Stem cell transplant for people with VEXAS syndrome
Treatments studied
Part of Blood & lymphatic, Immune system & allergy clinical trials.
This Phase 2 trial tests whether an allogeneic stem cell transplant (from a donor) can treat VEXAS syndrome. It may help calm the body’s overactive inflammation and improve symptoms in eligible adults.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 75 years old.
- You can get to the NIH hospital (or live within 2 hours) and stay nearby for at least 100 days after transplant, with an adult caregiver if outpatient early on.
- You can have a donor: a closely matched related or unrelated donor (7/8 or 8/8 match) or a haploidentical related donor.
- Your heart, kidneys, liver, and lungs tests are strong enough for transplant (specific lab and breathing test thresholds).
- You have VEXAS syndrome (often confirmed by a UBA1 mutation) with inflammatory symptoms and/or low blood counts, or you have not responded to usual treatments like prednisone.
- If you can become pregnant or are a man, you must use birth control during the study and for at least 1 year after transplant; pregnancy and breastfeeding are not allowed.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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