VEXAS syndrome

This phase 2 trial tests the drug momelotinib in 57 people with VEXAS syndrome, a rare inflammatory condition that may also involve a bone marrow disorder called MDS. Participants receive momelotinib daily, and doctors check if symptoms improve and if steroid doses can be lowered…

This phase 1 trial tests the safety and tolerability of pacritinib, a pill that blocks JAK2 and IRAK1, in people with VEXAS syndrome—a rare, severe autoinflammatory disorder caused by a UBA1 gene mutation. Up to 15 participants will take pacritinib daily for up to 12 cycles, star…

This study tests whether a stem cell transplant from a donor can help people with VEXAS syndrome, a rare autoinflammatory disease. Participants receive donor stem cells after drugs to prepare their bone marrow. The goal is to see if the transplant can reverse symptoms and allow p…

This study tests a drug called pacritinib for people with VEXAS syndrome, a rare genetic disease that causes severe inflammation. About 78 adults will receive either the drug or a placebo to see if it controls symptoms and reduces flare-ups. The goal is to find a safe and effecti…

This study aims to learn more about the genetics and natural history of autoinflammatory diseases, which cause repeated fevers and inflammation. Researchers will study up to 5,000 people, including patients, their relatives, and healthy volunteers. Participants provide blood and …

This study will analyze DNA from up to 20,000 people, including those with immune disorders and their relatives, to find genetic variants linked to conditions like atopy, autoimmunity, and primary immunodeficiency. Researchers will also study how best to return genetic results to…

This study aims to better understand VEXAS syndrome and other autoinflammatory conditions by analyzing participants' DNA from blood, saliva, or skin samples. Researchers hope to discover new genetic variants that cause or contribute to these diseases. The study enrolls people of …

This study is looking at 50 children and adults with rare autoinflammatory diseases—conditions where the immune system causes inflammation without a clear reason. Researchers want to find what leads to a faster diagnosis, what factors are linked to these diseases, and which treat…

This study is building a national registry of 500 adults with VEXAS syndrome, a rare inflammatory disease. Researchers will collect medical information and biological samples during routine care to learn more about how the disease behaves, what treatments work best, and how to di…

This study aims to create a large collection of blood samples and medical information from 800 people with autoimmune or inflammatory skin diseases. Researchers will use these samples to find new biomarkers and better understand what causes these conditions. The goal is to help d…

This study is looking for new genes and biological markers that cause systemic autoinflammatory diseases (SAIDs), a group of rare conditions that cause repeated fevers and inflammation. Researchers will analyze the DNA and blood of 300 patients to find disease-causing mutations a…

This study is looking at 2,000 people who have early warning signs of blood cancers or bone marrow failure. Researchers use a special genetic test (next generation sequencing) to find changes in cancer-related genes. The goal is to better understand who is at risk and to catch th…

This study is testing whether a special ultrasound technique called VExUS can help doctors manage fluids better during chest surgery. The goal is to see if it lowers the chance of lung and kidney problems after surgery. About 230 adults having video-assisted lung surgery will tak…

This study looks at people with autoinflammatory diseases or AA amyloidosis who have genetic changes that are hard to interpret. Researchers want to understand if these changes actually cause the disease. The goal is to improve diagnosis and knowledge, not to test a new treatment…