Multicenter, Interdisciplinary National VEXAS Registry With Accompanying Biomaterial Collection

Recruiting

• Conditions: VEXAS Syndrome

• Registration Number: NCT06377462
• Lead Sponsor: Technische Universität Dresden

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

Study Timeline

• Study Start: 2024-03-13
• Status Verified: 2024-04
• Study First Submitted: 2024-04-17
• Study First Submitted QC: 2024-04-17
• Last Update Submitted: 2024-04-17
• Study First Posted: 2024-04-22
• Last Update Posted: 2024-04-22
• Primary Completion: 2029-12-31
• Study Completion: 2030-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 500

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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
Analysis of clinical-relevant clinical endpoints	5 years	complete remission (CR), hematological and clinical remission, molecular remissions, treatment-free remission (TFR)
Collection of epidemiological data on VEXAS	enrollment	incidence, age and sex distribution in adults in Germany
Documentation of the treatment approaches and therapy sequences	5 years	Documentation of the treatment approaches and therapy sequences

Secondary Outcome Measures

Name	Time	Method
Quality of life data (QoL), fatigue questionnaire (Facit-F)	5 years	Quality of life data (QoL), fatigue questionnaire (Facit-F)
Collection of Biospecimens	5 years	Collection of Biospecimens
Correlation clinical endpoints with treatment approaches	5 years	Correlation clinical endpoints with treatment approaches
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
Accompanying translational research	5 years	* Metabolic signature; * Inflammatory signature; * Microbiome; * Identify disease trigger /risc factors for disease onset

Trial Locations

Locations (1)

Universitätsklinikum Carl Gustav Carus; 🇩🇪 Dresden, Germany