OncoMatch/Clinical Trials/NCT05027945
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome
Is NCT05027945 recruiting? Yes, currently enrolling (May 2026). This Phase 2 trial studies multiple treatments for immunodeficiency.
Treatment: Busulfan test dose · Mycophenolate mofetil (MMF) · Tacrolimus · Busulfan · Fludarabine · Cyclophosphamide (CY) · Post-Transplant Cyclophosphamide (PTCY) — 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....
Check if I qualifyExtracted eligibility criteria
Biomarker criteria
Required: UBA1 somatic mutation
Prior therapy
Must have received: standard medical management (corticosteroids and/or steroid sparing medications and biological response modifiers)
Participants who have failed standard medical management (requiring >= 0.5mg/kg per day of prednisone for the above listed inflammatory condition or intolerance or refractory to use of corticosteroids and/or steroid sparing medications as well as biological response modifiers over the last 6 months), or when no standard medical treatment is available.
Cannot have received: investigational agents
Participants who are receiving any other investigational agents within the last 30 days before treatment initiation.
Lab requirements
Blood counts
Presence of cytopenia defined as at least one of: ANC <=1000/µL; platelet count <= 75,000/µL or platelet transfusion dependence (at least 4 platelet transfusions in the 8 weeks prior to study entry); hemoglobin <= 10.0g/dL or red cell transfusion-dependence (at least 4 units of PRBCs in the 8 weeks prior to treatment initiation) or meeting criteria for myeloid neoplasm (MN) by updated 2022 WHO criteria or 2022 International Consensus Classification (ICC) of myeloid neoplasms and acute leukemia
Kidney function
Creatinine <= 2.0 mg/dl and creatinine clearance >= 30 ml/min
Liver function
Serum conjugated bilirubin < 3.0 mg/dl; serum ALT and AST <= 5 times upper limit of normal.
Cardiac function
Left ventricular ejection fraction > 35%, preferably by 2-D echocardiogram (ECHO) obtained within 60 days prior to treatment initiation.
Adequate end-organ function, defined as follow: Left ventricular ejection fraction > 35%, preferably by 2-D echocardiogram (ECHO) obtained within 60 days prior to treatment initiation. Creatinine <= 2.0 mg/dl and creatinine clearance >= 30 ml/min; Serum conjugated bilirubin < 3.0 mg/dl; serum ALT and AST <= 5 times upper limit of normal. Pulmonary function tests: FEV1 and DLCO >30%
Structured fields extracted by AI. May contain errors — verify against the official protocol.
US trial sites
- National Institutes of Health Clinical Center · Bethesda, Maryland
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