Patient-Reported Outcome Measures in Wild-Type & Variant Cardiac

Status Recruiting

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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04563286
Study type	Observational
Source	University of Messina
Contact
Status	Recruiting
Phase
Start date	February 22, 2020
Completion date	June 22, 2022

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT03190577` - Assessment of the Prevalence of TTR Amyloid Neuropathy in a Population of Patients With Neuropathy of Unknown Aetiology	N/A
Completed	`NCT03588468` - Expanding the Biomarkers in Familial Amyloid Neuropathy: MRI and Motor Unit Estimation by Electrophysiological Study	N/A
Completed	`NCT03591757` - Short-term Effects of TOLCAPONE on Transthyretin Stability in Subjects With Leptomeningeal TTR Amyloidosis (ATTR)	Early Phase 1
Completed	`NCT05075798` - Study of Cerebral MRI Anomalies in Mutated Transthyretin Amyloidosis Patients
Recruiting	`NCT05577819` - Prevalence and Prediction of ATTR in Ambulatory Patients With HFpEF	N/A
Terminated	`NCT04611204` - Transthyretin Cardiac Amyloidosis in Patients With Idiopathic Carpal Tunnel Syndrome Referred for Release Surgery
Recruiting	`NCT03352089` - Positron Emission Tomography / Magnetic Resonance Imaging in Aortic Stenosis	N/A
Recruiting	`NCT05814380` - The Regional Scintigraphic DPD Uptake in Cardiac Transthyretin Amyloidosis.
Completed	`NCT03923920` - Screening for Systemic Amyloidosis Via the Ligamentum Flavum
Completed	`NCT03886155` - Cardiac Amyloidosis Screening at Trigger Finger Release
Completed	`NCT03862807` - Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant	Phase 3
Completed	`NCT02792790` - Carpal Tunnel Syndrome and Amyloid Cardiomyopathy
Completed	`NCT03860935` - Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy	Phase 3
Recruiting	`NCT04963985` - The Effect of Tafamidis on Transthyretin Stabilization, Safety, Tolerability and Efficacy in Transthyretin Amyloid Polyneuropathy Patients	Phase 4
Recruiting	`NCT05409833` - Systemic Transthyretin Amyloidosis: Carpal Tunnel Syndrome in a Portuguese Population
Recruiting	`NCT05635045` - Evuzamitide in PET/CT to Measure Potential Therapeutic Response in ATTR	Phase 2
Completed	`NCT01171859` - Safety, Efficacy and Pharmacokinetics of Doxycycline Plus Tauroursodeoxycholic Acid in Transthyretin Amyloidosis	Phase 2
Recruiting	`NCT03237494` - TRAMmoniTTR Study Genetic Screening of an At-risk Population for hATTR and Monitoring of TTR Positive Subjects
Recruiting	`NCT04899180` - Prevalence of Transthyretin Cardiac Amyloidosis in Clinically Significant Aortic Stenosis	Early Phase 1
Recruiting	`NCT06345235` - New Biomarkers and Plasma Prothrombotic Potential in Cardiac Transthyretin Amyloidosis

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

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

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms