ClinicalTrials.gov record NCT06657846

HRQoL and Financial Toxicity in Patients With VEXAS Syndrome

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
Age18 years old to 99 years old
SexAll
LocationItaly
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This multicenter, cross-sectional observational study collects questionnaire data from adults with confirmed VEXAS syndrome. It measures health-related quality of life, fatigue, symptoms, financial toxicity, and treatment tolerability. Findings will be compared with a matched group of myelodysplastic syndrome (MDS) patients to provide context. The study aims to establish benchmark HRQoL data to inform future studies and trial design.

What it is studying. The study is measuring how VEXAS affects patients' quality of life, symptom burden, fatigue, financial hardship, and reported tolerability of treatments. It will compare these patient-reported measures with a matched group of MDS patients to put results in context and help set benchmarks for future research.

What the researchers hope to learn. To describe health-related quality of life and symptom profiles in VEXAS, investigate financial toxicity and treatment tolerability, compare results with matched MDS patients, and create benchmark HRQoL data for future prospective studies and clinical trials.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants complete quality-of-life and symptom questionnaires one time at study entry (including EORTC QLQ-C30, FACIT-Fatigue and PROFFIT items). These ask about physical and emotional function, fatigue, symptoms, financial difficulties and treatment tolerability. The record lists questionnaires only; no invasive procedures are specified.

How the study is structured. Multicenter, cross-sectional observational cohort: participants complete quality-of-life, fatigue, symptom, and financial burden questionnaires once at study entry; those responses are analyzed and compared with a matched MDS control group. No experimental treatments are assigned.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in Italy. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on VEXAS syndrome

Clinical trials11 trials listed for VEXAS syndromeEvery trial the registry lists for VEXAS syndrome, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in VEXAS syndrome researchLatest monthly update: September 2026. Includes: IL-6 and JAK drugs. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Nov 1, 2024
Primary completion
Jun 10, 2025
Study completion
Sep 10, 2025 (estimated)
Target enrollment
100 participants (estimated)
Last updated on registry
Jul 15, 2025
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Gruppo Italiano Malattie EMatologiche dell'Adulto

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (17)

  • Aou Ospedali Riuniti "Umberto I - G.M. Lancisi - G. Salesi"- Ancona - Sod Clinica EmatologicaRecruiting

    Ancona · Italy

    • Erika Morsia
  • Asst Papa Giovanni Xxiii - Ospedale Di Bergamo - Sc EmatologiaRecruiting

    Bergamo · Italy

  • Aou Di Bologna - Policlinico S. Orsola-Malpighi - Uoc EmatologiaRecruiting

    Bologna · Italy

  • Asst Degli Spedali Civili Di Brescia - Uo EmatologiaRecruiting

    Brescia · Italy

  • Istituto Di Candiolo - Fondazione Del Piemonte Per L'Oncologia - Irccs - Centro TrapiantiRecruiting

    Candiolo · Italy

    • Elena Crisà
  • Aou Careggi - Firenze - Sod EmatologiaRecruiting

    Florence · Italy

  • Fondazione Irccs Ca' Granda, Ospedale Maggiore Policlinico - Milano - Ematologia - Padiglione MarcoraRecruiting

    Milan · Italy

    • Bruno Fattizzo
  • Irccs Ospedale S. Raffaele - Milano - Uo OncoematologiaRecruiting

    Milan · Italy

    • Elisa Diral
  • Aou Federico Ii - Napoli - Uoc EmatologiaRecruiting

    Napoli · Italy

  • Aou Di Padova - Uo EmatologiaRecruiting

    Padua · Italy

Terms used on this page · explanations by MedicalUpdates.ai

Written informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
cohort
A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
clinical trial
A study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

← Back to clinical trials