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Patient-Reported Outcome Measures in Wild-Type and Variant Cardiac Transthyretin Amyloidosis (ITALY)

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ClinicalTrials.gov Identifier: NCT04563286
Recruitment Status : Recruiting
First Posted : September 24, 2020
Last Update Posted : September 24, 2020
Sponsor:
Collaborators:
Fondazione Toscana Gabriele Monasterio
University of Pavia
Università degli Studi di Ferrara
Careggi Hospital
Information provided by (Responsible Party):
Gianluca Di Bella, University of Messina

Brief 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). 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).

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.


Condition or disease Intervention/treatment
Transthyretin Amyloidosis Other: questionnaire on life quality

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Study Type : Observational
Estimated Enrollment : 250 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Patient-Reported Outcome Measures in Wild-Type and Variant Cardiac Transthyretin Amyloidosis: The Impact of Transthyretin Amyloidosis on Life qualitY (ITALY) Study
Actual Study Start Date : February 22, 2020
Estimated Primary Completion Date : April 22, 2022
Estimated Study Completion Date : June 22, 2022



Intervention Details:
  • Other: questionnaire on life quality
    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.


Primary Outcome Measures :
  1. reliability [ Time Frame: baseline to 6 months ]
    internal consistency


Secondary Outcome Measures :
  1. responsiveness [ Time Frame: baseline to HF hospitalization (<6 months) ]
    changes in scores in response to clinical changes

  2. validity of each domain [ Time Frame: baseline to 6 months ]
    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)



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
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).

Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • Lack of informed consent.
  • Inability of understanding a written text in Italian.
  • Absence of the conditions of clinical stability, as defined above.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04563286


Locations
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Italy
Università di Ferrara Recruiting
Ferrara, Italy
Contact: Claudio Rapezzi    +390532293111    claudio.rapezzi@unife.it   
Careggi Hospital Recruiting
Firenze, Italy
Contact: Francesco Cappelli, MD    +39 055 794 9946    cappellifrancesco@inwind.it   
Contact: MD         
Sub-Investigator: Francesco Cappelli, MD         
Principal Investigator: Federico Perfetto         
Università di Messina Recruiting
Messina, Italy
Contact: Gianluca Di Bella    +393205506147    gianluca.dibella@unime.it   
Università di Pavia Recruiting
Pavia, Italy
Contact: Giovanni Palladini    +390382989898    giovanni.palladini@unipv.it   
Fondazione Toscana Gabriele Monasterio (FTGM) Recruiting
Pisa, Italy, 56124
Contact: Michele Emdin    +393454744053    emdin@ftgm.it   
Sub-Investigator: Claudio Passino, MD         
Sponsors and Collaborators
University of Messina
Fondazione Toscana Gabriele Monasterio
University of Pavia
Università degli Studi di Ferrara
Careggi Hospital
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Responsible Party: Gianluca Di Bella, Professor, University of Messina
ClinicalTrials.gov Identifier: NCT04563286    
Other Study ID Numbers: IT08012020
First Posted: September 24, 2020    Key Record Dates
Last Update Posted: September 24, 2020
Last Verified: September 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Gianluca Di Bella, University of Messina:
amyloidosis
transthyretin
quality of life
score
cardiomyopathy
Additional relevant MeSH terms:
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Amyloid Neuropathies, Familial
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Amyloid Neuropathies
Peripheral Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Amyloidosis, Familial
Metabolism, Inborn Errors