the body. First, healthy volunteers will receive single doses. Then, people with hemophilia A, hemophilia B, or Factor VII deficiency will receive multiple do…

This study is testing how well a medication called Nuwiq controls bleeding during and after major surgery in women and girls with hemophilia A. It will involve about 28 participants aged 12 and older who are scheduled for surgery. The main goal is to see if Nuwiq successfully pre…

This study monitors the safety of an already-approved hemophilia A treatment called Jivi (damoctocog alfa pegol) in Korean patients. Researchers will collect real-world data from about 20 patients who are already receiving this medication as part of their regular care. The goal i…

This early-stage study is testing a new drug called NXT007 in males with moderate to severe hemophilia A, including those with and without inhibitors. The main goals are to check if the drug is safe, how it moves through the body, and if it helps reduce bleeding episodes. The stu…

This study is evaluating a new, non-invasive blood test to diagnose serious inherited genetic disorders in unborn babies. The test analyzes tiny bits of the baby's DNA found in the mother's blood, aiming to provide a safe and accurate diagnosis as early as 9 weeks into pregnancy.…

This study aims to learn more about hemophilia A by following mother-child pairs from pregnancy through the child's early years. Researchers want to understand why some mothers with severe hemophilia have dangerous bleeding after childbirth and why some children develop antibodie…

This study is observing a single adult patient with severe hemophilia A who is taking long-term blood-thinning medication. Researchers want to understand how to safely manage this combination, as blood thinners increase bleeding risk while hemophilia already causes poor clotting.…

This study aims to gather detailed information on many different blood diseases in China, including cancers like leukemia and lymphoma, and conditions like hemophilia. It will follow 2,300 patients to learn how common these diseases are, what treatments patients receive, how well…

A study to learn about the long-term safety and efficacy of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B respectively, who have received treatment through prior participation in a Pfizer-sponsored clinical trial. Data colle…