NatiOnal Referral cenTEr Study of Transthyretin Amyloid Cardiomyopathy(ATTR) Patients on Tafamidis (NOTE-ATTR)

NatiOnal Referral cenTEr Study of Transthyretin Amyloid Cardiomyopathy(ATTR) Patients on Tafamidis: Real World Prospective Study

All ATTRwt patients on tafamidis 61 mg treatment will be clinically evaluated before treatment initiation and subsequently every six months for the eligibility to continue tafamidis treatment, according to Italian Medicines Agency regulations. C onsidering the significant risk of developing heart rhythm disturbances due to cardiac amyloidosis, especially in transthyretin form (ATTRwt), in routine clinical practice a stricter heart rhythm monitoring is recommended in ATTRwt patients. Moreover, particular attention is usually paid for those who present atrio-ventricular and/or intraventricular block at the baseline electrocardiogram. Data about rhythm disturbances and diuretic dose need will be collected during the planned physical examination every six months and the Holter ECG monitoring requested by the physician at the end of every planned clinical evaluation.

Study Overview

• Status: Recruiting
• Conditions: Amyloidosis; Amyloidosis Cardiac; ATTR Amyloidosis Wild Type

• Study Type: Observational
• Enrollment (Estimated): 200

Contacts and Locations

Study Locations

• Italy
  • Pavia, Italy, 27100
    • Recruiting
    • Fondazione IRCCS Policlinico San Matteo
    • Contact: Paolo Milani, MD; Phone Number: +390382502994; Email: segreteria.amiloidosi@smatteo.pv.it

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

All patients with a diagnosis of ATTRwt disease and eligible for tafamidis treatment according to the national drug regulatory agency criteria and no prior history of atrial trachy-arrhythmia and/or bradyarrhythmias in their past medical history will undergo screening procedure to be enrolled in the present study.

Description

Inclusion Criteria:

• Diagnosis of ATTRwt amyloidosis.
• 18 years or older;
• evidence of cardiac involvement at echocardiography or cardiac MRI.
• No known prior history of atrial fibrillation or major bradyarrhythmia (second degree atrioventricular block, third-degree atrioventricular block, high-grade atrioventricular block or alternating right and left branch block)
• female patients who are postmenopausal for at least 1 year before the screening visit
• patients on therapy or candidates for therapy with tafamidis 61 mg
• voluntary written consent must be given before performance of any study-related procedure not part of standard medical care with the understanding that consent may be withdrawn by the patient at any time without prejudice to future medical care.

Exclusion Criteria:

• Non-ATTR amyloidosis;
• NYHA class III and IV;
• Pregnant or nursing women;
• Previous pacemaker implantation;
• any serious medical or psychiatric illness that could, in the investigator's opinion, potentially interfere with a fully comprehension of the written consent form

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cumulative incidence of atrial tachy-arrhythmia and brady-arrhythmias requiring pacemaker and/or defibrillator	All ATTRwt patients eligible for tafamidis with no prior history of atrial tachy-arrhythmia or brady-arrhythmias will be prospectively enrolled. The events (arrythmias incidence) will be collected by the usually rhythm monitoring that physician will recommend at the end of clinical evaluation according to clinical practice	18 months

Secondary Outcome Measures

Outcome Measure	Time Frame
patient diuretic dose need (i.e. Furosemide), reported as mg/Kg/die	18 months

Collaborators and Investigators

• Sponsor: Paolo Milani

Study record dates

Study Major Dates

• Study Start (Actual): January 26, 2024
• Primary Completion (Estimated): January 26, 2026
• Study Completion (Estimated): January 26, 2026

Study Registration Dates

• First Submitted: February 1, 2024
• First Submitted That Met QC Criteria: February 1, 2024
• First Posted (Actual): February 9, 2024

Study Record Updates

• Last Update Posted (Actual): March 7, 2024
• Last Update Submitted That Met QC Criteria: March 5, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Heart Diseases; Cardiovascular Diseases; Metabolic Diseases; Proteostasis Deficiencies; Amyloidosis; Cardiomyopathies
• Other Study ID Numbers: AC-022-IT

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No