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Study Design

ATTR-ACT - a clinical study in patients with ATTR-CM4
A phase 3, multicenter, international, randomized, double-blind, placebo-controlled study, which evaluated pooled VyndamaxTM doses of 20mg and 80mg in 441 patients with wild-type or hereditary ATTR-CM.
  • Two-thirds of patients in the pooled tafamidis group were on the 80mg dose.
    Inclusion criteria4
    • Patients between 18 and 90 years of age
    • Confirmation of ATTR-CM, wild-type or hereditary
    • Echocardiography with an end-diastolic interventricular septal wall thickness>12mm
    • History of heart failure with at least 1 prior hospitalization for heart failure, or clinical evidence of heart failure (without hospitalization)§
    • NT-proBNP level ≥600pg/mL
    • 6MWT >100m
    Exclusion criteria4
    • Heart failure unrelated to ATTR-CM
    • NYHA class IV heart failure
    • Light-chain amyloidosis
    • History of liver or heart transplantation
    • Implanted cardiac mechanical assist device||
    • eGFR <25mL/min/1.73m
    • Liver transaminase levels >2x the upper limit of normal
    • mBMI <600
    • Concurrent treatment with NSAIDs, tauroursodeoxycholate, doxycycline, calcium channel blockers, or digitalis
    As determined by the predefined 90% confidence interval criteria of 80%-125% bioequivalence limits for tafamidis AUC and Cmax after repeated oral daily dosing for 7 days.6
    Standard of care (e.g. diuretics).
    ‡ The primary analysis was conducted using the Finkelstein-Schoenfeld method.
    § Manifested in signs or symptoms of volume overload or elevated intracardiac pressures requiring treatment with a diuretic for improvement; an NT-proBNP level greater than or equal to 600pg per milliliter; and a 6-minute walk-test distance exceeding 100m.
    || Patients with a permanent pacemaker or implanted cardiac defibrillator were permitted in this study.7
    ¶ Select NSAIDs were permitted, e.g., aspirin, ibuprofen, naproxen.