Multicenter, Interdisciplinary National VEXAS Registry With Accompanying Biomaterial Collection

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT06377462

Enrollment

500

Registered

2024-04-22

Start date

2024-03-13

Completion date

2030-12-31

Last updated

2026-05-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

VEXAS Syndrome

Keywords

VEXAS

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

Detailed description

see brief summary

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

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

Secondary

Measure	Time frame	Description
Describe disease cluster	5 years	* Phenotype cluster? * Genotype-phenotype associations? * Identification of biomarker?
Laboratory diagnostics	5 years	* 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
Correlation clinical endpoints with treatment approaches	5 years	Correlation clinical endpoints with treatment approaches
Quality of life data (QoL), fatigue questionnaire (Facit-F)	5 years	Quality of life data (QoL), fatigue questionnaire (Facit-F)
Accompanying translational research	5 years	* Metabolic signature * Inflammatory signature * Microbiome * Identify disease trigger /risc factors for disease onset
Collection of Biospecimens	5 years	Collection of Biospecimens

Countries

Germany

Contacts

CONTACTKatja Sockel, Dr. med.

katja.sockel@ukdd.de+493514585627

CONTACTKatharina Goetze, Prof.

katharina.goetze@tum.de+498941404618

STUDY_CHAIRKatja Sockel, Dr. med.

TU Dresden

Outcome results