Complement deficiency

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 study tests a new medicine called crovalimab for people with a rare disease called atypical hemolytic uremic syndrome (aHUS), which causes blood clots and kidney damage. The trial includes 83 adults and teens who receive the drug to see if it can control the disease and impr…

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 phase 3 trial tests crovalimab in 41 children with atypical hemolytic uremic syndrome (aHUS), a rare disease that causes blood clots and kidney damage. The drug is given first through a vein, then as a shot under the skin every four weeks. The goal is to see if it can stop d…

This study tests a pill called deucrictibant for quickly treating swelling attacks in people with hereditary angioedema (HAE), including life-threatening throat swelling. About 150 adults who have had HAE attacks before will take the drug as needed and be monitored for safety and…

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 observes 367 Chinese patients with atypical hemolytic uremic syndrome (aHUS), a rare condition that causes blood clots and kidney damage. Researchers will track how patients are treated in routine care and monitor their health for up to 12-24 months. The goal is to bet…

This study aims to develop better lab tests to diagnose and monitor two rare kidney diseases: atypical hemolytic uremic syndrome (aHUS) and C3 glomerulopathy (C3G). Researchers will analyze blood, cells, and kidney tissue from 180 patients and healthy volunteers to find markers t…

This study looks at how the complement system, a part of the immune system, may contribute to kidney damage in people with diabetes. Researchers will measure complement proteins in blood and urine from 90 participants to see if they are linked to kidney disease severity and blood…