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.
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