Atypical hemolytic uremic syndrome with complement gene abnormality

This phase 3 trial is testing a new medicine called Newnorm, a concentrated antibody solution given as a shot under the skin, in 50 people with primary immunodeficiency (a condition where the immune system doesn't work properly). The goal is to see if Newnorm can prevent serious …

This study tests a medicine called Kedrion IVIG 10% in 30 children with primary immunodeficiency, a condition where the immune system doesn't work properly. The treatment is made from donated antibodies and is given through a vein to help prevent serious bacterial infections. Res…

This study looks at whether giving Cutaquig® (a medicine that helps fight infections) by a faster manual push method is safe and easy for adults with primary or secondary immunodeficiency. About 36 participants will use the rapid push method at home. The goal is to see if this ap…

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 tests the long-term safety of TAK-881, a subcutaneous immunoglobulin treatment, in 53 adults and children with primary immunodeficiency diseases (PIDD) who completed a prior study. Participants receive regular infusions every 2-4 weeks and are monitored for side effect…

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 collects small skin samples from people with primary immunodeficiency or immune regulation disorders, as well as from healthy volunteers. Researchers will use these cells to create cell lines for laboratory research. The goal is to better understand immune diseases and…

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…