Decoding VEXAS syndrome: emerging insights into pathogenesis and clinical management.
Martina Fiumara, Corrado Campochiaro, Raffaella Molteni
https://pubmed.ncbi.nlm.nih.gov/41175032Actively Recruiting
Phase 2
Age: 18Years - 75Years
All Genders
ID05027945
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant to Treat VEXAS Syndrome
Led by National Cancer Institute (NCI) · Updated on 2026-05-08
54
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are investigating the use of allogeneic hematopoietic stem cell transplantation (HSCT) to treat VEXAS Syndrome, a newly identified disease characterized by inflammatory and blood-related problems. This syndrome involves symptoms such as fever, skin lesions, and bone marrow failure, often resistant to standard treatments. The study aims to determine if HSCT can successfully replace the patient's bone marrow with donor cells and improve or reverse the disease's clinical features over time. Participants will receive a stem cell transplant from a matched or haploidentical donor after undergoing a reduced intensity conditioning regimen tailored to the donor match type. This includes drugs like fludarabine, busulfan, cyclophosphamide, and possibly low-dose total body irradiation. After the transplant, participants will receive medications such as mycophenolate mofetil and tacrolimus to prevent graft-versus-host disease. The transplant is given via a central venous catheter, and participants must stay near the hospital for at least 100 days with frequent follow-up visits. During the study, participants will undergo extensive screening including physical exams, imaging scans, blood and urine tests, bone marrow biopsies, and specialist consultations. Post-transplant, they will attend scheduled visits up to two years after receiving the transplant to monitor donor cell engraftment, disease reversal, safety outcomes, and any complications like graft-versus-host disease. The study includes yearly follow-ups by phone after two years, with total participation possibly extending beyond three years.
CONDITIONS
Brief 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
Age: 18Years - 75Years
All Genders
Eligibility Criteria
You may qualify if you...
- Age 18 to 75 years
- 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 ejection fraction over 35%
- Creatinine level 2.0 mg/dl or less and creatinine clearance 30 ml/min or higher
- Liver function with conjugated bilirubin less than 3.0 mg/dl and ALT/AST less than or equal to 5 times upper limit of normal
- Lung function with FEV1 and DLCO greater than 30%
- Ability and willingness to sign informed consent
- Use of adequate contraception during the study and for one year after transplant if of childbearing potential or male
- Willingness to stay near the hospital for at least 100 days after transplant with adult caregiver support if outpatient
- Diagnosis or clinical history consistent with VEXAS Syndrome with characteristic inflammatory symptoms or cytopenia or myeloid neoplasm
- Refractory inflammatory symptoms requiring prednisone at 0.5 mg/kg per day or more, or intolerance or failure of standard treatments
You will not qualify if you...
- HCT Comorbidity Index of 5 or higher
- Diagnosis of multiple myeloma (except low risk smoldering or monoclonal gammopathy of unknown significance)
- Use of other investigational agents within 30 days before treatment
- HIV infection
- History of allergic reactions to study drugs including steroids, cyclophosphamide, busulfan, tacrolimus, mycophenolate mofetil, or filgrastim
- Pregnant or breastfeeding individuals
- Uncontrolled illnesses or social situations limiting study compliance
- Active uncontrolled infections
- Active psychiatric disorders that may affect compliance
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Approximately 6 months
Participants receive reduced intensity conditioning chemotherapy and/or radiation followed by allogeneic hematopoietic stem cell transplant (HSCT) on day 0. Post-transplant graft-versus-host disease (GVHD) prophylaxis medications are given for up to approximately 6 months.
Daily visits during conditioning and transplant period plus frequent visits for post-transplant GVHD prophylaxis management
Follow-up
Duration - Up to 3 years post-transplant
Participants are monitored for clinical response, reversal of VEXAS symptoms, donor engraftment, safety, and transplant-related complications including GVHD for up to 3 years after transplant.
Regular follow-up visits for assessments at day +100, 1 year, 2 years, and 3 years post-transplant
Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Loading map...
Research Team
B
Bhavisha A Patel, M.D.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here
Published Research Related To This Trial
VEXAS Syndrome-A Review of Pathophysiology, Presentation, and Prognosis.
Matthew J Koster, Matthew J Samec, Kenneth J Warrington
https://pubmed.ncbi.nlm.nih.gov/36251488