Multicenter, Interdisciplinary National VEXAS Registry With Accompanying Biomaterial Collection

NCT06377462 | Recruiting

• 1 other identifier: VEXAS-Registry
• Study Type: observational
• Target: 500
• Locations: 1 country
• Sites: 13

Brief Summary

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

Conditions

VEXAS Syndrome

Keywords

VEXAS

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (6)

• Describe disease cluster

  5 years

• Laboratory diagnostics

  5 years

• Correlation clinical endpoints with treatment approaches

  5 years

• Quality of life data (QoL), fatigue questionnaire (Facit-F)

  5 years

• Accompanying translational research

  5 years

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Technische Universität Dresden: lead
• Bristol-Myers Squibb: collaborator
• University Hospital Munich: collaborator

Study Sites (13)

Universitätsklinikum Aachen

Aachen, Germany

RECRUITING

Klinikum Chemnitz gGmbH

Chemnitz, Germany

RECRUITING

Universitätsklinikum Carl Gustav Carus

Dresden, 01307, Germany

RECRUITING

Universitätsklinikum Düsseldorf

Düsseldorf, Germany

RECRUITING

Evang. Kliniken Essen-Mitte

Essen, Germany

RECRUITING

Universitätsklinikum Hamburg-Eppendorf

Hamburg, Germany

RECRUITING

Universitätsklinikum Leipzig AöR

Leipzig, Germany

RECRUITING

Universitätsmedizin Mannheim

Mannheim, Germany

RECRUITING

Klinikum rechts der Isar TUM

München, Germany

RECRUITING

Klinikum Osnabrück GmbH

Osnabrück, Germany

RECRUITING

Helios Kliniken Schwerin GmbH

Schwerin, Germany

RECRUITING

Robert Bosch Gesellschaft für Medizinische Forschung mb

Stuttgart, Germany

RECRUITING

Krankenhaus der Barmherzigen Brüder

Trier, Germany

RECRUITING

Related Links

• Website VEXAS Registry

Biospecimen

Retention: SAMPLES WITH DNA

18 mL whole blood (peripheral venipuncture) and 9 ml bone marrow

MeSH Terms

Conditions

VEXAS syndrome

Study Officials

• Katja Sockel, Dr. med.

  TU Dresden

  STUDY CHAIR

Central Study Contacts

Katja Sockel, Dr. med.

CONTACT

+493514585627; katja.sockel@ukdd.de

Katharina Goetze, Prof.

CONTACT

+498941404618; katharina.goetze@tum.de

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 17, 2024
• First Posted: April 22, 2024
• Study Start: March 13, 2024
• Primary Completion (Estimated): December 31, 2029
• Study Completion (Estimated): December 31, 2030
• Last Updated: September 19, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

DE (13)