Actively Recruiting
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome
Led by National Cancer Institute (NCI) · Updated on 2026-05-08
54
Participants Needed
1
Research Sites
174 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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. Eligibility: People ages 18-75 who have VEXAS Syndrome that has caused significant health problems and standard treatment either has not worked or is not available. Design: Participants will be screened with: Physical exam Medical review Blood and urine tests Heart and lung function tests Bone marrow biopsy Participants will have a chest x-ray. They will have an imaging scan of the head, chest, abdomen, pelvis, and sinus. They will have a bone density scan. They will have a dental exam and eye exam. They will meet with specialists. They will repeat some screening tests. Participants will be admitted to the NIH hospital. They have a central venous catheter put into a vein in the chest or neck. They will receive drugs to prepare their bone marrow for the transplant. They may have total body irradiation. They will receive the donor stem cells through the catheter. They will get other drugs to prevent complications and infections. After discharge, they must stay in the DC area for 3 months for weekly study visits. Participants will have study visits 30, 60, 100, 180, 210, 240, 300, and 360 days later. After that, they will have yearly visits for 2 years and then be contacted yearly by phone....
CONDITIONS
Official Title
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 18 and 75 years 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% or higher
- Adequate heart function with left ventricular ejection fraction over 35% (tested within 60 days before treatment)
- Kidney function with creatinine 2.0 mg/dl or less and creatinine clearance of 30 ml/min or more
- Liver function with serum conjugated bilirubin less than 3.0 mg/dl and ALT and AST levels no more than 5 times the upper limit of normal
- Lung function with FEV1 and DLCO greater than 30%
- Ability to understand and willing to sign informed consent
- Use of effective contraception for individuals of childbearing potential and men during the study and for one year after transplant
- Willingness to stay at NIH hospital or live within 2 hours of it for at least 100 days after transplant with an adult caregiver if outpatient
- Somatic mutation in UBA1 confirmed by certified lab or clinical history characteristic of VEXAS with at least two relevant symptoms
- Presence of inflammatory symptoms such as fever, fatigue, skin issues, lung problems, musculoskeletal inflammation, or other major organ involvement
- Presence of blood cell deficiencies including low neutrophil, platelet, or hemoglobin counts or transfusion dependence, or diagnosis of myeloid neoplasm
- Failure of standard medical treatments or intolerance to steroids or other medications for the inflammatory condition
You will not qualify if you...
- Hematopoietic cell transplant (HCT) comorbidity index score of 5 or more
- Diagnosis of multiple myeloma (low risk smoldering multiple myeloma or monoclonal gammopathy of unknown significance allowed)
- Use of other investigational agents within 30 days before treatment
- HIV-positive status
- History of allergic reactions to study drugs including steroids, cyclophosphamide, busulfan, tacrolimus, mycophenolate mofetil, or filgrastim
- Pregnancy or breastfeeding during study treatment due to potential risks
- Uncontrolled illnesses or social situations limiting study compliance
- Active uncontrolled infections considered unsafe for transplant
- Active psychiatric disorders that may impair compliance with transplant protocol
AI-Screening
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Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
B
Bhavisha A Patel, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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