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 associations, determine optimal treatment schedule, identify diagnostic features and biomarkers

Study Overview

• Status: Recruiting
• Conditions: VEXAS Syndrome

Detailed Description

see brief summary

• Study Type: Observational
• Enrollment (Estimated): 500

Contacts and Locations

• Study Contact: Name: Katja Sockel, Dr. med.; Phone Number: +493514585627; Email: katja.sockel@ukdd.de
• Study Contact Backup: Name: Katharina Goetze, Prof.; Phone Number: +498941404618; Email: katharina.goetze@tum.de

Study Locations

• Germany
  • Aachen, Germany
    • Recruiting
    • Universitätsklinikum Aachen
  • Chemnitz, Germany
    • Recruiting
    • Klinikum Chemnitz gGmbH
  • Dresden, Germany, 01307
    • Recruiting
    • Universitatsklinikum Carl Gustav Carus
    • Contact: Katja Sockel, Dr. med.
  • Düsseldorf, Germany
    • Recruiting
    • Universitätsklinikum Düsseldorf
  • Essen, Germany
    • Recruiting
    • Evang. Kliniken Essen-Mitte
  • Göttingen, Germany
    • Recruiting
    • Universitätsmedizin Göttingen
  • Hamburg, Germany
    • Recruiting
    • Universitatsklinikum Hamburg-Eppendorf
  • Jena, Germany
    • Recruiting
    • Universitatsklinikum Jena
  • Leipzig, Germany
    • Recruiting
    • Universitätsklinikum Leipzig AöR
  • Mannheim, Germany
    • Recruiting
    • Universitätsmedizin Mannheim
  • München, Germany
    • Recruiting
    • Klinikum rechts der Isar TUM
  • Nuremberg, Germany
    • Recruiting
    • Klinikum Nürnberg, Campus Nord
  • Osnabrück, Germany
    • Recruiting
    • Klinikum Osnabrück GmbH
  • Schwerin, Germany
    • Recruiting
    • Helios Kliniken Schwerin GmbH
  • Stuttgart, Germany
    • Recruiting
    • Robert Bosch Gesellschaft für Medizinische Forschung mb
  • Trier, Germany
    • Recruiting
    • Krankenhaus der Barmherzigen Brüder
  • Tübingen, Germany
    • Recruiting
    • Universitatsklinikum Tubingen

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

• Planned recruitment: 20-30 patients/year
• Number of planned study centres: 15-20

Description

Inclusion Criteria:

• Patients with established or suspected (clinical and hematological criteria) VEXAS Syndrome
• Age ≥18 years
• Signed informed consent form

Exclusion Criteria:

• patients who are not in a position to understand the nature and scope of participation in this register

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Collection of epidemiological data on VEXAS	incidence, age and sex distribution in adults in Germany	enrollment
Collection and monitoring of initial disease manifestations	spectrum and frequency of inflammatory manifestations; Association with hematological (pre)malignancies (MDS, Myeloma, MGUS, CHIP); Monitor cardiovascular risk/thromboembolic events	5 years
Documentation of the treatment approaches and therapy sequences	Documentation of the treatment approaches and therapy sequences	5 years
Analysis of clinical-relevant clinical endpoints	complete remission (CR), hematological and clinical remission, molecular remissions, treatment-free remission (TFR)	5 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Describe disease cluster	Phenotype cluster?; Genotype-phenotype associations?; Identification of biomarker?	5 years
Laboratory diagnostics	Establish Diagnostic FACS signature; Establish biomarker for disease monitoring; Molecular characterization of the key regulatory gene UBA1, but also accompanying molecular mutations; Correlation with HLA polymorphisms; Coagulation Diagnostics	5 years
Correlation clinical endpoints with treatment approaches	Correlation clinical endpoints with treatment approaches	5 years
Quality of life data (QoL), fatigue questionnaire (Facit-F)	Quality of life data (QoL), fatigue questionnaire (Facit-F)	5 years
Accompanying translational research	Metabolic signature; Inflammatory signature; Microbiome; Identify disease trigger /risc factors for disease onset	5 years
Collection of Biospecimens	Collection of Biospecimens	5 years

Collaborators and Investigators

• Sponsor: Technische Universität Dresden
• Collaborators: Bristol-Myers Squibb; University Hospital Munich
• Investigators: Study Chair: Katja Sockel, Dr. med., TU Dresden

Publications and helpful links

Helpful Links

• Website VEXAS Registry

Study record dates

Study Major Dates

• Study Start (Actual): March 13, 2024
• Primary Completion (Estimated): December 31, 2029
• Study Completion (Estimated): December 31, 2030

Study Registration Dates

• First Submitted: April 17, 2024
• First Submitted That Met QC Criteria: April 17, 2024
• First Posted (Actual): April 22, 2024

Study Record Updates

• Last Update Posted (Actual): May 15, 2026
• Last Update Submitted That Met QC Criteria: May 13, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: VEXAS
• Additional Relevant MeSH Terms: VEXAS syndrome
• Other Study ID Numbers: VEXAS-Registry

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No