A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome

Recruiting Phase 2 Interventional Study
Immunodeficiency Hematopoietic Stem Cell Transplantation
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
18 – 75
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
54 (estimated)
Sponsor
National Cancer Institute (NCI) · NIH
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About This Trial
Background: Allogeneic hematopoietic stem cell transplant involves taking blood stem cells from a donor and giving them to a recipient. The transplants are used to treat certain diseases and cancers. Researchers want to see if the transplant can treat VEXAS Syndrome. Objective: To see if stem cell transplants can be successfully performed in people with VEXAS and even improve the disease. Elig…
Trial Locations
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Eligibility Criteria
* INCLUSION CRITERIA: Non-disease related * Age \>= 18-year-old and \<= 75-year-old * Availability of an 8/8 or 7/8 HLA-matched related or unrelated donor, or a haploidentical related donor * Karnofsky performance status of \>= 40% * Adequate end-organ function, defined as follow: 1. Left ventr…
Contacts

Bhavisha A Patel, M.D.

(301) 402-3477

bhavisha.patel@nih.gov

CONTACT