Severe hemophilia B

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 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 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 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 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…