HEMOPHILIA A

This study tests a one-time gene therapy called GS1191-0445 in 50 adults with hemophilia A. The treatment uses a harmless virus to deliver a working gene to the liver, helping the body produce its own clotting factor. The goal is to reduce or prevent bleeding episodes, but patien…

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, helping the body produce its own clotting factor. The goal is to reduce or prevent dangerous bleeding episodes. Researchers will…

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 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 early-phase study tests a gene therapy for hemophilia A, a bleeding disorder. Researchers take a patient's own stem cells, add a working gene for clotting factor VIII, and infuse them back. The goal is to see if this is safe and can reduce bleeding episodes. The trial involv…

This study looks at whether a low, personalized dose of aPCC (FEIBA) is safe and helps control bleeding 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 watch for s…

This study is testing a new gene therapy called SPK-8011QQ in adult men with severe or moderately severe hemophilia A, a bleeding disorder. The main goal is to see if the treatment is safe and tolerable. Only 5 participants will be enrolled, and they will be monitored for side ef…

This study looks at how well a medicine called efanesoctocog alfa works to protect joints and reduce bleeding in people with hemophilia A. About 100 participants in Taiwan will be followed for up to 5 years during their regular clinic visits. The goal is to see if this treatment …

This study tests a medicine called Nuwiq (a clotting factor) in women and girls with mild to moderate hemophilia A who are having major surgery. The goal is to see if Nuwiq can successfully control bleeding during and after the operation. About 28 participants will receive the tr…

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 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 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 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 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 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 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 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 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 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 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 participants using special tests and questionnaires. The goal is to find which factors most impact daily life…

This study looks at how long a new drug for severe hemophilia A stays active in the body compared to two existing drugs. About 24 adult men will receive a single dose of their current drug, then later a single dose of the new drug. The goal is to measure and compare the drugs' ha…

This study reports on one adult with severe hemophilia A who receives long-term anticoagulation (blood thinners). Hemophilia A is a rare bleeding disorder, and blood thinners are usually avoided because they raise bleeding risk. Newer factor VIII therapies may provide enough prot…

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 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 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…