Csl Behring

This phase 2 trial tests an experimental drug called vamifeport in 84 adults with HFE-related hereditary hemochromatosis, a genetic condition causing iron buildup. The drug aims to reduce liver iron levels, potentially offering an alternative to regular blood removal. Participant…

This study tests a single-dose gene therapy called CSL222 (Etranacogene dezaparvovec) in 35 adults with severe or moderately severe hemophilia B who have detectable AAV5 antibodies—a group often excluded from gene therapy. The goal is to see if it reduces bleeding episodes and is…

This study tests a drug called CSL300 in people with end-stage kidney disease who are on dialysis and have signs of inflammation. The goal is to see if it can reduce the risk of heart attacks or heart-related death. About 3110 adults will take part, and half will get the drug whi…

This study tests a single-dose gene therapy called CSL222 in 20 teenage boys with severe hemophilia B, a bleeding disorder. The goal is to see if the treatment reduces bleeding episodes and improves quality of life. Participants must already be on regular factor IX replacement th…

This study follows 200 people with hereditary angioedema (HAE) who are starting garadacimab in routine medical care. Researchers will track how well the drug prevents swelling attacks, its safety, and its impact on quality of life over 48 months. The goal is to see if results fro…

This study looks at two different doses of a medicine called Privigen in children aged 2 to 17 with a nerve disease called CIDP. The goal is to see which dose better prevents the disease from getting worse. About 30 children will take part, and the study is currently recruiting.

This study tests a new drug called anumigilimab in 63 adults with sickle cell disease to see if it is safe. Participants get weekly injections for 64 weeks, starting with a low dose that increases to their personal maximum. The goal is to reduce painful crises, but people will st…

This phase 3 trial tests a concentrated clotting factor (4F-PCC) against standard fresh frozen plasma to stop bleeding in 200 adults undergoing complex heart surgery with a heart-lung machine. The goal is to see if the new treatment corrects clotting problems faster and more effe…

This study tests whether a concentrated clotting drug (fibrinogen) can better control major blood loss during surgery for pseudomyxoma peritonei, a rare abdominal cancer. About 90 adults will be randomly assigned to receive either the drug or a standard blood product during their…

This study follows 500 people with hemophilia B who received HEMGENIX gene therapy, plus a comparison group on standard factor IX treatment. Researchers will track bleeding rates and safety over several years to see if the gene therapy provides lasting control. The goal is to und…

This study tests an inhaled medicine called CSL787 in adults with non-cystic fibrosis bronchiectasis, a lung condition that causes frequent infections and breathing problems. The goal is to see if CSL787 can delay the time until a person's next lung flare-up (exacerbation) compar…

This study offers children aged 2 to 11 with hereditary angioedema (HAE) continued access to garadacimab, a lab-made antibody that helps prevent sudden, painful swelling attacks. Participants must have completed a prior study and shown good response with few or no attacks. The go…

This study tests an investigational drug called CSL889 in 70 adults and adolescents with sickle cell disease who are having a painful vaso-occlusive crisis. The goal is to see if the drug can safely shorten the crisis and reduce the need for strong pain medicines. Participants wi…