Transthyretin Amyloidosis Cardiomyopathy Clinical Trial (ATTR-ACT) - A Clinical Study in Patients with ATTR-CM1,2

Study Overview
ATTR-CM
patients
(N=441)
Wild-type (n=335)
Hereditary (n=106)

Stratified by presence or absence of variant TTR genotype and by baseline severity of disease (NYHA class).

Two-thirds of patients in the pooled tafamidis meglumine group were on the 80-mg dose.

Randomization
2:1:2
Tafamidis meglumine
80 mg/day (n=176)
Tafamidis meglumine
20 mg/day (n=88)
Placebo*
(n=177)
Pooled tafamidis
meglumine
(n=264)
Primary Analysis

A hierarchical combination of all-cause mortality and frequency of cardiovascular related (CV-related) hospitalizations†

Components of primary analysis for tafamidis meglumine vs. placebo:

  • All-cause mortality
  • Frequency of CV-related hospitalizations

Key Secondary
Endpoints
  • 6-minute walk test (6MWT)
  • Kansas City Cardiomyopathy Questionnaire-Overall Summary (KCCQ-OS)
30 months

ATTR-CM: Transthyretin amyloid cardiomyopathy; NYHA: New York Heart Association; TTR: Transthyretin.

*Standard of care (e.g., diuretics).
†The primary analysis was conducted using the Finkelstein-Schoenfeld method.