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

NCT06251778 | Recruiting

• 1 other identifier: AC-022-IT
• Study Type: observational
• Target: 200
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Amyloidosis; Amyloidosis Cardiac; ATTR Amyloidosis Wild Type

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• patient diuretic dose need (i.e. Furosemide), reported as mg/Kg/die

  18 months

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• Diagnosis of ATTRwt amyloidosis.
• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Paolo Milani: lead

Study Sites (1)

Fondazione IRCCS Policlinico San Matteo

Pavia, 27100, Italy

RECRUITING

MeSH Terms

Conditions

Amyloidosis; Amyloid Neuropathies, Familial

Condition Hierarchy (Ancestors)

Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Amyloid Neuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Amyloidosis, Familial; Metabolism, Inborn Errors

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: February 1, 2024
• First Posted: February 9, 2024
• Study Start: January 26, 2024
• Primary Completion (Estimated): January 26, 2027
• Study Completion (Estimated): January 26, 2027
• Last Updated: April 16, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1)