Could a stem cell transplant tame VEXAS syndrome?

NCT ID NCT05027945

Recruiting now Disease control Sponsor: National Cancer Institute (NCI) Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jul 10, 2026 · Updated 3 times

Summary

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 patients to stop other treatments.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: stem cell transplant
What this could lead to: If it works, this could offer a way to control VEXAS syndrome and reduce or eliminate the need for ongoing steroid therapy.
What could go wrong: This is an early phase II trial with only 54 participants, so results may not apply to everyone. The transplant carries risks like graft failure, infection, and side effects from the conditioning drugs.

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Conditions

The condition(s) this trial relates to.

autoimmune disease immunodeficiency disease inborn error of immunity Myelodysplastic Syndromes VEXAS syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HEMATOPOIETIC STEM CELL TRANSPLANTATION IMMUNODEFICIENCY

Contacts and locations

Locations

National Institutes of Health Clinical Center
RECRUITING

Bethesda, Maryland, 20892, United States

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