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 daily injection called concizumab in children under 12 with hemophilia A or B, with or without inhibitors. The goal is to see if it safely prevents bleeding episodes. Participants receive daily shots under the skin for at least 2 years, and the study tracks how…

This study follows 30 people with hemophilia A or B (without inhibitors) in Japan who are already using Alhemo (concizumab) as part of their routine care. Researchers will monitor side effects and how well the drug works over about 2 years per person. The goal is to see if Alhemo…

This study tests a drug called concizumab, given as a daily shot under the skin, to prevent bleeding in people with hemophilia A or B who have inhibitors (antibodies that block standard treatment). About 134 participants either start concizumab right away or continue their usual …

This study tests a medicine called concizumab for people with hemophilia A or B who do not have inhibitors. The goal is to see if a daily shot under the skin can prevent bleeding episodes safely. About 156 participants, aged 12 and older, will either start concizumab right away o…

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 phase 2 trial tests whether the drug itacitinib, given with standard care, can prevent graft-versus-host disease (GVHD) in people who receive a donor stem cell transplant for blood cancers. GVHD happens when the donor cells attack the patient's body. The study includes 31 ad…

This study tracks the safety of Hympavzi (marstacimab) in 50 people with hemophilia A or B who do not have inhibitors. Participants receive weekly injections to help control bleeding. The main goal is to monitor any side effects in real-world use.

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 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 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 Phase 3 trial tests a new drug called KN057, given as a weekly injection, to prevent bleeding in people with hemophilia A or B who do not have inhibitors. The study includes 125 males aged 12 to 70 with severe or moderately severe hemophilia. Participants are split into grou…

This study tests a weekly injection called KN057 to see if it can prevent bleeding episodes in people with hemophilia A or B who have developed inhibitors (antibodies that block standard treatment). About 53 male participants aged 12 to 70 will be randomly assigned to receive KN0…

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 pilot study tests whether a daily mindful music listening program is practical and acceptable for patients hospitalized after an allogeneic stem cell transplant. About 35 adults will listen to music for one hour each day using a web-based platform. The study measures how fea…

This study looks at whether adding art therapy to physical therapy can help children aged 5 to 18 recover emotionally and physically after a stem cell transplant. For two weeks, some children receive both art and physical therapy daily, while others get only physical therapy. Res…

This study evaluates whether artificial intelligence models can accurately interpret ROTEM blood clotting tests and recommend treatments for coagulopathy. Researchers will compare AI decisions to those of a panel of clinical experts using data from adults undergoing elective card…

This study tracks the safety and effectiveness of the drug Alhemo (concizumab) in 23 people with hemophilia A or B in Japan. Participants receive the drug as part of their normal care, and researchers will monitor side effects for about 2 years. The goal is to see how well Alhemo…

This study follows 60 males with haemophilia B who are already using the drug Refixia (nonacog beta pegol) to prevent bleeding. Researchers will collect information on side effects, bleeding episodes, and quality of life over 4 to 9 years. No new treatment is given—participants c…

This study follows 50 young men who had cancer or blood disorders as children and received treatments that can harm fertility. Some stored testicular tissue before treatment; others did not. Researchers will check their fertility through exams, ultrasounds, blood tests, and semen…

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 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…

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…