Takeda

This study tests a new drug called zasocitinib in 20 adults with active Crohn's disease. The goal is to see if it safely reduces gut inflammation at the cellular level. Participants take the drug for 12 weeks and undergo a colonoscopy to check the bowel. This is an early-phase tr…

This study tests an experimental drug called TAK-226 in about 27 Japanese adults with a bone marrow condition called myelodysplastic syndromes (MDS) who need regular blood transfusions. The goal is to see if TAK-226 can help them go without transfusions for at least 8 weeks. Part…

This study looks at how safe and effective Takhzyro is for teenagers and adults with hereditary angioedema (HAE) in everyday medical care in South Korea. HAE is a rare condition that causes sudden swelling under the skin or inside the body. The study will track side effects and h…

This study looks back at medical records of 94 people with a rare inherited blood disorder called cTTP, which causes dangerous blood clots. Participants received a manufactured version of the missing enzyme (rADAMTS13) through an early access program. Researchers want to see if t…

This study looks at whether a single test measuring C1-inhibitor function can accurately diagnose hereditary angioedema (HAE), a rare condition causing sudden swelling. About 514 people in Algeria who are suspected of having HAE or have family members with it will take part. Rese…

This study will look at how doctors in Egypt diagnose and treat people with hereditary angioedema (HAE) types I and II. Researchers will review past medical records and collect new information during regular clinic visits from about 100 participants. The goal is to understand cur…

This survey aims to see how well patients, caregivers, and home infusion nurses understand and use educational materials for VPRIV home treatment for Gaucher disease. About 60 people from European countries will fill out a questionnaire. The results will help improve these materi…

This study looks at the safety of a drug called Adzynma in people with a rare inherited blood disorder (congenital TTP). The main goals are to see how many patients develop allergic reactions or antibodies that stop the drug from working. Researchers will review medical records f…