Hemophagocytic syndrome

This phase 3 trial tests a drug called plonmarlimab in 30 adults with a severe immune overreaction (macrophage activation syndrome) linked to rheumatic diseases that hasn't responded to prior treatment. Participants receive weekly IV infusions for 8 weeks. The main goal is to see…

This study tests a bone marrow transplant from a family donor for children under 21 with severe non-cancer blood disorders like sickle cell disease, bone marrow failure, or immune problems. The goal is to see if the transplant can replace the diseased cells with healthy donor cel…

This study tests a new drug, ELA026, for people with secondary hemophagocytic lymphohistiocytosis (sHLH), a rare and life-threatening condition where the immune system becomes dangerously overactive. The drug aims to calm this overreaction by removing certain immune cells. The tr…

This study tests a drug called ruxolitinib as the first treatment for children with a rare, life-threatening immune condition called primary HLH. The goal is to calm the overactive immune system and help more children survive long enough to receive a stem cell transplant. The tri…

This study tests whether adding memory immune cells (CD45RO) to a stem cell transplant can help children fight off dangerous viral and fungal infections after the procedure. The transplant uses donor stem cells that have been stripped of certain cells to prevent graft-versus-host…

This phase 2 trial is testing a combination of ruxolitinib and a reduced-intensity version of the standard HLH-94 regimen (dexamethasone and etoposide) in 36 adults newly diagnosed with hemophagocytic lymphohistiocytosis (HLH), a rare condition where the immune system attacks the…

This trial tests a new combination of drugs (MEPL-G) for people with a rare and aggressive lymphoma (NK/T-cell) that has triggered a life-threatening immune overreaction called HLH. The goal is to control both the cancer and the dangerous inflammation quickly. About 25 adults wil…

This study tests whether the drug letermovir can prevent serious viral infections in children with EBV-related blood disorders who receive a stem cell transplant. About 80 children will be enrolled, and researchers will monitor for infections and other transplant complications. T…

This study tests a cord blood transplant in children and young adults (up to age 21) with life-threatening blood cancers or non-cancer blood disorders who do not have a matched family donor. The goal is to see if this approach lowers the risk of death from treatment one year afte…

This phase 2 trial tests a combination of three chemotherapy drugs (treosulfan, fludarabine, thiotepa) plus an immune-suppressing antibody before a donor stem cell transplant in up to 40 people under 50 with non-cancerous blood disorders. The goal is to see if this milder conditi…

This early-phase study tests whether an investigational drug called Tadekinig alfa can safely and feasibly treat severe immune overreactions (CRS and HLH-like syndrome) that can occur after CAR T cell therapy. Ten adult participants who are already in a CAR T cell trial will rece…

This study tests whether a transplant of donated umbilical cord blood can help people aged 14 to 80 with serious diseases caused by the Epstein-Barr virus (EBV), such as certain lymphomas or chronic active EBV infection. The goal is to control the disease and improve survival, bu…

This study tests whether combining two drugs, golidocitinib and selinexor, can control chronic active Epstein-Barr virus disease (CAEBVD). Researchers will enroll 28 adults with CAEBVD and track how well the treatment reduces symptoms and lowers virus levels in the blood. It is a…

This study tests a drug called golidocitinib for people with hemophagocytic lymphohistiocytosis (HLH), a rare condition where the immune system attacks the body. The trial will include 28 people aged 14 to 70 with newly diagnosed or returning HLH. The goal is to see if the drug c…

This study tracks 50 children and adults with non-malignant disorders like immune deficiencies and anemias who receive a stem cell transplant after a reduced-intensity chemotherapy regimen. The goal is to see if this approach improves survival and reduces severe graft-versus-host…

This study is testing a less intense chemotherapy and radiation regimen before a stem cell transplant for children and young adults up to age 55 with non-cancerous blood disorders like immune deficiencies, anemias, and metabolic diseases. The goal is to see if this gentler prepar…

This study tests a combination of two drugs, zanubrutinib and rituximab, for people with B-cell lymphoma who develop a severe immune overreaction called hemophagocytic lymphohistiocytosis (HLH). The trial will enroll 40 participants and aims to see how well the treatment controls…

This study is looking for a better way to diagnose three serious conditions that can look alike: Still's disease, sepsis, and macrophage activation syndrome. Researchers will measure a specific form of a blood protein called glycosylated ferritin in 60 people suspected of having …

This study tests a new type of ultrasound called super-resolution ultrasound (SRUS) to see if it can better tell the difference between benign (non-cancerous) and malignant (cancerous) swollen lymph nodes. About 779 adults with abnormal lymph nodes will receive an injection of ul…

This study aims to learn more about how diseases related to blood clots, the immune system, and blood vessels start and change over time. Researchers will enroll up to 1,000 people aged 5 and older, including those with these conditions, their healthy relatives, and healthy volun…

This study looks for better ways to detect lung inflammation and infections early in people who have received a stem cell transplant from a donor. About 40 participants aged 5 to 70 will get regular lung scans, breathing tests, blood draws, and a procedure where a tube collects f…

This study follows 6,000 people with histiocytic disorders (like Langerhans cell histiocytosis) to understand long-term health problems, pain, mental health, and causes of death. Researchers compare participants to healthy controls to identify factors that lead to new health issu…

This study aims to collect DNA samples and detailed eye exam data from 1,000 people with rare inherited eye diseases like aniridia, Best disease, and albinism. Participants provide a saliva or blood sample and share their eye health records. The goal is to expand a research repos…

This study aims to learn more about Chediak-Higashi syndrome, a rare genetic disorder that causes light skin and hair, easy bruising, and frequent infections. Researchers will observe up to 60 patients over time, collecting clinical and genetic data to better understand the disea…

This study is following 81 adults with a rare condition called hemophagocytic lymphohistiocytosis (HLH), where the immune system becomes dangerously overactive. Researchers aim to better understand the disease's features and how patients respond to treatments like steroids and ch…

This study is testing a tool called B-COMPASS that uses a 22-question survey to predict whether people will follow their prescribed treatments. Researchers will enroll 3100 adults with cardiovascular, endocrine, immune, nerve, cancer, or rare diseases across Europe. The goal is t…

This study aims to understand why some adults develop secondary hemophagocytic lymphohistiocytosis (sHLH), a serious immune condition causing fevers and organ damage. Researchers will follow 300 participants for up to 3 years, collecting blood samples and medical data to identify…

This study is a registry that collects information from 200 people with hemophagocytic lymphohistiocytosis (HLH), a rare condition where the immune system becomes dangerously overactive. Researchers will track how quickly HLH is diagnosed, what treatments are used, and what compl…

This study is creating a large registry of 800 people receiving biologic medications for immune conditions like autoimmune diseases and primary immune deficiencies. Researchers will collect blood samples and health data to better understand how these treatments work and who respo…

This study is observing 2,000 lymphoma patients who also develop hemophagocytic lymphohistiocytosis (HLH), a severe immune overreaction. Researchers will collect data on which treatments patients receive and how they respond, aiming to identify factors that improve survival at 18…

This study looks at whether a tool called the HScore can accurately diagnose hemophagocytic lymphohistiocytosis (HLH) in intensive care patients with liver failure or cirrhosis. Researchers will review medical records of 100 adults admitted to one hospital over ten years who had …

This study collects information from about 100 adults in Alsace who have had a confirmed case of human anaplasmosis, a bacterial infection spread by ticks. Researchers want to learn more about how common it is, what symptoms people have, and how serious it can be. The goal is to …

This registry is tracking 3,000 critically ill patients who receive the CytoSorb blood-filtering device in real-world intensive care settings. The goal is to collect data on how the device is used and whether it relates to patient survival. It covers many conditions like sepsis, …

This study is collecting blood, urine, and stool samples from 500 children with autoimmune diseases and healthy controls. Researchers will analyze these samples to find molecular patterns that could help diagnose these conditions and guide treatment decisions. The goal is to crea…