HRQoL and Financial Toxicity in Patients With VEXAS Syndrome

July 14, 2025 updated by: Gruppo Italiano Malattie EMatologiche dell'Adulto

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

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

Study Overview

Status

Recruiting

Conditions

VEXAS

Detailed Description

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.

Study Type

Observational

Enrollment (Estimated)

100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Name: Fabio Efficace
Phone Number: +39 06 441639831
Email: f.efficace@gimema.it

Study Locations

Italy
- - Ancona, Italy
    - Recruiting
    - Aou Ospedali Riuniti "Umberto I - G.M. Lancisi - G. Salesi"- Ancona - Sod Clinica Ematologica
    - Contact:
      
      Erika Morsia
  - Bergamo, Italy
    - Recruiting
    - Asst Papa Giovanni Xxiii - Ospedale Di Bergamo - Sc Ematologia
    - Contact:
      
      Alessandro Rambaldi
      
      Phone Number: 3484526901
      
      Email: arambaldi@asst-pg23.it
  - Bologna, Italy
    - Recruiting
    - AOU di Bologna - Policlinico S. Orsola-Malpighi - UOC Ematologia
    - Contact:
      
      Cristina Papayannidis
      
      Phone Number: 3496484441
      
      Email: cristina.papayannidis@unibo.it
  - Brescia, Italy
    - Recruiting
    - Asst Degli Spedali Civili Di Brescia - Uo Ematologia
    - Contact:
      
      Chiara Cattaneo
      
      Email: chiara.cattaneo@libero.it
  - Candiolo, Italy
    - Recruiting
    - Istituto Di Candiolo - Fondazione Del Piemonte Per L'Oncologia - Irccs - Centro Trapianti
    - Contact:
      
      Elena Crisà
  - Firenze, Italy
    - Recruiting
    - AOU Careggi - Firenze - SOD Ematologia
    - Contact:
      
      Paola Guglielmelli
      
      Email: paola.guglielmelli@unifi.it
  - Milano, Italy
    - Recruiting
    - Irccs Ospedale S. Raffaele - Milano - Uo Oncoematologia
    - Contact:
      
      Elisa Diral
  - Milano, Italy
    - Recruiting
    - Fondazione Irccs Ca' Granda, Ospedale Maggiore Policlinico - Milano - Ematologia - Padiglione Marcora
    - Contact:
      
      Bruno Fattizzo
  - Napoli, Italy
    - Recruiting
    - Aou Federico Ii - Napoli - Uoc Ematologia
    - Contact:
      
      Fabrizio Pane
      
      Email: fabrizio.pane@unina.it
  - Padova, Italy
    - Recruiting
    - AOU di Padova - UO Ematologia
    - Contact:
      
      Gianni Binotto
      
      Email: gianni.binotto@unipd.it
  - Pavia, Italy
    - Recruiting
    - Fondazione Ircss Policlinico San Matteo - Pavia - Uo Ematologia
    - Contact:
      
      Luca Malcovati
  - Reggio Emilia, Italy
    - Recruiting
    - Ausl Di Reggio Emilia - Arcispedale Santa Maria Nuova, Irccs - Sc Ematologia
    - Contact:
      
      Isabella Capodanno
      
      Email: isabella.capodanno@ausl.re.it
  - Roma, Italy
    - Recruiting
    - Aou Policlinico Tor Vergata - Roma - Uoc Trapianto Cellule Staminali
    - Contact:
      
      Carmelo Gurnari
  - Rozzano, Italy
    - Recruiting
    - Ististuto Clinico Humanitas - Rozzano - Uo Oncologia Medica Ed Ematologia
    - Contact:
      
      Gabriele Todisco
  - San Giovanni Rotondo, Italy
    - Not yet recruiting
    - Ente Ecclesiastico Casa Sollievo Della Sofferenza - San Giovanni Rotondo - Ematologia
    - Contact:
      
      Angelo Michele Carella
  - Torino, Italy
    - Recruiting
    - Ospedale Mauriziano Umberto I - Torino - Scdu Ematologia
    - Contact:
      
      Daniela Cilloni
  - Verona, Italy
    - Recruiting
    - Aou Integrata Di Verona, Policlinico G.B. Rossi - Uoc Ematologia
    - Contact:
      
      Cinzia Sissa

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

Adult
Older Adult

Accepts Healthy Volunteers

Sampling Method

Non-Probability Sample

Study Population

Adult patients with a confirmed diagnosis of VEXAS syndrome

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Number of groups / cohorts

Cohorts and Interventions

Group / Cohort
Quality of life assessment Quality of life questionnaires

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	one time at study entry
FACIT-Fatigue Time Frame: one time at study entry	Mean score of FACIT-Fatigue (0 to 52) --> higher scores indicating higher levels of fatigue.	one time at study entry

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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)	one time at study entry
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	one time at study entry
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	one time at study entry
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.	one time at study entry
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.	one time at study entry
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	one time at study entry
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.	one time at study entry

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Gruppo Italiano Malattie EMatologiche dell'Adulto

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 1, 2024

Primary Completion (Actual)

June 10, 2025

Study Completion (Estimated)

September 10, 2025

Study Registration Dates

First Submitted

June 20, 2024

First Submitted That Met QC Criteria

October 23, 2024

First Posted (Actual)

October 26, 2024

Study Record Updates

Last Update Posted (Actual)

July 15, 2025

Last Update Submitted That Met QC Criteria

July 14, 2025

Last Verified

October 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

QoL-Vexas

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Studies a U.S. FDA-regulated device product

product manufactured in and exported from the U.S.

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.