Clinical trials: VEXAS, active
Immune System Diseases, Autoimmune Diseases
Clonal Hematopoiesis of Immunological Significance
Ambispective, national, multicenter observational cohort study aimed at characterizing the satellite dysimmune manifestations of clonal hematopoiesis, including Vexas (Vacuoles, E1 enzyme, X-linked, Autoinflammatory and Somatic)…
- Conditions
- Immune System Diseases, Autoimmune Diseases, Inflammation, Autoinflammatory Diseases
- Trial name
- CHIS (NCT05969821)
- Testing
- observational cohort study
- Where
- France
- Volunteers
- 5,000 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
VEXAS, VEXAS Syndrome
A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)
This trial is to assess the effectiveness and safety of pacritinib in patients with VEXAS (i.e., Vacuoles in myeloid progenitors, E1 ubiquitin-activating enzyme, X-linked, autoinflammatory manifestations, and somatic) syndrome.
- Trial name
- NCT06782373
- Testing
- Pacritinib
- Where
- 39 sites in United States, Canada and 6 more
- Volunteers
- 156 people
- Sponsor
- Swedish Orphan Biovitrum
Immunodeficiency, Hematopoietic Stem Cell Transplantation
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome
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.
- Trial name
- NCT05027945
- Testing
- Allogeneic HSCT, Busulfan test dose, Mycophenolate mofetil (MMF)
- Where
- United States
- Volunteers
- 54 people
- Sponsor
- National Cancer Institute (NCI)
E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic Syndrome, VEXAS
Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome
VEXAS (vacuoles, E1 ubiqutin-activating enzyme, X-linked, autoinflammatory, somatic syndrome) is a recently described disorder with severe hematologic and rheumatologic manifestations caused by somatic variants in the ubiquitin…
- Conditions
- E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic Syndrome, VEXAS, Vexas Syndrome
- Trial name
- NCT06538181
- Testing
- Pacritinib
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Washington University School of Medicine
VEXAS
HRQoL and Financial Toxicity in Patients With VEXAS Syndrome
This multicenter cross-sectional observational study aims to describe health-related quality of life (HRQoL) and symptom profiles of patients with VEXAS syndrome.
- Trial name
- NCT06657846
- Where
- 17 sites in Italy
- Volunteers
- 100 people
- Sponsor
- Gruppo Italiano Malattie EMatologiche dell'Adulto
Vexas Syndrome
Clinical Phenotype and Prevalence of VEXAS Syndrome in Internal Medicine
The FIND-VEXAS project is a multicenter, cross-sectional observational study conducted in Internal Medicine departments in the Friuli Venezia Giulia region of Italy.
- Trial name
- FIND-VEXAS (NCT07708688)
- Testing
- UBA1 Genetic Testing
- Volunteers
- 50 people
- Sponsor
- Centre Hospitalier Universitaire Vaudois
VEXAS Syndome, Myelo Dysplastic Syndrome
Momelotinib in VEXAS Syndrome
Multicenter, phase II trial with safety run-in to evaluate the efficacy and safety of momelotinib in patients with VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory and Somatic) syndrome with or without associated…
- Trial name
- NCT07098936
- Testing
- Momelotinib treatment
- Where
- 11 sites in France
- Volunteers
- 57 people
- Sponsor
- Groupe Francophone des Myelodysplasies
Hereditary Autoinflammatory Diseases, Schnitzler Syndrome
AutoInflammatory Disease Alliance Registry (AIDA)
Autoinflammatory diseases (AID) are clinical entities characterized by recurrent inflammatory attacks in absence of infection, neoplasm or deregulation of the adaptive immune system.
- Conditions
- Hereditary Autoinflammatory Diseases, Schnitzler Syndrome, Behcet Syndrome, PFAPA Syndrome
- Trial name
- AIDA (NCT05200715)
- Testing
- No intervention is foreseen by the protocol.
- Where
- 112 sites in Algeria, Australia and 20 more
- Volunteers
- 3,500 people
- Sponsor
- University of Siena
VEXAS Syndrome
Multicenter, Interdisciplinary National VEXAS Registry With Accompanying Biomaterial Collection
The aim is rapid collection of real-life data on the epidemiology, treatment and disease course in patients with VEXAS syndrome during routine clinical practice and collect biomaterials to evaluate genotype-phenotype…
- Trial name
- NCT06377462
- Where
- 17 sites in Germany
- Volunteers
- 500 people
- Sponsor
- Technische Universität Dresden
VEXAS Syndrome
A 52-Week Study Evaluating the Efficacy and Safety of Arumakimig (MAS825) in Participants With VEXAS Followed by Open-Label Extension (OLE) Period
The purpose of this study is to evaluate clinical efficacy and safety of arumakimig (MAS825) compared to placebo in patients with Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) syndrome.
- Trial name
- NCT07748624
- Testing
- Arumakimig, Oral glucocorticoids
- Where
- 4 sites in Japan
- Volunteers
- 120 people
- Sponsor
- Novartis Pharmaceuticals
VEXAS Syndrome
A Study Evaluating the Efficacy and Safety of Momelotinib in Participants With Vacuoles, E1-enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome
This study will assess the efficacy and safety of momelotinib in participants with a diagnosis of VEXAS.
- Trial name
- ATLAS (NCT07569081)
- Testing
- Momelotinib, Glucocorticoids
- Volunteers
- 136 people
- Sponsor
- GlaxoSmithKline
VEXAS Syndome, Autoinflammatory Disease
Autoimmune and Autoinflammatory Genetics Study
This is an exploratory natural history protocol that will enroll patients with known genetic diseases, such as VEXAS syndrome, or as yet undiagnosed disorders of inflammation with the goal of improving our understanding of…
- Conditions
- VEXAS Syndome, Autoinflammatory Disease, Autoimmune Disease, Rheumatic Disease
- Trial name
- NCT06004349
- Where
- 2 sites in United States
- Volunteers
- 1,500 people, healthy volunteers welcome
- Sponsor
- NYU Langone Health
Bone Marrow Failure Disorders, VEXAS Syndrome
Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study
STUDY DESCRIPTION: Bone marrow failure can result from immune mediated attack on stem cells or from inherited genetic defects such as telomere biology disorders or Fanconi anemia.
- Conditions
- Bone Marrow Failure Disorders, VEXAS Syndrome, Hemoglobinurea, Paroxysmal, Myelodysplastic Syndromes
- Trial name
- NCT07102849
- Where
- United States
- Volunteers
- 1,400 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)