VEXASNCT06657846
HRQoL and Financial Toxicity in Patients With VEXAS Syndrome
An observational study of VEXAS, run by Gruppo Italiano Malattie EMatologiche dell'Adulto at 17 sites in Italy.
Trial summary
This multicenter cross-sectional observational study aims to describe health-related quality of life (HRQoL) and symptom profiles of patients with VEXAS syndrome. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- VEXAS
- How many people
- 100 participants
- Where
- 17 sitesItaly
- Study dates
- 1 Nov 2024to 10 Sep 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adult patients with a confirmed diagnosis of VEXAS syndrome
Who may be able to join
- Adult patients (≥ 18 years old) with a confirmed diagnosis of VEXAS syndrome (UBA1 pathogenic mutation detected)
- Written informed consent
Who cannot join
- - Having any kind of psychiatric disorder or major cognitive dysfunction.
- Not able to read and understand local language
Trial locations and contacts
17 sites in 1 country.
17 sites in 1 country
Italy17 sites, 17 recruiting
- Recruiting
Aou Ospedali Riuniti "Umberto I - G.M. Lancisi - G. Salesi"- Ancona - Sod Clinica Ematologica
Ancona
Erika Morsia
- Recruiting
Asst Papa Giovanni Xxiii - Ospedale Di Bergamo - Sc Ematologia
Bergamo
Alessandro Rambaldi, 3484526901, arambaldi@asst-pg23.it
- Recruiting
Aou Di Bologna - Policlinico S. Orsola-Malpighi - Uoc Ematologia
Bologna
Cristina Papayannidis, 3496484441, cristina.papayannidis@unibo.it
- Recruiting
Asst Degli Spedali Civili Di Brescia - Uo Ematologia
Brescia
Chiara Cattaneo, chiara.cattaneo@libero.it
- Recruiting
Istituto Di Candiolo - Fondazione Del Piemonte Per L'Oncologia - Irccs - Centro Trapianti
Candiolo
Elena Crisà
- Recruiting
Aou Careggi - Firenze - Sod Ematologia
Florence
Paola Guglielmelli, paola.guglielmelli@unifi.it
- Recruiting
Fondazione Irccs Ca' Granda, Ospedale Maggiore Policlinico - Milano - Ematologia - Padiglione Marcora
Milan
Bruno Fattizzo
- Recruiting
Irccs Ospedale S. Raffaele - Milano - Uo Oncoematologia
Milan
Elisa Diral
- Recruiting
Aou Federico Ii - Napoli - Uoc Ematologia
Naples
Fabrizio Pane, fabrizio.pane@unina.it
- Recruiting
Aou Di Padova - Uo Ematologia
Padova
Gianni Binotto, gianni.binotto@unipd.it
- Recruiting
Fondazione Ircss Policlinico San Matteo - Pavia - Uo Ematologia
Pavia
Luca Malcovati
- Recruiting
Ausl Di Reggio Emilia - Arcispedale Santa Maria Nuova, Irccs - Sc Ematologia
Reggio Emilia
Isabella Capodanno, isabella.capodanno@ausl.re.it
- Recruiting
Aou Policlinico Tor Vergata - Roma - Uoc Trapianto Cellule Staminali
Roma
Carmelo Gurnari
- Recruiting
Ististuto Clinico Humanitas - Rozzano - Uo Oncologia Medica Ed Ematologia
Rozzano
Gabriele Todisco
- Not yet recruiting
Ente Ecclesiastico Casa Sollievo Della Sofferenza - San Giovanni Rotondo - Ematologia
San Giovanni Rotondo
Angelo Michele Carella
- Recruiting
Ospedale Mauriziano Umberto I - Torino - Scdu Ematologia
Torino
Daniela Cilloni
- Recruiting
Aou Integrata Di Verona, Policlinico G.B. Rossi - Uoc Ematologia
Verona
Cinzia Sissa
About this study
Official title: 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.
Read the 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.
- Conditions
- VEXAS
- Medical subject headings
- VEXAS syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Quality of life assessment | Quality of life questionnaires |
What is being measured
The primary outcome is the main question the study is designed to answer.
EORTC QLQ-C30
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
Measured: one time at study entry
FACIT-Fatigue
Mean score of FACIT-Fatigue (0 to 52)
--> higher scores indicating higher levels of fatigue.
Measured: one time at study entry
Secondary outcomes (7)
PROFFIT-score
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)
Measured: 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
To assess the percentage of patients above previously validated cut-off scores for the EORTC QLQ-C30 indicating clinically relevant problems and symptoms
Measured: one time at study entry
EORTC QLQ-C30 mean scale score stratified by age groups and sex.
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
Measured: one time at study entry
FACIT-Fatigue mean score stratified by age groups and sex
To assess FACIT-Fatigue mean score stratified by age groups and sex. (0 to 52) --> higher scores indicating higher levels of fatigue.
Measured: one time at study entry
Score of the FACIT item GP5
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.
Measured: one time at study entry
Compare EORTC QLQ-C30 mean scale scores with a matched case control group of MDS patients
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
Measured: one time at study entry
Compare FACIT-Fatigue mean score with a matched case control group of MDS patients
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.
Measured: one time at study entry
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Oct 2024
- Study start1 Nov 2024
- Primary completion10 Jun 2025
- Study completion10 Sep 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for VEXASStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Gruppo Italiano Malattie EMatologiche dell'Adulto
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06657846
- Sponsor's protocol ID
- QoL-Vexas
- Status last verified
- Oct 2024
- First submitted
- 20 Jun 2024
- Last updated
- 15 Jul 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Health-Related Quality of Life and Financial Toxicity in Patients With VEXAS Syndrome: An Italian GIMEMA Study
- ClinicalTrials.gov ID
- NCT06657846
- Acronym / sponsor ID
- QoL-Vexas
- Conditions
- VEXAS
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Gruppo Italiano Malattie EMatologiche dell'AdultoUniversity, hospital or other
- Study start
- 1 Nov 2024 (actual)
- Primary completion
- 10 Jun 2025 (actual)
- Study completion
- 10 Sep 2025 (estimated)
- First posted
- 26 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This multicenter cross-sectional observational study aims to describe health-related quality of life (HRQoL) and symptom profiles of patients with VEXAS syndrome.
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 design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Quality of life assessment | – | –Quality of life questionnaires |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult patients with a confirmed diagnosis of VEXAS syndrome
Key inclusion criteria (2 listed)
- Adult patients (≥ 18 years old) with a confirmed diagnosis of VEXAS syndrome (UBA1 pathogenic mutation detected)
- Written informed consent
Key exclusion criteria (2 listed)
- - Having any kind of psychiatric disorder or major cognitive dysfunction.
- Not able to read and understand local language
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
FACIT-Fatigue
Time frame: one time at study entry
Mean score of FACIT-Fatigue (0 to 52) --> higher scores indicating higher levels of fatigue.
7 secondary outcomes
PROFFIT-score
Time frame: 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
EORTC QLQ-C30 mean scale score stratified by age groups and sex.
Time frame: one time at study entry
FACIT-Fatigue mean score stratified by age groups and sex
Time frame: one time at study entry
Score of the FACIT item GP5
Time frame: 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
Compare FACIT-Fatigue mean score with a matched case control group of MDS patients
Time frame: one time at study entry
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jul 2025.
Related trials for VEXAS
RecruitingPhase 2
A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)
VEXAS, VEXAS Syndrome
RecruitingPhase 2
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome
Immunodeficiency, Hematopoietic Stem Cell Transplantation
Enrolling by invitationObservational study
Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study
Bone Marrow Failure Disorders, VEXAS Syndrome
RecruitingPhase 2
A 52-Week Study Evaluating the Efficacy and Safety of Arumakimig (MAS825) in Participants With VEXAS Followed by Open-Label Extension (OLE) Period
VEXAS Syndrome
RecruitingPhase 2
Momelotinib in VEXAS Syndrome
VEXAS Syndome, Myelo Dysplastic Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
17 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of VEXAS, run by Gruppo Italiano Malattie EMatologiche dell'Adulto at 17 sites in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 15 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Fabio Efficace (+39 06 441639831, f.efficace@gimema.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
EORTC QLQ-C30 (time frame: one time at study entry). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jul 2025.