Skip to main content
OpenTrials
Recruiting

NCT Number: NCT06657846

HRQoL and Financial Toxicity in Patients With VEXAS Syndrome

This multicenter cross-sectional observational study aims to describe health-related quality of life (HRQoL) and symptom profiles of patients with VEXAS syndrome.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

VEXAS VEXAS syndrome

Age range

18 year–99 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Aou Ospedali Riuniti "Umberto I - G.M. Lancisi - G. Salesi"- Ancona - Sod Clinica Ematologica, Ancona, Italy

Loading trial locations.

About this study

Moreover, the study will investigate financial toxicity and treatment tolerability. To contextualize the results the data of VEXAS patients will be compared with a cohort of matched MDS patients.

The overall aim of the study is to establish benchmark HRQoL data to be used in future prospective studies and clinical trial design.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Adult patients (≥ 18 years old) with a confirmed diagnosis of VEXAS syndrome (UBA1 pathogenic mutation detected)
  • Written informed consent

Exclusion criteria

  • - Having any kind of psychiatric disorder or major cognitive dysfunction.
  • Not able to read and understand local language

Treatment and study plan

Primary outcomes

  1. EORTC QLQ-C30

    Time frame: one time at study entry

    Mean score of EORTC QLQ-C30 scales (0-100):

    • 5 functional scales (physical, role, emotional, social, and cognitive)
    • global health status/QoL scale

    --> higher scores indicating better function, QoL

    • 3 symptom scales (fatigue, nausea and vomiting, pain)
    • 6 single items (dyspnoea, insomnia, appetite loss, constipation, diarrhoea, financial difficulties)
    • higher scores indicating higher level of symptoms

  2. FACIT-Fatigue

    Time frame: one time at study entry

    Mean score of FACIT-Fatigue (0 to 52)

    --> higher scores indicating higher levels of fatigue.

Secondary outcomes

  1. PROFFIT-score

    Time frame: one time at study entry

    To investigate the association of the primary outcomes EORTC QLQ-C30, FACIT-Fatigue) and the PROFFIT-score (consisting of 7 items rated on a 4-point Likert scale)

  2. Percentage of patients above previously validated cut-off scores for the EORTC QLQ-C30 indicating clinically relevant problems and symptoms

    Time frame: one time at study entry

    To assess the percentage of patients above previously validated cut-off scores for the EORTC QLQ-C30 indicating clinically relevant problems and symptoms

  3. EORTC QLQ-C30 mean scale score stratified by age groups and sex.

    Time frame: one time at study entry

    To assess EORTC QLQ-C30 mean scale score stratified by age groups and sex.

    EORTC QLQ-C30 scales (0-100):

    • 5 functional scales (physical, role, emotional, social, and cognitive)
    • global health status/QoL scale
    • higher scores indicating better function, QoL
    • 3 symptom scales (fatigue, nausea and vomiting, pain)
    • 6 single items (dyspnoea, insomnia, appetite loss, constipation, diarrhoea, financial difficulties)
    • higher scores indicating higher level of symptoms

  4. FACIT-Fatigue mean score stratified by age groups and sex

    Time frame: one time at study entry

    To assess FACIT-Fatigue mean score stratified by age groups and sex. (0 to 52) --> higher scores indicating higher levels of fatigue.

  5. Score of the FACIT item GP5

    Time frame: one time at study entry

    To assess the score of the FACIT item GP5. Item GP5 from the FACIT Measurement System is a summary measure to assess the patient-reported overall impact of treatment toxicity on a 5-point Likert scale, with a higher score indicating higher treatment toxicity.

  6. Compare EORTC QLQ-C30 mean scale scores with a matched case control group of MDS patients

    Time frame: one time at study entry

    To compare EORTC QLQ-C30 mean scales scores with a matched case control group of MDS patients.

    EORTC QLQ-C30 scales (0-100):

    • 5 functional scales (physical, role, emotional, social, and cognitive)
    • global health status/QoL scale

    --> higher scores indicating better function, QoL

    • 3 symptom scales (fatigue, nausea and vomiting, pain)
    • 6 single items (dyspnoea, insomnia, appetite loss, constipation, diarrhoea, financial difficulties)
    • higher scores indicating higher level of symptoms

  7. Compare FACIT-Fatigue mean score with a matched case control group of MDS patients

    Time frame: one time at study entry

    To compare FACIT-Fatigue mean score with a matched case control group of MDS patients (0 to 52) --> higher scores indicating higher levels of fatigue.

Study contacts

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

Fabio Efficace

CONTACT

[email protected]

+39 06 441639831

Sponsors and collaborators

Lead sponsor

Gruppo Italiano Malattie EMatologiche dell'Adulto

Other

Registry information

Official study title

Health-Related Quality of Life and Financial Toxicity in Patients With VEXAS Syndrome: An Italian GIMEMA Study

Important dates

Study start
2024
Primary completion
2025
Study completion
2025
First posted
Oct 26, 2024
Registry last updated
Jul 15, 2025

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.

Related clinical trials

Published trials that share one or more normalized conditions with this study.