메뉴 건너뛰기




Volumn 10, Issue 6, 2017, Pages

Design and Rationale of the Phase 3 ATTR-ACT Clinical Trial (Tafamidis in Transthyretin Cardiomyopathy Clinical Trial)

Author keywords

amyloid; cardiomyopathies; double blind method; heart failure; mutation

Indexed keywords

AMYLOID; BRAIN NATRIURETIC PEPTIDE; DIFLUNISAL; PROTON PUMP INHIBITOR; TAFAMIDIS; TECHNETIUM 99M; BENZOXAZOLE DERIVATIVE;

EID: 85021084170     PISSN: 19413289     EISSN: 19413297     Source Type: Journal    
DOI: 10.1161/CIRCHEARTFAILURE.116.003815     Document Type: Article
Times cited : (65)

References (53)
  • 2
    • 84864674714 scopus 로고    scopus 로고
    • Prospective evaluation of the morbidity and mortality of wild-type and V122I mutant transthyretin amyloid cardiomyopathy: The Transthyretin Amyloidosis Cardiac Study (TRACS)
    • e1
    • Ruberg FL, Maurer MS, Judge DP, Zeldenrust S, Skinner M, Kim AY, Falk RH, Cheung KN, Patel AR, Pano A, Packman J, Grogan DR. Prospective evaluation of the morbidity and mortality of wild-type and V122I mutant transthyretin amyloid cardiomyopathy: the Transthyretin Amyloidosis Cardiac Study (TRACS). Am Heart J. 2012;164:222-228. e1. doi: 10. 1016/j. ahj. 2012. 04. 015.
    • (2012) Am Heart J. , vol.164 , pp. 222-228
    • Ruberg, F.L.1    Maurer, M.S.2    Judge, D.P.3    Zeldenrust, S.4    Skinner, M.5    Kim, A.Y.6    Falk, R.H.7    Cheung, K.N.8    Patel, A.R.9    Pano, A.10    Packman, J.11    Grogan, D.R.12
  • 3
    • 84955747183 scopus 로고    scopus 로고
    • Heart failure resulting from age-related cardiac amyloid disease associated with wild-type transthyretin: A prospective, observational cohort study
    • Connors LH, Sam F, Skinner M, Salinaro F, Sun F, Ruberg FL, Berk JL, Seldin DC. Heart failure resulting from age-related cardiac amyloid disease associated with wild-type transthyretin: a prospective, observational cohort study. Circulation. 2016;133:282-290. doi: 10. 1161/ circulationaha. 115. 018852.
    • (2016) Circulation. , vol.133 , pp. 282-290
    • Connors, L.H.1    Sam, F.2    Skinner, M.3    Salinaro, F.4    Sun, F.5    Ruberg, F.L.6    Berk, J.L.7    Seldin, D.C.8
  • 6
    • 0017824077 scopus 로고
    • Structure of prealbumin: Secondary, tertiary and quaternary interactions determined by Fourier refinement at 1. 8 A
    • Blake CC, Geisow MJ, Oatley SJ, Rérat B, Rérat C. Structure of prealbumin: secondary, tertiary and quaternary interactions determined by Fourier refinement at 1. 8 A. J Mol Biol. 1978;121:339-356.
    • (1978) J Mol Biol. , vol.121 , pp. 339-356
    • Blake, C.C.1    Geisow, M.J.2    Oatley, S.J.3    Rérat, B.4    Rérat, C.5
  • 7
    • 0029062667 scopus 로고
    • Structure of a complex of two plasma proteins: Transthyretin and retinol-binding protein
    • Monaco HL, Rizzi M, Coda A. Structure of a complex of two plasma proteins: transthyretin and retinol-binding protein. Science. 1995;268:1039-1041.
    • (1995) Science. , vol.268 , pp. 1039-1041
    • Monaco, H.L.1    Rizzi, M.2    Coda, A.3
  • 8
    • 84861421529 scopus 로고    scopus 로고
    • The transthyretin amyloidoses: From delineating the molecular mechanism of aggregation linked to pathology to a regulatory-agency-approved drug
    • Johnson SM, Connelly S, Fearns C, Powers ET, Kelly JW. The transthyretin amyloidoses: from delineating the molecular mechanism of aggregation linked to pathology to a regulatory-agency-approved drug. J Mol Biol. 2012;421:185-203. doi: 10. 1016/j. jmb. 2011. 12. 060.
    • (2012) J Mol Biol. , vol.421 , pp. 185-203
    • Johnson, S.M.1    Connelly, S.2    Fearns, C.3    Powers, E.T.4    Kelly, J.W.5
  • 9
    • 0035920156 scopus 로고    scopus 로고
    • Tetramer dissociation and monomer partial unfolding precedes protofibril formation in amyloidogenic transthyretin variants
    • Quintas A, Vaz DC, Cardoso I, Saraiva MJ, Brito RM. Tetramer dissociation and monomer partial unfolding precedes protofibril formation in amyloidogenic transthyretin variants. J Biol Chem. 2001;276:27207-27213. doi: 10. 1074/jbc. M101024200.
    • (2001) J Biol Chem. , vol.276 , pp. 27207-27213
    • Quintas, A.1    Vaz, D.C.2    Cardoso, I.3    Saraiva, M.J.4    Brito, R.M.5
  • 10
    • 0035018450 scopus 로고    scopus 로고
    • Transthyretin mutations in hyperthyroxinemia and amyloid diseases
    • Saraiva MJ. Transthyretin mutations in hyperthyroxinemia and amyloid diseases. Hum Mutat. 2001;17:493-503. doi: 10. 1002/humu. 1132.
    • (2001) Hum Mutat. , vol.17 , pp. 493-503
    • Saraiva, M.J.1
  • 12
    • 84940703039 scopus 로고    scopus 로고
    • Transthyretin (ATTR) amyloidosis: Clinical spectrum, molecular pathogenesis and disease-modifying treatments
    • Sekijima Y. Transthyretin (ATTR) amyloidosis: clinical spectrum, molecular pathogenesis and disease-modifying treatments. J Neurol Neurosurg Psychiatry. 2015;86:1036-1043. doi: 10. 1136/jnnp-2014-308724.
    • (2015) J Neurol Neurosurg Psychiatry. , vol.86 , pp. 1036-1043
    • Sekijima, Y.1
  • 14
    • 84871383407 scopus 로고    scopus 로고
    • THAOS-The Transthyretin Amyloidosis Outcomes Survey: Initial report on clinical manifestations in patients with hereditary and wild-type transthyretin amyloidosis
    • Coelho T, Maurer MS, Suhr OB. THAOS-The Transthyretin Amyloidosis Outcomes Survey: initial report on clinical manifestations in patients with hereditary and wild-type transthyretin amyloidosis. Curr Med Res Opin. 2013;29:63-76. doi: 10. 1185/03007995. 2012. 754348.
    • (2013) Curr Med Res Opin. , vol.29 , pp. 63-76
    • Coelho, T.1    Maurer, M.S.2    Suhr, O.B.3
  • 16
    • 0242407579 scopus 로고    scopus 로고
    • Tabulation of human transthyretin (TTR) variants, 2003
    • Connors LH, Lim A, Prokaeva T, Roskens VA, Costello CE. Tabulation of human transthyretin (TTR) variants, 2003. Amyloid. 2003;10:160-184. doi: 10. 3109/13506120308998998.
    • (2003) Amyloid. , vol.10 , pp. 160-184
    • Connors, L.H.1    Lim, A.2    Prokaeva, T.3    Roskens, V.A.4    Costello, C.E.5
  • 17
    • 34848818004 scopus 로고    scopus 로고
    • The molecular biology and clinical features of amyloid neuropathy
    • Benson MD, Kincaid JC. The molecular biology and clinical features of amyloid neuropathy. Muscle Nerve. 2007;36:411-423. doi: 10. 1002/ mus. 20821.
    • (2007) Muscle Nerve. , vol.36 , pp. 411-423
    • Benson, M.D.1    Kincaid, J.C.2
  • 19
    • 84940036342 scopus 로고    scopus 로고
    • Prevalence of the amyloidogenic transthyretin (TTR) V122I allele in 14, 333 African-Americans
    • Jacobson DR, Alexander AA, Tagoe C, Buxbaum JN. Prevalence of the amyloidogenic transthyretin (TTR) V122I allele in 14, 333 African-Americans. Amyloid. 2015;22:171-174. doi: 10. 3109/13506129. 2015. 1051219.
    • (2015) Amyloid. , vol.22 , pp. 171-174
    • Jacobson, D.R.1    Alexander, A.A.2    Tagoe, C.3    Buxbaum, J.N.4
  • 21
    • 84859853882 scopus 로고    scopus 로고
    • Transthyretin cardiac amyloidoses in older North Americans
    • Dharmarajan K, Maurer MS. Transthyretin cardiac amyloidoses in older North Americans. J Am Geriatr Soc. 2012;60:765-774. doi: 10. 1111/j. 1532-5415. 2011. 03868. x.
    • (2012) J Am Geriatr Soc. , vol.60 , pp. 765-774
    • Dharmarajan, K.1    Maurer, M.S.2
  • 24
    • 84865849112 scopus 로고    scopus 로고
    • Transthyretin (TTR) cardiac amyloidosis
    • Ruberg FL, Berk JL. Transthyretin (TTR) cardiac amyloidosis. Circulation. 2012;126:1286-1300. doi: 10. 1161/circulationaha. 111. 078915.
    • (2012) Circulation. , vol.126 , pp. 1286-1300
    • Ruberg, F.L.1    Berk, J.L.2
  • 25
    • 70349210200 scopus 로고    scopus 로고
    • Cardiac amyloidosis in African Americans: Comparison of clinical and laboratory features of transthyretin V122I amyloidosis and immunoglobulin light chain amyloidosis
    • Connors LH, Prokaeva T, Lim A, Théberge R, Falk RH, Doros G, Berg A, Costello CE, O'Hara C, Seldin DC, Skinner M. Cardiac amyloidosis in African Americans: comparison of clinical and laboratory features of transthyretin V122I amyloidosis and immunoglobulin light chain amyloidosis. Am Heart J. 2009;158:607-614. doi: 10. 1016/j. ahj. 2009. 08. 006.
    • (2009) Am Heart J. , vol.158 , pp. 607-614
    • Connors, L.H.1    Prokaeva, T.2    Lim, A.3    Théberge, R.4    Falk, R.H.5    Doros, G.6    Berg, A.7    Costello, C.E.8    O'Hara, C.9    Seldin, D.C.10    Skinner, M.11
  • 28
    • 84877325205 scopus 로고    scopus 로고
    • (99m)Tc-pyrophosphate scintigraphy for differentiating light-chain cardiac amyloidosis from the transthyretin-related familial and senile cardiac amyloidoses
    • Bokhari S, Castaño A, Pozniakoff T, Deslisle S, Latif F, Maurer MS. (99m)Tc-pyrophosphate scintigraphy for differentiating light-chain cardiac amyloidosis from the transthyretin-related familial and senile cardiac amyloidoses. Circ Cardiovasc Imaging. 2013;6:195-201. doi: 10. 1161/circimaging. 112. 000132.
    • (2013) Circ Cardiovasc Imaging. , vol.6 , pp. 195-201
    • Bokhari, S.1    Castaño, A.2    Pozniakoff, T.3    Deslisle, S.4    Latif, F.5    Maurer, M.S.6
  • 30
    • 85002862397 scopus 로고    scopus 로고
    • Can 99m-Tc-Pyrophosphate aid in early detection of cardiac involvement in asymptomatic variant TTR amyloidosis? [published online ahead of print August 20, 2016]
    • Haq M, Pawar S, Berk JL, Miller EJ, Ruberg FL. Can 99m-Tc-Pyrophosphate aid in early detection of cardiac involvement in asymptomatic variant TTR amyloidosis? [published online ahead of print August 20, 2016]. JACC Cardiovasc Imaging. doi: 10. 1016/j. jcmg. 2016. 06. 003.
    • JACC Cardiovasc Imaging
    • Haq, M.1    Pawar, S.2    Berk, J.L.3    Miller, E.J.4    Ruberg, F.L.5
  • 31
    • 79959982521 scopus 로고    scopus 로고
    • Clinical features and survival in senile systemic amyloidosis: Comparison to familial transthyretin cardiomyopathy
    • Connors LH, Doros G, Sam F, Badiee A, Seldin DC and Skinner M. Clinical features and survival in senile systemic amyloidosis: comparison to familial transthyretin cardiomyopathy. Amyloid. 2011;18(suppl 1):157-159. doi: 10. 3109/13506129. 2011. 574354059.
    • (2011) Amyloid. , vol.18 , pp. 157-159
    • Connors, L.H.1    Doros, G.2    Sam, F.3    Badiee, A.4    Seldin, D.C.5    Skinner, M.6
  • 32
    • 84875956829 scopus 로고    scopus 로고
    • Comparison of cardiac amyloidosis due to wild-type and V122I transthyretin in older adults referred to an academic medical center
    • Givens RC, Russo C, Green P, Maurer MS. Comparison of cardiac amyloidosis due to wild-type and V122I transthyretin in older adults referred to an academic medical center. Aging Health. 2013;9:229-235. doi: 10. 2217/ahe. 13. 10.
    • (2013) Aging Health. , vol.9 , pp. 229-235
    • Givens, R.C.1    Russo, C.2    Green, P.3    Maurer, M.S.4
  • 35
    • 35648964702 scopus 로고    scopus 로고
    • Progression of cardiac amyloid deposition in hereditary transthyretin amyloidosis patients after liver transplantation
    • Liepnieks JJ, Benson MD. Progression of cardiac amyloid deposition in hereditary transthyretin amyloidosis patients after liver transplantation. Amyloid. 2007;14:277-282. doi: 10. 1080/13506120701614032.
    • (2007) Amyloid. , vol.14 , pp. 277-282
    • Liepnieks, J.J.1    Benson, M.D.2
  • 36
    • 33751023007 scopus 로고    scopus 로고
    • Diflunisal stabilizes familial amyloid polyneuropathy-associated transthyretin variant tetramers in serum against dissociation required for amyloidogenesis
    • Tojo K, Sekijima Y, Kelly JW, Ikeda S. Diflunisal stabilizes familial amyloid polyneuropathy-associated transthyretin variant tetramers in serum against dissociation required for amyloidogenesis. Neurosci Res. 2006;56:441-449. doi: 10. 1016/j. neures. 2006. 08. 014.
    • (2006) Neurosci Res. , vol.56 , pp. 441-449
    • Tojo, K.1    Sekijima, Y.2    Kelly, J.W.3    Ikeda, S.4
  • 43
    • 84962220171 scopus 로고    scopus 로고
    • Effects of tafamidis treatment on transthyretin (TTR) stabilization, efficacy, and safety in Japanese patients with familial amyloid polyneuropathy (TTR-FAP) with Val30Met and non-Val30Met: A phase III, open-label study
    • Ando Y, Sekijima Y, Obayashi K, Yamashita T, Ueda M, Misumi Y, Morita H, Machii K, Ohta M, Takata A, Ikeda S. Effects of tafamidis treatment on transthyretin (TTR) stabilization, efficacy, and safety in Japanese patients with familial amyloid polyneuropathy (TTR-FAP) with Val30Met and non-Val30Met: A phase III, open-label study. J Neurol Sci. 2016;362:266-271. doi: 10. 1016/j. jns. 2016. 01. 046.
    • (2016) J Neurol Sci. , vol.362 , pp. 266-271
    • Ando, Y.1    Sekijima, Y.2    Obayashi, K.3    Yamashita, T.4    Ueda, M.5    Misumi, Y.6    Morita, H.7    Machii, K.8    Ohta, M.9    Takata, A.10    Ikeda, S.11
  • 46
    • 84939946700 scopus 로고    scopus 로고
    • Cardiac findings and events observed in an open-label clinical trial of tafamidis in patients with non-Val30Met and non-Val122Ile hereditary transthyretin amyloidosis
    • Damy T, Judge DP, Kristen AV, Berthet K, Li H, Aarts J. Cardiac findings and events observed in an open-label clinical trial of tafamidis in patients with non-Val30Met and non-Val122Ile hereditary transthyretin amyloidosis. J Cardiovasc Transl Res. 2015;8:117-127. doi: 10. 1007/ s12265-015-9613-9.
    • (2015) J Cardiovasc Transl Res. , vol.8 , pp. 117-127
    • Damy, T.1    Judge, D.P.2    Kristen, A.V.3    Berthet, K.4    Li, H.5    Aarts, J.6
  • 48
    • 84940035415 scopus 로고    scopus 로고
    • Personalized medicine approach for optimizing the dose of tafamidis to potentially ameliorate wild-type transthyretin amyloidosis (cardiomyopathy)
    • Cho Y, Baranczak A, Helmke S, Teruya S, Horn EM, Maurer MS, Kelly JW. Personalized medicine approach for optimizing the dose of tafamidis to potentially ameliorate wild-type transthyretin amyloidosis (cardiomyopathy). Amyloid. 2015;22:175-180. doi: 10. 3109/13506129. 2015. 1063485.
    • (2015) Amyloid. , vol.22 , pp. 175-180
    • Cho, Y.1    Baranczak, A.2    Helmke, S.3    Teruya, S.4    Horn, E.M.5    Maurer, M.S.6    Kelly, J.W.7
  • 50
    • 0033564460 scopus 로고    scopus 로고
    • Combining mortality and longitudinal measures in clinical trials
    • Finkelstein DM, Schoenfeld DA. Combining mortality and longitudinal measures in clinical trials. Stat Med. 1999;18:1341-1354.
    • (1999) Stat Med. , vol.18 , pp. 1341-1354
    • Finkelstein, D.M.1    Schoenfeld, D.A.2
  • 53
    • 84963586313 scopus 로고    scopus 로고
    • U. S. Food and Drug Administration. Accessed April 5, 2017
    • U. S. Food and Drug Administration. Rare Diseases: Common Issues in Drug Development. 2015. http://www. fda. gov/Drugs/ GuidanceComplianceRegulatoryInformation/Guidances/ucm423881. htm. Accessed April 5, 2017.
    • (2015) Rare Diseases: Common Issues in Drug Development


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.