HEMOPHILIA B

This study tests fitusiran, a monthly injection, in boys aged 1 to under 12 with severe hemophilia A or B who have developed inhibitors (antibodies that block standard treatment). The goal is to find the right dose to safely lower a protein called antithrombin, which helps preven…

This study tests a one-time gene therapy for adult men with moderate to severe hemophilia B, a bleeding disorder. The therapy delivers a working gene to help the body produce a clotting protein called Factor IX. Researchers will track how many bleeding episodes participants have …

This study tests a gene therapy (PF-06838435) for people with severe hemophilia B, a bleeding disorder. The therapy uses a harmless virus to deliver a working gene for clotting factor IX. Researchers are following 21 participants long-term to see if it is safe and reduces bleedin…

This clinical trial is testing a one-time gene therapy called BBM-H901 for people with hemophilia B, a bleeding disorder caused by low levels of clotting factor IX. The therapy uses a harmless virus to deliver a working copy of the factor IX gene to the liver, so the body can pro…

This study tests a single intravenous dose of a gene therapy called AskBio009 in up to 30 adults with severe hemophilia B. The therapy delivers a working copy of the factor IX gene to help the body produce its own clotting factor. The main goal is to check safety, but researchers…

This phase 3 trial tests a long-acting clotting factor (rIX-FP) in 23 Chinese men with severe hemophilia B. The drug is given regularly to prevent bleeding episodes. Researchers will measure how well it works, how long it lasts in the body, and check for side effects like allergi…

This study looks at the long-term safety and effectiveness of a drug called fitusiran in people with hemophilia A or B, including those with inhibitors that make standard treatments less effective. The main goal is to see if fitusiran is safe over time and if it reduces bleeding …

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study follows 11 males with Hemophilia B who already receive regular factor IX therapy to prevent bleeding. Researchers will collect information on bleeding events, treatment use, and quality of life over at least 6 months. No experimental treatment is given; the goal is to …

This study collects information from doctors and patient groups across Asia and the Pacific to better understand how haemophilia affects people in the region. No treatments or medications are given; instead, researchers will analyze data on how many people have the condition, how…