VEXAS syndrome
MONDO:0026777An adult-onset inflammatory disease that affects only males and is caused by somatic, not germline, mutations. The disorder is characterized by adult onset of rheumatologic symptoms at a mean age of 64 years. Features include recurrent fevers, pulmonary and dermatologic inflammatory manifestations, vasculitis, deep vein thrombosis, arthralgias, and ear and nose chondritis. Laboratory studies indicate hematologic abnormalities, including macrocytic anemia, as well as increased levels of acute-phase reactants; about half of patients have positive autoantibodies. Bone marrow biopsy shows degenerative vacuolization restricted to myeloid and erythroid precursor cells, as well as variable hematopoietic dyspoiesis and dysplasias. The condition does not respond to rheumatologic medications and the features may result in premature death.
Also known as: VEXAS, VEXAS syndrome, somatic, vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic syndrome
27 clinical trials for this condition and its sub-types, 18 tagged with VEXAS syndrome itself.
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New drug hope for rare inflammatory VEXAS syndrome
Disease control Recruiting nowThis 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…
Phase 2 • Sponsor: Groupe Francophone des Myelodysplasies • Aim: Disease control
Last updated Aug 12, 2026 00:00 UTC
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Experimental drug pacritinib takes aim at rare inflammatory VEXAS syndrome
Disease control Recruiting nowThis 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…
Phase 1 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated Jul 17, 2026 00:00 UTC
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Could a stem cell transplant tame VEXAS syndrome?
Disease control Recruiting nowThis 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…
Phase 2 • Sponsor: National Cancer Institute (NCI) • Aim: Disease control
Last updated Jul 12, 2026 00:00 UTC
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Genetic hunt for hidden causes of mysterious inflammatory diseases
Knowledge-focused Recruiting nowThis 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 …
Sponsor: NYU Langone Health • Aim: Knowledge-focused
Last updated Jul 10, 2026 00:00 UTC
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New registry aims to unlock secrets of rare VEXAS syndrome
Knowledge-focused Recruiting nowThis 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…
Sponsor: Technische Universität Dresden • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:32 UTC
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New study uses DNA tests to catch blood cancers before they start
Knowledge-focused Recruiting nowThis 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…
Sponsor: Mayo Clinic • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:07 UTC
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Ultrasound technique may reduce surgery complications
Knowledge-focused Recruiting nowThis 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…
Sponsor: University of Crete • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:01 UTC