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
53 clinical trials for this condition and its sub-types.
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Hope for rare disease: new drug trial for VEXAS syndrome begins
Disease control Not yet recruitingThis study tests a drug called momelotinib in 136 adults with VEXAS syndrome, a rare inflammatory disease caused by a genetic mutation. The goal is to see if the drug can reduce disease activity, with participants receiving either momelotinib or a placebo. This is a phase 2/3 tri…
Phase: PHASE2, PHASE3 • Sponsor: GlaxoSmithKline • Aim: Disease control
Last updated Jun 27, 2026 13:01 UTC
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Ultrasound-Guided fluid removal could save kidneys in ICU patients
Disease control Not yet recruitingThis study tests whether using a special ultrasound technique (mVExUS) to guide how much fluid is removed during dialysis can improve outcomes for critically ill patients with acute kidney injury. 126 adults in the ICU will be randomly assigned to either standard care or the ultr…
Phase: NA • Sponsor: Chulalongkorn University • Aim: Disease control
Last updated Jun 27, 2026 12:31 UTC
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Urine test could guide heart failure treatment and save lives
Disease control Not yet recruitingThis study tests whether adjusting diuretic doses based on urine sodium levels improves outcomes for people hospitalized with acute heart failure and severe fluid buildup. About 270 participants will be randomly assigned to either standard care or a strategy where diuretic doses …
Phase: NA • Sponsor: University Hospital, Akershus • Aim: Disease control
Last updated Jun 27, 2026 12:25 UTC
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Pausing arthritis drug may boost pneumonia Shot's power
Disease control Not yet recruitingThis study tests whether temporarily stopping methotrexate for 2 weeks after receiving a pneumonia vaccine (PCV20) helps the vaccine work better in adults with autoimmune rheumatic diseases like rheumatoid arthritis or lupus. About 192 participants with stable, low disease activi…
Phase: PHASE4 • Sponsor: University of Sao Paulo General Hospital • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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Ultrasound tool aims to prevent kidney failure in critically ill
Knowledge-focused Not yet recruitingThis study will test whether using a special ultrasound technique called VeXUS to guide fluid treatment can help protect kidney function in 100 ICU patients with acute kidney injury. Researchers will compare outcomes like survival, need for dialysis, and lasting kidney damage. Th…
Phase: NA • Sponsor: University of Alabama at Birmingham • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:29 UTC
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Massive study to uncover hidden link between blood mutations and immune disorders
Knowledge-focused Not yet recruitingThis study will follow 5,000 adults with immune system problems to see if certain blood cell mutations (called clonal hematopoiesis) are linked to their condition. Researchers will track new cases of immune-related diseases over time. No treatments or drugs are tested—this is pur…
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:00 UTC
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Simple ultrasound may predict ventilator weaning failure in ICU patients
Knowledge-focused Not yet recruitingThis study tests whether a special ultrasound technique, called VExUS, can predict which ICU patients will have trouble breathing on their own after being on a ventilator. Researchers will scan the veins of 350 patients just before a breathing trial. The goal is to see if signs o…
Phase: NA • Sponsor: Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:13 UTC
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Can a quick skin scan reveal hidden inflammation in arthritis?
Knowledge-focused Not yet recruitingThis study will measure harmful molecules called AGEs in 300 people with various rheumatic diseases (like rheumatoid arthritis and ankylosing spondylitis) and compare them to healthy volunteers. AGEs build up over time and can cause inflammation. Researchers will use a non-invasi…
Sponsor: Bursa City Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:10 UTC
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New blood tests aim to unlock mysteries of rare inflammatory diseases
Knowledge-focused Not yet recruitingThis study aims to better understand rare autoinflammatory diseases by developing blood tests that measure inflammation markers. Researchers will analyze blood samples from 60 adults and children with these conditions. The goal is to identify specific inflammation pathways, which…
Sponsor: Hospices Civils de Lyon • Aim: Knowledge-focused
Last updated Jun 26, 2026 17:44 UTC