Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy

Summary

This Phase 3 study will investigate the efficacy, safety and tolerability of an oral daily dose of 20 mg or 80 mg tafamidis meglumine capsules compared to placebo in subjects with either transthyretin genetic variants or wild-type transthyretin resulting in amyloid cardiomyopathy.

Timeline

Start

2013-12-09

Primary completion

2018-02-07

Completion

2018-02-07

Publications

• Damy T, Wang R, Maurer MS, Gillmore JD, Fontana M. Long-term efficacy of tafamidis in patients with transthyretin amyloid cardiomyopathy by National Amyloidosis Centre stage. Eur J Heart Fail. 2025 Dec;27(12):2998-3009. doi: 10.1002/ejhf.3696. Epub 2025 Jun 9.
• Grogan M, Davis MK, Crespo Leiro MG, Sultan MB, Gundapaneni B, Angeli FS, Hanna M. The effect of long-term tafamidis treatment on quality of life in people with transthyretin amyloid cardiomyopathy (ATTR-CM): A plain language summary. Future Cardiol. 2024;20(11-12):595-603. doi: 10.1080/14796678.2024.2391250. Epub 2024 Sep 11.
• Witteles R, Jefferies JL, Kapa S, Cappelli F, Sultan MB, Gundapaneni B, Davis MK, Garcia-Pavia P. Atrial Fibrillation as a Prognostic Factor for All-Cause Mortality in Patients With Transthyretin Amyloid Cardiomyopathy. JACC CardioOncol. 2024 Apr 30;6(4):592-598. doi: 10.1016/j.jaccao.2024.03.007. eCollection 2024 Aug.
• Hanna M, Fine NM, Gundapaneni B, Sultan MB, Witteles RM. Improvements in Efficacy Measures With Tafamidis in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial. JACC Adv. 2022 Dec 14;1(5):100148. doi: 10.1016/j.jacadv.2022.100148. eCollection 2022 Dec.
• Drachman B, Damy T, Hanna M, Wang R, Angeli FS, Garcia-Pavia P. Long-term tafamidis efficacy in patients with transthyretin amyloid cardiomyopathy by baseline left ventricular ejection fraction. Eur J Heart Fail. 2024 Sep;26(9):2038-2046. doi: 10.1002/ejhf.3330. Epub 2024 Jun 26.
• Sperry BW, Sultan MB, Gundapaneni B, Tai SS, Witteles RM. Effect of Tafamidis on Renal Function in Patients With Transthyretin Amyloid Cardiomyopathy in ATTR-ACT. JACC CardioOncol. 2024 Apr 16;6(2):300-306. doi: 10.1016/j.jaccao.2024.02.007. eCollection 2024 Apr.
• Shah SJ, Fine N, Garcia-Pavia P, Klein AL, Fernandes F, Weissman NJ, Maurer MS, Boman K, Gundapaneni B, Sultan MB, Elliott P. Effect of Tafamidis on Cardiac Function in Patients With Transthyretin Amyloid Cardiomyopathy: A Post Hoc Analysis of the ATTR-ACT Randomized Clinical Trial. JAMA Cardiol. 2024 Jan 1;9(1):25-34. doi: 10.1001/jamacardio.2023.4147.
• Garcia-Pavia P, Sultan MB, Gundapaneni B, Sekijima Y, Perfetto F, Hanna M, Witteles R. Tafamidis Efficacy Among Octogenarian Patients in the Phase 3 ATTR-ACT and Ongoing Long-Term Extension Study. JACC Heart Fail. 2024 Jan;12(1):150-160. doi: 10.1016/j.jchf.2023.08.032. Epub 2023 Nov 8.
• Elliott P, Gundapaneni B, Sultan MB, Ines M, Garcia-Pavia P. Improved long-term survival with tafamidis treatment in patients with transthyretin amyloid cardiomyopathy and severe heart failure symptoms. Eur J Heart Fail. 2023 Nov;25(11):2060-2064. doi: 10.1002/ejhf.2974. Epub 2023 Jul 26.
• Bhambri R, Colavecchia AC, Bruno M, Chen Y, Alvir J, Roy A, Kemner J, Crowley A, Benjumea D, Gilstrap L. Real-World Characteristics of Patients with Wild-Type Transthyretin Amyloid Cardiomyopathy: An Analysis of Electronic Healthcare Records in the United States. Am J Cardiovasc Drugs. 2023 Mar;23(2):197-206. doi: 10.1007/s40256-022-00563-4. Epub 2023 Feb 13.
• Sperry BW, Hanna M, Maurer MS, Nativi-Nicolau J, Floden L, Stewart M, Wyrwich KW, Barsdorf AI, Kapadia H, Spertus JA. Association of Tafamidis With Health Status in Patients With ATTR Cardiac Amyloidosis: A Post Hoc Analysis of the ATTR-ACT Randomized Clinical Trial. JAMA Cardiol. 2023 Mar 1;8(3):275-280. doi: 10.1001/jamacardio.2022.5251.
• Elliott P, Drachman BM, Gottlieb SS, Hoffman JE, Hummel SL, Lenihan DJ, Ebede B, Gundapaneni B, Li B, Sultan MB, Shah SJ. Long-term survival in people with transthyretin amyloid cardiomyopathy who took tafamidis: A Plain Language Summary. Future Cardiol. 2023 Jan;19(1):7-17. doi: 10.2217/fca-2022-0096. Epub 2023 Jan 30.
• Tess DA, Maurer TS, Li Z, Bulawa C, Fleming J, Moody AT. Relationship of binding-site occupancy, transthyretin stabilisation and disease modification in patients with tafamidis-treated transthyretin amyloid cardiomyopathy. Amyloid. 2023 Jun;30(2):208-219. doi: 10.1080/13506129.2022.2145876. Epub 2022 Nov 18.
• Maeda-Ogata S, Tahara N, Tahara A, Bekki M, Honda A, Sugiyama Y, Igata S, Abe T, Sekijima Y, Ueda M, Ando Y, Fukumoto Y. Treatment response to Tafamidis quantitatively assessed by serial bone scintigraphy in transthyretin amyloid cardiomyopathy. J Nucl Cardiol. 2023 Feb;30(1):403-404. doi: 10.1007/s12350-022-03137-8. Epub 2022 Nov 8. No abstract available.
• Rozenbaum MH, Tran D, Bhambri R, Nativi-Nicolau J. Annual Cardiovascular-Related Hospitalization Days Avoided with Tafamidis in Patients with Transthyretin Amyloid Cardiomyopathy. Am J Cardiovasc Drugs. 2022 Jul;22(4):445-450. doi: 10.1007/s40256-022-00526-9. Epub 2022 Mar 30.
• Nativi-Nicolau J, Judge DP, Hoffman JE, Gundapaneni B, Keohane D, Sultan MB, Grogan M. How did transthyretin amyloid cardiomyopathy progress in patients who took placebo in the study ATTR-ACT? A plain language summary. Future Cardiol. 2022 Mar 17. doi: 10.2217/fca-2021-0150. Online ahead of print.
• Elliott P, Drachman BM, Gottlieb SS, Hoffman JE, Hummel SL, Lenihan DJ, Ebede B, Gundapaneni B, Li B, Sultan MB, Shah SJ. Long-Term Survival With Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy. Circ Heart Fail. 2022 Jan;15(1):e008193. doi: 10.1161/CIRCHEARTFAILURE.120.008193. Epub 2021 Dec 20.
• Hanna M, Damy T, Grogan M, Stewart M, Gundapaneni B, Sultan MB, Maurer MS. Tafamidis and quality of life in people with transthyretin amyloid cardiomyopathy in the study ATTR-ACT: A plain language summary. Future Cardiol. 2022 Mar;18(3):165-172. doi: 10.2217/fca-2021-0095. Epub 2021 Nov 15.
• Rozenbaum MH, Garcia A, Grima D, Tran D, Bhambri R, Stewart M, Li B, Heeg B, Postma M, Masri A. Health impact of tafamidis in transthyretin amyloid cardiomyopathy patients: an analysis from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) and the open-label long-term extension studies. Eur Heart J Qual Care Clin Outcomes. 2022 Aug 17;8(5):529-538. doi: 10.1093/ehjqcco/qcab031.
• Vong C, Boucher M, Riley S, Harnisch LO. Modeling of Survival and Frequency of Cardiovascular-Related Hospitalization in Patients with Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis. Am J Cardiovasc Drugs. 2021 Sep;21(5):535-543. doi: 10.1007/s40256-021-00464-y. Epub 2021 Mar 26.
• Miller AB, Januzzi JL, O'Neill BJ, Gundapaneni B, Patterson TA, Sultan MB, Lopez-Sendon J. Causes of Cardiovascular Hospitalization and Death in Patients With Transthyretin Amyloid Cardiomyopathy (from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial [ATTR-ACT]). Am J Cardiol. 2021 Jun 1;148:146-150. doi: 10.1016/j.amjcard.2021.02.035. Epub 2021 Mar 3.
• Rapezzi C, Elliott P, Damy T, Nativi-Nicolau J, Berk JL, Velazquez EJ, Boman K, Gundapaneni B, Patterson TA, Schwartz JH, Sultan MB, Maurer MS. Efficacy of Tafamidis in Patients With Hereditary and Wild-Type Transthyretin Amyloid Cardiomyopathy: Further Analyses From ATTR-ACT. JACC Heart Fail. 2021 Feb;9(2):115-123. doi: 10.1016/j.jchf.2020.09.011. Epub 2020 Dec 9.
• Hanna M, Damy T, Grogan M, Stewart M, Gundapaneni B, Patterson TA, Schwartz JH, Sultan MB, Maurer MS. Impact of Tafamidis on Health-Related Quality of Life in Patients With Transthyretin Amyloid Cardiomyopathy (from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial). Am J Cardiol. 2021 Feb 15;141:98-105. doi: 10.1016/j.amjcard.2020.10.066. Epub 2020 Nov 19.
• Damy T, Garcia-Pavia P, Hanna M, Judge DP, Merlini G, Gundapaneni B, Patterson TA, Riley S, Schwartz JH, Sultan MB, Witteles R. Efficacy and safety of tafamidis doses in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) and long-term extension study. Eur J Heart Fail. 2021 Feb;23(2):277-285. doi: 10.1002/ejhf.2027. Epub 2020 Nov 12.
• Li B, Alvir J, Stewart M. Extrapolation of Survival Benefits in Patients with Transthyretin Amyloid Cardiomyopathy Receiving Tafamidis: Analysis of the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial. Cardiol Ther. 2020 Dec;9(2):535-540. doi: 10.1007/s40119-020-00179-2. Epub 2020 Jun 10.
• Maurer MS, Schwartz JH, Gundapaneni B, Elliott PM, Merlini G, Waddington-Cruz M, Kristen AV, Grogan M, Witteles R, Damy T, Drachman BM, Shah SJ, Hanna M, Judge DP, Barsdorf AI, Huber P, Patterson TA, Riley S, Schumacher J, Stewart M, Sultan MB, Rapezzi C; ATTR-ACT Study Investigators. Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy. N Engl J Med. 2018 Sep 13;379(11):1007-1016. doi: 10.1056/NEJMoa1805689. Epub 2018 Aug 27.
• Maurer MS, Elliott P, Merlini G, Shah SJ, Cruz MW, Flynn A, Gundapaneni B, Hahn C, Riley S, Schwartz J, Sultan MB, Rapezzi C; ATTR-ACT Study Investigators. Design and Rationale of the Phase 3 ATTR-ACT Clinical Trial (Tafamidis in Transthyretin Cardiomyopathy Clinical Trial). Circ Heart Fail. 2017 Jun;10(6):e003815. doi: 10.1161/CIRCHEARTFAILURE.116.003815.

Drugs

Indications