Beta-thalassemia intermedia

This study tests whether adding the drug vorinostat to standard care can prevent graft-versus-host disease (GVHD) in children and young adults (ages 1-26) with non-cancerous blood disorders who are getting a bone marrow transplant. GVHD is a serious complication where donor cells…

This Phase 3 trial tests whether the drug mitapivat can improve anemia in children aged 1 to 17 with alpha- or beta-thalassemia who do not need regular blood transfusions. About 45 participants will receive either mitapivat or a placebo for 24 weeks. The main goal is to see if mi…

This study tests a new drug called SNH-119014 in 30 adults with a type of thalassemia that does not require regular blood transfusions. The goal is to see if the drug is safe and helps improve anemia. Participants will be monitored for side effects and how their body responds to …

This early-stage study tests a new treatment using special immune cells (CAR-T cells) designed to reduce harmful antibodies that can prevent or damage bone marrow transplants. About 18 people with blood diseases who have these antibodies will receive the cells in increasing doses…

This study tests whether starting tube feeding right after a stem cell transplant can reduce severe gut graft-versus-host disease (GVHD) in people with blood cancers or disorders. About 112 participants will be randomly assigned to either standard care or early tube feeding. The …

This study looks at how thalassemia patients in Greece feel day-to-day while taking a standard iron-removing medicine called deferasirox. Researchers will survey 150 adults to measure their physical and mental well-being. The goal is to understand real-world quality of life, not …