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NCT Number: NCT06377462

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

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Key information

Conditions

Vexas Syndrome

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Universitätsklinikum Aachen, Aachen, Germany

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About this study

see brief summary

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. Collection of epidemiological data on VEXAS

    Time frame: enrollment

    incidence, age and sex distribution in adults in Germany

  2. Collection and monitoring of initial disease manifestations

    Time frame: 5 years

    • spectrum and frequency of inflammatory manifestations
    • Association with hematological (pre)malignancies (MDS, Myeloma, MGUS, CHIP)
    • Monitor cardiovascular risk/thromboembolic events

  3. Documentation of the treatment approaches and therapy sequences

    Time frame: 5 years

    Documentation of the treatment approaches and therapy sequences

  4. Analysis of clinical-relevant clinical endpoints

    Time frame: 5 years

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

Secondary outcomes

  1. Describe disease cluster

    Time frame: 5 years

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

  2. Laboratory diagnostics

    Time frame: 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

  3. Correlation clinical endpoints with treatment approaches

    Time frame: 5 years

    Correlation clinical endpoints with treatment approaches

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

    Time frame: 5 years

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

  5. Accompanying translational research

    Time frame: 5 years

    • Metabolic signature
    • Inflammatory signature
    • Microbiome
    • Identify disease trigger /risc factors for disease onset

  6. Collection of Biospecimens

    Time frame: 5 years

    Collection of Biospecimens

Study contacts

Contact information is provided by the study sponsor or research team.

Katharina Goetze, Prof.

CONTACT

[email protected]

+498941404618

Katja Sockel, Dr. med.

CONTACT

[email protected]

+493514585627

Sponsors and collaborators

Lead sponsor

Technische Universität Dresden

Other

Collaborators

  • Bristol-Myers Squibb
  • University Hospital Munich

Registry information

Important dates

Study start
2024
Primary completion
2029
Study completion
2030
First posted
Apr 22, 2024
Registry last updated
May 15, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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