Thrombotic microangiopathy

This study tests a drug called iptacopan in 30 people who have a serious blood vessel problem after a stem cell transplant. The drug aims to block a part of the immune system that damages blood vessels. Researchers will check if it helps patients live longer and recover organ fun…

This trial tests the drug eculizumab in adults who develop a severe blood clotting disorder called high-risk transplant-associated thrombotic microangiopathy (TA-TMA) after a stem cell transplant. The condition can cause multiple organ failure and has a poor outlook. The study ai…

This study looks at the long-term safety and effectiveness of the drug iptacopan in people with a rare blood disorder called atypical hemolytic uremic syndrome (aHUS). Participants must have completed a previous iptacopan study and still benefit from the drug. The goal is to moni…

This study is for adults with a rare blood disorder called aHUS who are currently receiving antibody infusions. Researchers want to see if switching to a daily pill called iptacopan is safe and works just as well to prevent dangerous blood clots. About 50 participants will take i…

This study tests a new treatment using a patient's own immune cells, modified to target and attack faulty immune cells that cause autoimmune diseases. It is for people with severe, hard-to-treat conditions like lupus. The goal is to see if this therapy is safe and can control the…

This study tests whether a simpler treatment for immune thrombotic thrombocytopenic purpura (iTTP) works as well as the standard approach. Instead of daily plasma exchange (an invasive procedure), patients receive plasma infusions, immune-suppressing drugs, and caplacizumab. The …

This study is testing a new blood test (the HMEC test) to see if it can better identify which patients with thrombotic microangiopathy (TMA) have a specific immune system problem driving their disease. TMA is a severe condition that damages kidneys and the brain. The researchers …

This study follows 2,000 children who receive stem cell transplants to better understand and detect lung injuries that can occur after the procedure. Researchers will track participants for two years, collecting samples and health data to find ways to diagnose problems earlier. T…

This study observes 80 transgender individuals starting cross-sex hormone therapy (testosterone or estrogen) to see how it changes eating behavior, metabolism, weight, and heart health. Participants are followed over time with no extra treatments or procedures. The goal is to und…

This study reviews medical records from over 1,200 people with immune thrombotic thrombocytopenic purpura (iTTP), a rare and life-threatening blood disorder. The goal is to understand long-term health problems, such as heart disease and thinking issues, that survivors face. By an…

This observational study follows 3,000 people with atypical hemolytic-uremic syndrome (aHUS), a rare and serious kidney condition. Researchers will collect safety and effectiveness data on patients treated with eculizumab or ravulizumab, as well as those receiving other care. The…