Transthyretin AmyloidosisITALYNCT04563286
Patient-Reported Outcome Measures in Wild-Type and Variant Cardiac Transthyretin Amyloidosis
An observational study of Transthyretin Amyloidosis, run by University of Messina at 5 sites in Italy.
Trial summary
Specific, standardized, comprehensive, universally accepted Patient-Reported Outcome Measures (PROMs) are currently lacking for variant and wild-type cardiac amyloid transthyretin amyloidosis (v-ATTR/wt-ATTR). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Transthyretin Amyloidosis
- What is being tested?
- questionnaire on life quality
- How many people
- 250 participants
- Where
- 5 sitesItaly
- Study dates
- 22 Feb 2020to 22 Jun 2022
- Phase
- Observational
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with cardiac ATTR amyloidosis, either biopsy proven or diagnosed according to the algorithm for nonbiopsy diagnosis of ATTR cardiomyopathy (Gillmore et al., 2016), will be evaluated. PROMs for v-ATTR and wt-ATTR will be created in close collaboration with 50 patients suffering from ATTR cardiomyopathy (see below), and will be validated on a cohort of 250 patients (i.e., 50 patients from each participating center; see below).
Who may be able to join
- Diagnosis of cardiomyopathy due to ATTR amyloidosis, diagnosed by endomyocardial biopsy or on the basis of the algorithm for the non-invasive diagnosis of cardiac ATTR amyloidosis (Gillmore et al., 2016).
- Clinical stability, defined as the lack of unscheduled hospitalizations and/or significant changes in cardiac therapies from at least 1 month.
Who cannot join
- Lack of informed consent.
- Inability of understanding a written text in Italian.
- Absence of the conditions of clinical stability, as defined above.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
Italy5 sites, 5 recruiting
- Recruiting
Università di Ferrara
Ferrara
Claudio Rapezzi, +390532293111, claudio.rapezzi@unife.it
- Recruiting
Careggi Hospital
Florence
Francesco Cappelli, MD, +39 055 794 9946, cappellifrancesco@inwind.it
MD
- Recruiting
Università di Messina
Messina
Gianluca Di Bella, +393205506147, gianluca.dibella@unime.it
- Recruiting
Università di Pavia
Pavia
Giovanni Palladini, +390382989898, giovanni.palladini@unipv.it
- Recruiting
Fondazione Toscana Gabriele Monasterio (FTGM)
Pisa, 56124
Michele Emdin, +393454744053, emdin@ftgm.it
Claudio Passino, MD
About this study
Official title: Patient-Reported Outcome Measures in Wild-Type and Variant Cardiac Transthyretin Amyloidosis: The Impact of Transthyretin Amyloidosis on Life qualitY (ITALY) Study
Specific, standardized, comprehensive, universally accepted Patient-Reported Outcome Measures (PROMs) are currently lacking for variant and wild-type cardiac amyloid transthyretin amyloidosis (v-ATTR/wt-ATTR). Our goal is then to create two scores able to provide a cumulative assessment of cardiac involvement, peripheral neuropathy (in v-ATTR), and comorbidities, and their impact on the quality of life.
In the setting of a nationwide collaboration involving 5 main Italian referral centers for this condition (in Ferrara, Florence, Pavia, Pisa and Messina), a panel will be created, including experts of ATTR cardiomyopathy, neurologists, geriatricians, health management specialists, as well as patients with either variant or wild-type ATTR cardiomyopathy (n=50).
Read more
The most clinically relevant domains for patients (such as physical limitations, symptoms, self-efficacy and knowledge, social interference, quality of life, age-related issues, social and family environment, frailty, comorbidities) will be identified. Two sets of 30 items (one for variant and another for wild-type ATTR cardiomyopathy) will be created in collaboration with patients. Questions will be formatted for gender neutrality, clarity, interpretability, and possible foreign language translations. PROMs scores will be validated through administration to around 250 consecutive outpatients. Score performance will be evaluated in terms of internal consistency, response to clinical changes, comparison with conventional clinical measures. The time needed for completion, the clarity of questions and the need for assistance from a family caregiver will be evaluated.
This project will hopefully lead to the identification of disease-specific metrics that may serve as a clinically meaningful outcome in cardiovascular research, patient management, and quality assessment.
- Conditions
- Transthyretin Amyloidosis
- Being tested
- questionnaire on life quality
- Keywords
- amyloidosis, transthyretin, quality of life, score, cardiomyopathy
- Medical subject headings
- Amyloidosis, Hereditary, Transthyretin-Related, Amyloidosis, Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
questionnaire on life qualityOther
Two sets of 30 PROMs questions will be created. The v-ATTR and wt-ATTR questionnaires will be critically evaluated by the whole panel, taking into account observations and feedback from patients. The 2 scores will be administered to consecutive patients evaluated at dedicated ambulatory clinics of the 4 Institutions. To confirm score reliability and responsiveness, 2 distinct patient cohorts will be recruited. The reliability cohort will be assembled to demonstrate the instrument's test-retest reliability. A second cohort of patients (responsiveness cohort) will be assembled to demonstrate the instrument's responsiveness to changes in clinical status. Patients experiencing a heart failure hospitalization within 6 months will enter the responsiveness cohort at the time of hospital admission.
What is being measured
The primary outcome is the main question the study is designed to answer.
reliability
internal consistency
Measured: baseline to 6 months
Secondary outcomes (2)
responsiveness
changes in scores in response to clinical changes
Measured: baseline to HF hospitalization (<6 months)
validity of each domain
comparison of scores with other measures that quantify similar concepts, namely other score points, NYHA class, 6MWD, or objective measures of cardiac dysfunction, i.e. circulating levels of NT-proBNP and hs-TnT)
Measured: baseline to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Sep 2020
- Study start22 Feb 2020
- Primary completion22 Apr 2022 (estimated)
- Study completion22 Jun 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transthyretin AmyloidosisStudies 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
- Prospective
- Enrolment
- 250 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Messina
- Sponsor type
- University, hospital or other
- Collaborators
- Fondazione Toscana Gabriele Monasterio, University of Pavia, Università degli Studi di Ferrara, Careggi Hospital
- Responsible party
- Principal investigator, Gianluca Di Bella, University of Messina
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04563286
- Sponsor's protocol ID
- IT08012020
- Acronym
- ITALY
- Status last verified
- Sep 2020
- First submitted
- 20 Sep 2020
- Last updated
- 24 Sep 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided