Csl Behring

This study tested a drug called Garadacimab in 22 children aged 2 to 11 with hereditary angioedema, a condition that causes sudden, painful swelling. The drug is given as a shot under the skin to prevent attacks. The study looked at safety and how well it works. It has already be…

This study tested the long-term safety and effectiveness of garadacimab, a monthly injection, for preventing attacks of hereditary angioedema (HAE) — a rare condition causing severe swelling. 171 people aged 12 and older who had frequent HAE attacks took part. The goal was to see…

This study follows 9 adults with severe or moderately severe hemophilia B who received a one-time gene therapy (AAV5-hFIX) to help their bodies produce clotting factor IX. Researchers are checking if the treatment remains safe and effective for up to 10 years after the initial do…

This study tested whether a drug called alpha-1 antitrypsin (AAT) can prevent graft-versus-host disease (GVHD) in people receiving stem cell transplants for blood cancers. GVHD occurs when donor immune cells attack the patient's body. The trial enrolled 222 participants and compa…

This phase 3 trial tested a one-time gene therapy called AMT-061 in 67 men with severe or moderately severe hemophilia B. The therapy uses a harmless virus to deliver a working copy of the gene for clotting factor IX, so the body can produce its own. The main goal was to see if i…

This early-stage study tested the safety of a new drug called CSL040 in 62 healthy adults. The goal was to see if the drug is safe and how the body processes it. This is the first time CSL040 has been given to humans, and it is being developed for diseases where the immune system…

This completed Phase 1 trial studied 28 healthy adults to see how food changes the absorption of a new prolonged-release version of vamifeport, an experimental drug. Participants took a single dose of the drug either with or without food, and researchers measured drug levels in t…