8 recruiting, 3 not yet recruiting, 1 completed, 1 enrolling by invitation. 4 Phase 2, 1 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Recruiting 3, Not yet recruiting 1 |
| Phase 1 | 1 | Recruiting 1 |
| Phase 3 | 1 | Not yet recruiting 1 |
Follow VEXAS syndrome: new trials by email Literature Review of VEXAS syndrome trials Research Report on VEXAS syndrome
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A 52-Week Study Evaluating the Efficacy and Safety of Arumakimig (MAS825) in Participants With VEXAS Followed by Open-Label Extension (OLE) Period Arumakimig, Placebo, Oral glucocorticoids - VEXAS Syndrome | Phase 2 | Recruiting | Novartis Pharmaceuticals | 120 | 2026-10-30 | 2026-10-13 |
| A Study Evaluating the Efficacy and Safety of Momelotinib in Participants With Vacuoles, E1-enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome Momelotinib, Glucocorticoids, Placebo - VEXAS Syndrome | Phase 2 / Phase 3 | Not yet recruiting | GlaxoSmithKline | 136 | 2026-08-05 | 2026-06-01 |
| Clinical Phenotype and Prevalence of VEXAS Syndrome in Internal Medicine UBA1 Genetic Testing - Vexas Syndrome | Not yet recruiting | Centre Hospitalier Universitaire Vaudois | 50 | 2026-07-01 | 2026-07-16 | |
| Clonal Hematopoiesis of Immunological Significance observational cohort study - Immune System Diseases, Autoimmune Diseases | Not yet recruiting | Assistance Publique - Hôpitaux de Paris | 5000 | 2026-04-01 | 2026-03-23 | |
| Momelotinib in VEXAS Syndrome Momelotinib treatment - VEXAS Syndome, Myelo Dysplastic Syndrome | Phase 2 | Recruiting | Groupe Francophone des Myelodysplasies | 57 | 2025-11-25 | 2026-08-10 |
| Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study Bone Marrow Failure Disorders, VEXAS Syndrome | Enrolling by invitation | National Heart, Lung, and Blood Institute (NHLBI) | 1400 | 2025-09-09 | 2026-10-15 | |
| A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS) Pacritinib, Placebo - VEXAS, VEXAS Syndrome | Phase 2 | Recruiting | Swedish Orphan Biovitrum | 156 | 2025-05-28 | 2026-10-16 |
| Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome Pacritinib - E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic Syndrome, VEXAS | Phase 1 | Recruiting | Washington University School of Medicine | 15 | 2025-02-13 | 2026-07-15 |
| HRQoL and Financial Toxicity in Patients With VEXAS Syndrome VEXAS | Recruiting | Gruppo Italiano Malattie EMatologiche dell'Adulto | 100 | 2024-11-01 | 2025-07-15 | |
| Multicenter, Interdisciplinary National VEXAS Registry With Accompanying Biomaterial Collection VEXAS Syndrome | Recruiting | Technische Universität Dresden | 500 | 2024-03-13 | 2026-05-15 | |
| Autoimmune and Autoinflammatory Genetics Study VEXAS Syndome, Autoinflammatory Disease | Recruiting | NYU Langone Health | 1500 | 2022-04-15 | 2026-07-08 | |
| DECIPHERING AND TARGETING VEXAS VEXAS | Completed | IRCCS San Raffaele | 60 | 2021-01-01 | 2026-08-05 | |
| AutoInflammatory Disease Alliance Registry (AIDA) No intervention is foreseen by the protocol. - Hereditary Autoinflammatory Diseases, Schnitzler Syndrome | Recruiting | University of Siena | 3500 | 2020-08-06 | 2025-07-10 |
Myelodysplastic Syndromes (4) · Autoimmune Diseases (2) · Inflammation (2) · Anemia, Aplastic (1) · Arthralgia (1) · Arthritis (1) · Arthritis, Juvenile (1) · Behcet Syndrome (1) · Bone Marrow Diseases (1) · Bone Marrow Failure Disorders (1) · Castleman Disease (1) · Hematologic Diseases (1) · Hematologic Neoplasms (1) · Hemoglobinuria, Paroxysmal (1)
Hemic and Lymphatic Diseases (5) · Bone Marrow Diseases (3) · Hematologic Diseases (3) · Musculoskeletal Diseases (3) · Pathological Conditions, Signs and Symptoms (3) · Connective Tissue Diseases (2) · Immune System Diseases (2) · Immunoproliferative Disorders (2) · Joint Diseases (2) · Lymphatic Diseases (2) · Neoplasms (2) · Paraproteinemias (2) · Pathologic Processes (2) · Skin and Connective Tissue Diseases (2) · Anemia (1)
11-(2-pyrrolidin-1-ylethoxy)-14,19-dioxa-5,7,26-triazatetracyclo(19.3.1.1(2,6).1(8,12))heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene (2) · Glucocorticoids (2) · N-(cyanomethyl)-4-(2-((4-(4-morpholinyl)phenyl)amino)-4-pyrimidinyl)benzamide (1)
Assistance Publique - Hôpitaux de Paris (1) · Centre Hospitalier Universitaire Vaudois (1) · GlaxoSmithKline (1) · Groupe Francophone des Myelodysplasies (1) · Gruppo Italiano Malattie EMatologiche dell'Adulto (1) · IRCCS San Raffaele (1) · NYU Langone Health (1) · National Heart, Lung, and Blood Institute (NHLBI) (1) · Novartis Pharmaceuticals (1) · Swedish Orphan Biovitrum (1) · Technische Universität Dresden (1) · University of Siena (1) · Washington University School of Medicine (1)
Italy (4) · United States (4) · France (3) · Germany (3) · Japan (2) · Spain (2) · Algeria (1) · Australia (1) · Belgium (1) · Brazil (1) · Canada (1) · China (1) · Colombia (1) · Egypt (1) · Greece (1)
Alberto Tommasini (1) · Bhavisha A Patel, M.D. (1) · David Beck, MD, PhD (1) · Fabio Fiammengo (1) · Francesco Zaja (1) · Giacomo Emmi, MD, PhD (1) · Katja Sockel, Dr. med. (1) · Maria Letizia Urban (1) · Meagan A Jacoby, M.D., Ph.D. (1) · Simone Cenci, MD (1)
PMID 35463015, PMID 35755024, PMID 35831701, PMID 35899212, PMID 35935379, PMID 36160138, PMID 36881285, PMID 37517110, PMID 38206518, PMID 40175882, PMID 41753113, PMID 42444147
2025 (1)