Efficacy and safety of tafamidis doses in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial ( ATTR‐ACT ) and long‐term extension study

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Key Points

To assess the efficacy and safety profiles of different tafamidis dosing regimens in patients with transthyretin amyloid cardiomyopathy across the pivotal trial and its extension phase.
Interventional multicenter evaluation analyzing participants from the double-blind randomized clinical trial ATTR-ACT (NCT01994889).
Followed patients receiving long-term treatment regimens in an open-label extension study (NCT02791230) to assess durability and safety.
Tafamidis administration demonstrated sustained clinical efficacy across evaluated doses with reductions in disease progression.
Safety and tolerability findings supported extended treatment durations without novel dose-limiting toxicities.

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Randomized trial demonstrates the efficacy and safety of tafamidis doses in transthyretin cardiomyopathy, indicating long-term therapeutic benefits.

ClinicalTrials.gov NCT01994889; NCT02791230.

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Damy et al. (2020) studied this question.

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