Swedish Orphan Biovitrum

This study gives people with moderate or severe hemophilia A who finished a previous trial continued access to the medication efanesoctocog alfa until it's commercially available in their country (or until March 2027). About 104 participants aged 6 and older will receive the drug…

This study tests a drug called pacritinib for people with VEXAS syndrome, a rare genetic condition that causes severe inflammation. About 78 adults will receive either one of two doses of pacritinib or a placebo to see if it helps control symptoms. The goal is to find the best do…

This study follows 250 people with hemophilia A who are taking efanesoctocog alfa to prevent bleeding. The goal is to see how well the drug protects joints from bleeds over the long term in everyday life. Participants will have their joint bleed rates tracked for several years.

This study looks at how long a new drug for severe hemophilia A stays active in the body compared to two existing drugs. About 24 adult men will receive a single dose of their current drug, then later a single dose of the new drug. The goal is to measure and compare the drugs' ha…