Multicenter, Interdisciplinary National VEXAS Registry With Accompanying Biomaterial Collection

Technische Universität Dresden500 enrolled

Overview

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

see brief summary

Study Type

OBSERVATIONAL

Enrollment

500

Conditions

VEXAS Syndrome

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (13)

Universitätsklinikum Aachen

Aachen, Germany

RECRUITING

Klinikum Chemnitz gGmbH

Chemnitz, Germany

RECRUITING

Outcomes

Primary Outcomes

Collection of epidemiological data on VEXAS

incidence, age and sex distribution in adults in Germany

Time frame: 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

Time frame: 5 years

Documentation of the treatment approaches and therapy sequences

Time frame: 5 years

Analysis of clinical-relevant clinical endpoints

complete remission (CR), hematological and clinical remission, molecular remissions, treatment-free remission (TFR)

Time frame: 5 years

Secondary Outcomes

Describe disease cluster

* Phenotype cluster? * Genotype-phenotype associations? * Identification of biomarker?

Time frame: 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

Time frame: 5 years

Correlation clinical endpoints with treatment approaches

Time frame: 5 years

Central Contacts

Katja Sockel, Dr. med.

CONTACT

+493514585627 katja.sockel@ukdd.de

Katharina Goetze, Prof.

CONTACT

+498941404618 katharina.goetze@tum.de

Data from ClinicalTrials.gov

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