Research — VEXAS Foundation
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Research & Science

We're working to advance the research, clinical trials, and emerging science that are changing what VEXAS means for patients.

Our Research Funding Program

The VEXAS Foundation is working to fund meaningful research into VEXAS syndrome. We're evaluating research grant opportunities now, with funding decisions expected later in 2026. In the meantime, here's how we're building toward a strong research program.

01
Genetics & Mechanism
Research priority area
Research Priority
UBA1 mutation mechanisms & novel treatment pathways

A key research priority is understanding the genetic underpinnings of VEXAS and identifying potential treatment pathways to improve patient outcomes.

Learn more
02
Patient Registry
Research priority area
Research Priority
International patient registry & biobank

Building a global database of VEXAS patients could accelerate discovery, standardize diagnostic criteria, and connect patients to specialist care centers worldwide.

Learn more
26
2026 Grant Program
Details being finalized
Coming Soon
2026 Research Grant Program

We're working toward funding significant VEXAS research grants, with details expected later in 2026. Grant criteria will be posted here once finalized.

Contact us

Are you a researcher working on VEXAS?

We're finalizing our research grant program and welcome you to get in touch about future opportunities.

Clinical trials for VEXAS

Clinical trials are how better VEXAS treatments get developed — and how patients gain access to therapies before they are widely available. Because trials open and close continually, we point you to ClinicalTrials.gov, the U.S. National Library of Medicine's official registry, so you always see the current list rather than an outdated one.

Always current
Search all VEXAS trials on ClinicalTrials.gov

Every registered VEXAS study worldwide — recruiting status, locations, eligibility criteria, and study contacts — maintained by the U.S. National Library of Medicine and updated by the trial sponsors themselves.

Why trials matter for VEXAS: There are currently no FDA-approved treatments specifically for VEXAS syndrome. Treatments used today are prescribed off-label or are being studied in clinical trials. Trials help researchers understand what works, for whom, and how safely, while moving us closer to treatments developed and approved specifically for VEXAS.

Currently registered VEXAS trials

Recruiting
A 52-Week Study Evaluating the Efficacy and Safety of Arumakimig (MAS825) in Participants With VEXAS Followed by Open-Label Extension (OLE) Period
Novartis Pharmaceuticals · PHASE2 · Japan
Active, Not Recruiting
A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)
Swedish Orphan Biovitrum · PHASE2 · 8 countries
Recruiting
Momelotinib in VEXAS Syndrome
Groupe Francophone des Myelodysplasies · PHASE2 · France
Completed
DECIPHERING AND TARGETING VEXAS
IRCCS San Raffaele · Italy
Not Yet Recruiting
Clinical Phenotype and Prevalence of VEXAS Syndrome in Internal Medicine
Centre Hospitalier Universitaire Vaudois
Recruiting
Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome
Washington University School of Medicine · PHASE1 · United States
Recruiting
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome
National Cancer Institute (NCI) · PHASE2 · United States
Recruiting
Autoimmune and Autoinflammatory Genetics Study
NYU Langone Health · United States
Not Yet Recruiting
A Study Evaluating the Efficacy and Safety of Momelotinib in Participants With Vacuoles, E1-enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome
GlaxoSmithKline · PHASE2, PHASE3
Recruiting
Multicenter, Interdisciplinary National VEXAS Registry With Accompanying Biomaterial Collection
Technische Universität Dresden · Germany
Not Yet Recruiting
Clonal Hematopoiesis of Immunological Significance
Assistance Publique - Hôpitaux de Paris · France
Enrolling by Invitation
Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study
National Heart, Lung, and Blood Institute (NHLBI) · United States
Recruiting
HRQoL and Financial Toxicity in Patients With VEXAS Syndrome
Gruppo Italiano Malattie EMatologiche dell'Adulto · Italy
Recruiting
AutoInflammatory Disease Alliance Registry (AIDA)
University of Siena · 22 countries

How to search

1
Search the condition

Enter VEXAS syndrome in the condition field. You can also try UBA1 to catch studies indexed by the gene rather than the syndrome name.

2
Filter by status and location

Narrow to Recruiting and Not yet recruiting to see studies you could still join, then filter by country or distance from home.

3
Read eligibility carefully

Each listing has an eligibility section and a study contact. Bring anything promising to your rheumatologist or hematologist before reaching out.

Understanding trial status

Recruiting Actively enrolling participants now. These are the studies most likely to be open to you.
Not yet recruiting Approved but not open yet. Worth noting and checking back, or asking the listed contact about timing.
Active, not recruiting Running, but enrollment has closed. Results may be published later — useful context, not an option to join.
Completed Finished. Findings often appear in the published literature — see our Published Studies tab.

Questions worth asking your care team

If you find a study that looks relevant, these are useful things to raise at your next appointment.

Do I meet the eligibility criteria for this study?
How would participating affect my current treatment?
What are the risks and possible side effects?
What travel, visits, or time commitment is involved?
Are any costs covered, and what would I pay?
Can I leave the study if I change my mind?

The VEXAS Foundation does not run clinical trials and cannot advise on eligibility. Trial decisions should always be made with your treating physician. If you do not currently have a physician experienced with VEXAS, our Medical Centers listing may help.

Published studies on VEXAS

Recent peer-reviewed publications on VEXAS syndrome, pulled automatically from PubMed and sorted by publication date.

2026
Orf-Like Ulcerated Digital Nodule as a Cutaneous Manifestation of VEXAS Syndrome.
Uğurlu S, Yıldız P, Dayı G
International journal of dermatology, 2026
2026
Early-onset VEXAS syndrome mimicking orbital cellulitis and successfully treated with allogeneic hematopoietic stem cell transplantation.
Burul G, Karaoglan Ç, Bektaş E, et al.
Journal of hematopathology, 2026
2026
Evaluating the Efficacy of Cytokine-Directed Therapies in the Management of VEXAS Syndrome.
Montes D, Langenfeld HE, Hanson AC, et al.
ACR open rheumatology, 2026
2026
Unique Aspects of Allogeneic Hematopoietic Stem Cell Transplantation in VEXAS Syndrome: One Size Does Not Fit All.
Mangaonkar AA, Alkhateeb HB
American journal of hematology, 2026
2026
Assessing the Frequency of VEXAS-Related Canonical UBA1 Mutations in Myelodysplastic Syndrome Patients.
Soleimanzadeh H, Sharifi M, Shokripour M, et al.
European journal of haematology, 2026
2026
Peripheral blood UBA1 variant burden predicts poor outcomes in VEXAS syndrome: a nationwide prospective study.
Kirino Y, Maeda A, Higashitani K, et al.
Annals of the rheumatic diseases, 2026
2026
VEXAS Syndrome Associated With a Non-Canonical UBA1 p.Ala478Ser Variant: Diagnostic and Clinical Implications.
Scarpelli I, Uhr M, Leoncini L, et al.
International journal of laboratory hematology, 2026
2026
Endotheliopathy and VWF-ADAMTS13 axis dysfunction in VEXAS thrombogenicity.
Hulshof AM, Groarke EM, Schneppenheim S, et al.
Blood, 2026
2026
UBA1 as a proteostasis-innate immunity checkpoint: lessons from vexas syndrome and therapeutic implications.
Zhang Y, Su Q
Frontiers in immunology, 2026
2026
Myelodysplasia in VEXAS syndrome.
Patel BA, Calvo KR, Reichard KK, et al.
Blood advances, 2026
2026
Human Monocytic Models Reveal Genotype-Dependent Inflammatory Programs in VEXAS Syndrome.
Higashitani K, Ban T, Kirino Y, et al.
Arthritis & rheumatology (Hoboken, N.J.), 2026
2026
Diagnostic and Therapeutic Challenge: VEXAS Syndrome with Multisystem Inflammation and Myelodysplasia: A Case Report.
Almaalouli B, Smith H, Kirsch G, et al.
Mediterranean journal of rheumatology, 2026
2026
Acute Fulminant Systemic Granulocytic Vasculitis Involving Arteries and Veins in Vacuoles, E1 Enzyme, X-Linked, Autoinflammatory, Somatic (VEXAS) Syndrome: An Autopsy Case Report.
Ueda K, Fujimoto M, Shiroishi Y, et al.
Pathology international, 2026
2026
Retraction notice to "Rapid cutaneous remission response to tofacitinib and systemic corticosteroids in VEXAS syndrome with a novel UBA1 mutation" [JAAD Case Reports 68 (2026) 105-107].
JAAD case reports, 2026
2026
Dual somatic UBA1 Met41 variants with distinct allele frequencies in VEXAS syndrome.
Carvajal Bedoya G, Ferrada M, Gnecco D
EULAR rheumatology open, 2026
2026
Dysfunctional UBA1-mutated myeloid cells promote inflammation in VEXAS syndrome.
Webster H
Nature reviews. Rheumatology, 2026
2026
Therapeutic Outcomes in VEXAS Syndrome: A Multicenter Comparative Cohort of Allogeneic Hematopoietic Stem Cell Transplantation and Hypomethylating Agents.
Fathima S, Wong MM, Gonzalez-Lugo J, et al.
American journal of hematology, 2026
2026
VEXAS Syndrome: Clues to Avoid Vexation.
Kumar D, Panse G, Ko CJ
Journal of cutaneous pathology, 2026
2026
VEXAS Syndrome:Report of One Case.
Liu HT, Zhou JX, Zhang RX, et al.
Zhongguo yi xue ke xue yuan xue bao. Acta Academiae Medicinae Sinicae, 2026
2026
Vexas Syndrome: A Multisystem Challenge with Sweet Syndrome and Myelodysplastic Syndrome.
Kouli A, Laflouf M, Alswij MA, et al.
European journal of case reports in internal medicine, 2026
2026
Sweet syndrome, macrocytic anaemia, and elevated IgE in VEXAS syndrome: a series of three cases from Taiwan.
Yeh TC, Wang SH, Chang LC, et al.
Pathology, 2026
2026
Hypomethylating Therapy in VEXAS Syndrome: Disease Modification Beyond Bridging to Transplant.
Ammatuna E
American journal of hematology, 2026
2026
Unmasking Macrophage Activation Syndrome in Vexas Syndrome: A Diagnostic Challenge.
Boissais A, Farrulo B, Moulinet T, et al.
European journal of case reports in internal medicine, 2026
2026
An Italian cartography of VEXAS-related thrombosis.
Ranucci G, Pascale MR, Forte V, et al.
HemaSphere, 2026
2026
VEXAS Syndrome: A Comprehensive Review for Dermatologists Part 1: Epidemiology, Pathophysiology, and Clinical Presentations.
Kaltchenko M, Patel S, Srikumar A, et al.
Journal of the American Academy of Dermatology, 2026
2026
VEXAS Syndrome: A Comprehensive Review for Dermatologists Part 2: Work-up, Diagnosis, Treatment, and Prognosis.
Patel S, Kaltchenko M, Srikumar A, et al.
Journal of the American Academy of Dermatology, 2026
2026
VEXAS Syndrome with Vibrio vulnificus Sepsis during Treatment with Tocilizumab.
Kawakami E, Suzuki T, Tsuji S, et al.
Internal medicine (Tokyo, Japan), 2026

VEXAS Medical Centers of Excellence

These centers have direct experience diagnosing and treating VEXAS syndrome. They can work alongside your local medical team to provide more informed, effective care.

“Finding the right specialist team made an enormous difference for me.”

Ian · Diagnosed with VEXAS, 2024

United States — Centers of Excellence
NYU Langone Health
New York, NY
Dr. David B. Beck (internist, geneticist)
Dr. Gary Ho (rheumatologist)
Dr. Jun Choi (hematologist/oncologist)
Dedicated multidisciplinary VEXAS Syndrome Care Program. Participates in ongoing research studies and clinical trials.
Mayo Clinic
Rochester, MN & Scottsdale, AZ
Dr. Mathew Koster · Dr. Kenneth Warrington (rheumatology)
Dr. Abhishek Mangaonkar · Dr. Mrinal Patnaik (hematology)
Dr. Megan Sullivan · Dr. Yael Kusne (Scottsdale)
Developed specialized testing and treatment approaches for VEXAS. Contributes significantly to diagnosis and management research.
National Institutes of Health (NIH)
Bethesda, MD
Dr. Peter Grayson (rheumatologist)
Dr. Dennis Hickstein (hematologist/oncologist)
Actively advancing VEXAS research through dedicated programs and clinical trials, including the Phase II HSCT trial. No cost to trial participants.
University of Maryland School of Medicine
Baltimore, MD
Dr. Marcela Ferrada
Multidisciplinary program involving rheumatologists and hematologists focused on VEXAS syndrome's complex symptoms.
Cleveland Clinic
Cleveland, OH
Rheumatology & Hematology departments
Comprehensive care for VEXAS patients involving rheumatologists and hematologists to manage the syndrome's complex symptoms.
MD Anderson Cancer Center
Houston, TX
Dr. Danielle Hammond (hematologist/oncologist)
Dr. Maryan Buni (rheumatologist)
Developed a practice serving patients with VEXAS syndrome and related clonally driven hemato-inflammatory diseases.
International — Research Networks & Registries

The networks below coordinate VEXAS research and registries in their countries. They are not a directory of clinics accepting direct referrals — if you are outside the United States, ask your rheumatologist or hematologist to contact the relevant national network about referral to a centre with VEXAS experience.

🇬🇧
VEXNET
United Kingdom
Leeds Teaching Hospitals NHS Trust
King's College Hospital, London
Royal Free Hospital, London
St George's University Hospitals, London
Cambridge University Hospitals
Oxford University Hospitals
The UK VEXAS interest group. These six tertiary centres are described in published research as the primary diagnostic and treatment hubs within the VEXNET collaboration.
🇫🇷
FRENVEX
France
Hôpital Tenon, AP-HP, Paris
Hôpital Saint-Antoine, AP-HP, Paris
Hôpital Cochin, AP-HP, Paris
Hôpital Saint-Louis, AP-HP, Paris
+ centres nationwide
The French VEXAS study group — one of the largest VEXAS registries worldwide. Source of the major azacitidine and targeted therapy studies referenced on this site.
🇩🇪
German VEXAS Registry
Germany
RWTH Aachen University
Universitätsklinikum Carl Gustav Carus, Dresden
Klinikum Rechts der Isar, TU München
Universitätsklinikum Leipzig
A national prospective registry established in May 2024 collecting clinical, treatment and outcome data across German centres.
🇪🇺
ERN RITA
European Reference Network
European Reference Network on Rare Immunodeficiency,
Autoinflammatory and Autoimmune Diseases
An EU-wide network connecting specialist centres for rare autoinflammatory conditions. Several VEXAS centres across Europe participate, including Leeds in the UK.
International VEXAS guidance now available
The American College of Rheumatology, working with the International VEXAS Working Group Expert Panel, has published the first international guidance statement for the diagnosis and management of VEXAS syndrome.
Visit ACR

Why your support matters

VEXAS was first identified in 2020, and research into its causes, treatments, and long-term outcomes is still rapidly evolving. Here's what's at stake.

VEXAS may affect nearly one million people worldwide

Current estimates suggest VEXAS may affect nearly one million people worldwide, yet it remains underdiagnosed and underrecognized. Greater awareness can help more people receive an accurate diagnosis and appropriate care.

No treatments are FDA-approved specifically for VEXAS

Treatments currently used for VEXAS are used off-label or are being studied in clinical trials. Continued research is essential to determine which treatments are most effective and to develop therapies specifically for VEXAS.

The opportunity to accelerate progress is now

Since VEXAS was identified in 2020, researchers have made remarkable progress in understanding the disease, its genetic variants, treatment options, and outcomes. Continued investment in awareness and research can help build on that momentum.

Stem cell transplant may offer a cure for selected patients

Allogeneic hematopoietic stem cell transplantation (HSCT) can eliminate the UBA1-mutated clone and may be curative for VEXAS. However, transplant carries significant risks and is appropriate only for carefully selected patients. Research continues to help determine who is most likely to benefit and when transplantation should be considered.

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