HRQoL and Financial Toxicity in Patients With VEXAS Syndrome

NCT06657846 · tracked via the Priya Life Science Italy tracker

Sponsor

Gruppo Italiano Malattie EMatologiche dell'Adulto

Phase

Observational

Started

2024-11-01

Last updated

2025-07-15

Condition(s) studied

VEXAS

Study summary

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

Eligibility

Sex

ALL

Min age

18 Years

Max age

99 Years

Healthy volunteers

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

Primary outcome measure(s)

EORTC QLQ-C30 — 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
FACIT-Fatigue — one time at study entry
Mean score of FACIT-Fatigue (0 to 52) --\> higher scores indicating higher levels of fatigue.

Trial sites (17)

Facility	City	Region	Status
Aou Ospedali Riuniti "Umberto I - G.M. Lancisi - G. Salesi"- Ancona - Sod Clinica Ematologica	Ancona	Italy	Recruiting
Asst Papa Giovanni Xxiii - Ospedale Di Bergamo - Sc Ematologia	Bergamo	Italy	Recruiting
Aou Di Bologna - Policlinico S. Orsola-Malpighi - Uoc Ematologia	Bologna	Italy	Recruiting
Asst Degli Spedali Civili Di Brescia - Uo Ematologia	Brescia	Italy	Recruiting
Istituto Di Candiolo - Fondazione Del Piemonte Per L'Oncologia - Irccs - Centro Trapianti	Candiolo	Italy	Recruiting
Aou Careggi - Firenze - Sod Ematologia	Florence	Italy	Recruiting
Fondazione Irccs Ca' Granda, Ospedale Maggiore Policlinico - Milano - Ematologia - Padiglione Marcora	Milan	Italy	Recruiting
Irccs Ospedale S. Raffaele - Milano - Uo Oncoematologia	Milan	Italy	Recruiting
Aou Federico Ii - Napoli - Uoc Ematologia	Naples	Italy	Recruiting
Aou Di Padova - Uo Ematologia	Padova	Italy	Recruiting
Fondazione Ircss Policlinico San Matteo - Pavia - Uo Ematologia	Pavia	Italy	Recruiting
Ausl Di Reggio Emilia - Arcispedale Santa Maria Nuova, Irccs - Sc Ematologia	Reggio Emilia	Italy	Recruiting
Aou Policlinico Tor Vergata - Roma - Uoc Trapianto Cellule Staminali	Roma	Italy	Recruiting
Ististuto Clinico Humanitas - Rozzano - Uo Oncologia Medica Ed Ematologia	Rozzano	Italy	Recruiting
Ente Ecclesiastico Casa Sollievo Della Sofferenza - San Giovanni Rotondo - Ematologia	San Giovanni Rotondo	Italy	Not Yet Recruiting
Ospedale Mauriziano Umberto I - Torino - Scdu Ematologia	Torino	Italy	Recruiting
Aou Integrata Di Verona, Policlinico G.B. Rossi - Uoc Ematologia	Verona	Italy	Recruiting

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Other trials for the same condition

A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS) Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06657846 on ClinicalTrials.gov ↗ ← All trials in Italy