SEVERE HEMOPHILIA A

This study tests a one-time gene therapy called BAY2599023 for adults with severe hemophilia A. The therapy uses a harmless virus to deliver a working copy of the factor VIII gene, so the body can produce its own clotting factor. The trial involves 11 participants and aims to fin…

This phase 3 trial tests a lab-made clotting factor VIII in 60 people with severe hemophilia A. The treatment is given on-demand for new bleeding episodes or before surgery. The goal is to see if it safely and effectively stops bleeding and supports surgical recovery.

This early-stage trial is testing a single-dose gene therapy called GS1191-0445 in 7 Chinese adults with severe hemophilia A. The therapy uses a harmless virus to deliver a working gene to the liver, so the body can produce its own clotting factor. The main goal is to check safet…

This Phase IV study is testing emicizumab (Hemlibra) in 136 people aged 13 to 70 with moderate or severe hemophilia A who do not have inhibitors. The goal is to see if this drug, given as a shot under the skin, can improve joint health, increase physical activity, and enhance ove…

This phase 3 trial is testing a new form of factor VIII (rVIII-SingleChain) in 60 Chinese men with severe hemophilia A. The goal is to see how well the drug works to prevent and treat bleeding episodes when given 2-3 times per week. Researchers will also check how the body proces…

This study is testing a medicine called emicizumab (Hemlibra) in babies from birth to 12 months old with severe hemophilia A. The goal is to see if a weekly or biweekly shot under the skin can prevent bleeding episodes and protect joints. About 55 infants will receive the drug fo…

This study tests a one-time gene therapy (valoctocogene roxaparvovec) in 6 Japanese men with severe hemophilia A. The goal is to help their bodies produce their own clotting factor, reducing or stopping the need for regular factor VIII infusions. Participants will be monitored fo…

This phase 3 trial tests fitusiran, an injectable drug, in 91 males aged 12 and older with severe hemophilia A or B, with or without inhibitors. Participants first receive standard care for 6 months, then switch to fitusiran for about 3 years. The goal is to see if fitusiran redu…

This study checks the long-term safety and effectiveness of a one-time gene therapy (BMN 270) for people with severe hemophilia A. Participants from earlier BioMarin trials will be followed for many years to see if the treatment continues to work and remains safe. The goal is to …

This study tests a program called FACE-Rare, designed to support family caregivers of children with rare, life-limiting diseases. The program includes three sessions to help families prepare for future medical decisions and improve their quality of life. Researchers will compare …

This study looks at whether measuring the amount of emicizumab in the blood can help predict bleeding risk in people with severe hemophilia A. Researchers will follow 100 adults and children who are already taking emicizumab and track any bleeding events over about a year. The go…

This study is observing 500 male patients aged 12 and older with severe hemophilia A or B across China. Researchers are collecting data on treatment patterns, costs, and quality of life to understand the true burden of the disease. The goal is to identify gaps in care and help im…