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

Transthyretin Amyloidosis Clinical Trial

— ITALY  

Official title:

Patient-Reported Outcome Measures in Wild-Type and Variant Cardiac Transthyretin Amyloidosis: The Impact of Transthyretin Amyloidosis on Life qualitY (ITALY) Study

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04563286
• Other study ID #: IT08012020
• Status: Recruiting
• Start date: February 22, 2020
• Est. completion date: June 22, 2022

Study information

• Verified date: September 2020
• Source: University of Messina
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical 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). 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 250
• Est. completion date: June 22, 2022
• Est. primary completion date: April 22, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Amyloid Neuropathies, Familial
• Amyloidosis
• Transthyretin Amyloidosis

Intervention

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.

Locations

Country	Name	City	State
Italy	Università di Ferrara	Ferrara
Italy	Careggi Hospital	Firenze
Italy	Università di Messina	Messina
Italy	Università di Pavia	Pavia
Italy	Fondazione Toscana Gabriele Monasterio (FTGM)	Pisa

Sponsors (5)

Lead Sponsor	Collaborator
University of Messina	Careggi Hospital, Fondazione Toscana Gabriele Monasterio, Università degli Studi di Ferrara, University of Pavia

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	reliability	internal consistency	baseline to 6 months
Secondary	responsiveness	changes in scores in response to clinical changes	baseline to HF hospitalization (<6 months)
Secondary	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)	baseline to 6 months