Transthyretin Amyloidosis Cardiomyopathy Clinical Trial1

Additional Analysis   >   Long-term analysis   >   Study Overview

Phase 3, multicenter, international, 3-arm, parallel-design, placebo-controlled, double-blind, randomized study in patients with ATTR-CM (N=441)1

Key inclusion criteria1
  • Patients aged ≥18 and ≤90 years with ATTR-CM
    (ATTRv-CM or ATTRwt-CM) and a medical history of
    heart failure
  • ≥1 prior hospitalization(s) due to heart failure, or
    clinical signs and symptoms associated with heart
    failure
  • End-diastolic intraventricular septal wall thickness
    >12 mm as seen in echocardiography
  • N-terminal pro-B-type natriuretic peptide (NT-
    proBNP) concentration ≥600 pg/mL
Key exclusion criteria1
  • Prior treatment with tafamidis
  • HF not due to ATTR-CM
  • New York Heart Association (NYHA) Class IV
  • Diagnosis of light chain amyloidosis
Patients with
ATTR-CM
(N=441)
Stratified by the presence or
absence of the variant TTR
genotype and by baseline
severity of the disease
(NYHA class)2
Randomization
2:1:2
Tafamidis meglumine
80 mg/day (N=176)
Tafamidis meglumine
20 mg/day (N=88)
Placebo
(N=177)
30-month
treatment
phase
Tafamidis meglumine reduced mortality and functional decline in patients with ATTR-CM over 30 months.1
• Patients who completed ATTR-ACT could enroll in the ongoing long-term extension (LTE) for up to 60 months10

ATTR-ACT: Transthyretin Amyloidosis Cardiomyopathy Clinical Trial; ATTR-CM: transthyretin amyloid cardiomyopathy; ATTRv-CM: hereditary transthyretin amyloid cardiomyopathy; ATTRwt-CM: wild-type transthyretin amyloid cardiomyopathy; LTE: long-term extension; NT-proBNP: N-terminal pro-B-type natriuretic peptide; NYHA: New York Heart Association; TTR: transthyretin.

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