Study Results
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View full resultsBasic Information
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COMPLETED
6718 participants
OBSERVATIONAL
2008-01-04
2023-06-19
Brief Summary
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Detailed Description
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Conditions
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Study Design
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OTHER
PROSPECTIVE
Study Groups
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Observational
None. Observational Study.
Interventions
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None. Observational Study.
Eligibility Criteria
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Inclusion Criteria
2. Males and females greater than or equal to 18 years of age.
3. Confirmed genotyped TTR mutation with or without a diagnosis of hereditary or wild-type ATTR amyloidosis. Confirmation of ATTRwt amyloidosis will be determined by genotyped confirmation that patient does not possess a known mutation in TTR gene (ie, is a carrier of wild-type allele only) via genetic testing and one of the following set of criteria (a, b, or c):
1. Presence of amyloid in cardiac biopsy tissue confirmed as TTR amyloid by mass spectrometry or immunohistochemistry; or
2. Evidence of cardiac involvement by echocardiogram as defined by left ventricle wall thickness of \>12 mm, and presence of amyloid in non-cardiac tissue confirmed as TTR amyloid by mass spectrometry or immunohistochemistry; or
3. Evidence of cardiac involvement by echocardiogram as defined by left ventricle wall thickness of \>12 mm, and presence of amyloid in cardiac tissue indirectly confirmed by scintigraphy with a "bone seeking tracer" eg, 99mTC-DPD \[99mTC-3,3-diphosphono-1,2-propano-dicarboxylic acid\], 99mTC- PYP \[Pyrophosphate\], and 99mTC-HMDP \[hydroxymethylene diphosphonate\] with Perugini grade greater than or equal to 2.
Exclusion Criteria
1\. Patient has evidence of primary (light chain) or secondary amyloidosis.
18 Years
ALL
No
Sponsors
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Pfizer
INDUSTRY
Responsible Party
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Principal Investigators
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Pfizer CT.gov Call Center
Role: STUDY_DIRECTOR
Pfizer
Locations
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University of Alabama at Birmingham
Birmingham, Alabama, United States
Mayo Clinic Arizona
Phoenix, Arizona, United States
VA Greater Los Angeles Healthcare System
Los Angeles, California, United States
University of California, Irvine
Orange, California, United States
University of California - San Francisco, UCSF Department of Neurology
San Francisco, California, United States
Office of Sponsored Research
San Francisco, California, United States
Stanford University School of Medicine
Stanford, California, United States
UC Denver
Aurora, Colorado, United States
Smilow Cancer Hospital at Yale-New Haven
New Haven, Connecticut, United States
UHealth Deerfield Beach
Deerfield Beach, Florida, United States
University of Miami Hospital & Clinics
Miami, Florida, United States
UHealth Plantation
Plantation, Florida, United States
Clinical Trials Unit
Chicago, Illinois, United States
Northwestern University
Chicago, Illinois, United States
University of Chicago Medical Center IRB
Chicago, Illinois, United States
University of Chicago Medical Center
Chicago, Illinois, United States
Advocate Christ Medical Centre
Oak Lawn, Illinois, United States
John Ochsner Heart & Vascular Institute
New Orleans, Louisiana, United States
University of Maryland School of Medicine
Baltimore, Maryland, United States
University of Maryland
Baltimore, Maryland, United States
Johns Hopkins Hospital
Baltimore, Maryland, United States
Harvard Vanguard Medical Associates
Boston, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
Mayo Clinic
Rochester, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, United States
NYU Medical Center
New York, New York, United States
Columbia University Medical Center
New York, New York, United States
Cardiology Clinic at Montefiore Hutchinson Campus
The Bronx, New York, United States
Montefiore Medical Center-Jack D. Weiler Hospital
The Bronx, New York, United States
Congestive Heart Failure Clinic
The Bronx, New York, United States
Montefiore Moses Division
The Bronx, New York, United States
Cleveland Clinic Foundation
Cleveland, Ohio, United States
The Ohio University College of Medicine
Columbus, Ohio, United States
OSU Wexner Medical Center
Columbus, Ohio, United States
Oregon Health and Science University
Portland, Oregon, United States
Peters Township Health and Wellness Pavillion
McMurray, Pennsylvania, United States
Penn Presbyterian Medical Center-University of Pennsylvania
Philadelphia, Pennsylvania, United States
University of Pennsylvania - Perelman Center for Advanced Medicine
Philadelphia, Pennsylvania, United States
Temple University School of Medicine
Philadelphia, Pennsylvania, United States
Allegheny General Hospital
Pittsburgh, Pennsylvania, United States
Admin
Pittsburgh, Pennsylvania, United States
University of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, United States
Wexford Health and Wellness Pavillion
Wexford, Pennsylvania, United States
IRB
Nashville, Tennessee, United States
Vanderbilt University School of Medicine
Nashville, Tennessee, United States
The University of Utah Health Sciences Center
Salt Lake City, Utah, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, United States
Hospital Italiano de Buenos Aires (HIBA)
Buenos Aires, , Argentina
FLENI
Buenos Aires, , Argentina
Instituto De Investigaciones Medicas Dr Alfredo Lanari
Buenos Aires, , Argentina
Afdeling Klinische Cardiologie, O&N I
Leuven, , Belgium
Hospital Universitário Clementino Fraga Filho -HUCFF Universidade Federal do Rio de Janeiro
Rio de Janeiro, , Brazil
Instituto Dante Pazzanese De Cardiologia
São Paulo, , Brazil
Alexandrovska University Hospital Clinic of Neurology
Sofia, , Bulgaria
University of Alberta Foothills Medical Centre
Calgary, Alberta, Canada
Toronto General Hospital-University Health Network
Toronto, Ontario, Canada
Toronto General Hospital
Toronto, Ontario, Canada
Cyprus Institute of Neurology and Genetics
Nicosia, , Cyprus
Aarhus University Hospital, Skejby
Aarhus, , Denmark
CHU de Bicetre
Cedex, , France
Hopital Louis Pasteur
Colmar, , France
CHU Henri Mondor
Créteil, , France
Chu De Fort De France
Fort de France, , France
CHRU de Lille, Hopital Claude Huriez
Lille, , France
Hopital Salengro - CHRU de Lille
Lille, , France
CHU de Toulouse - Hopital Rangueil
Toulouse, , France
University Hospital of RWTH Aachen
Aachen, , Germany
Charite CAmpus Rudolf-Virchow-Klinikum
Berlin, , Germany
Medical University of Heidelberg
Heidelberg, , Germany
Johann-Gutenberg-Universität
Mainz, , Germany
University Medical Center, Johannes Gutenberg-University Mainz
Mainz, , Germany
Universitätsmedizin der Johannes Gutenberg Universität Mainz KöR
Mainz, , Germany
Universitatsklinikum Muenster - Transplant Hepatology
Münster, , Germany
Wolfson Medical Center
Holon, , Israel
Sheba Medical Center
Ramat Gan, , Israel
Comitato Etico Indipendente dell Azienda
Bologna, , Italy
Azienda Ospedaliero-Universitaria di Careggi
Florence, , Italy
AOU Policlinico G. Martino - Messina - Comitato Etico Scientifico
Messina, , Italy
AOU Policlinico G. Martino - Messina - Dr. Vita
Messina, , Italy
Centro per lo Studio e la Cura delle Amiloidosi Sistemiche - Pavia - Prof. Merlini
Pavia, , Italy
Comitato di Bioetica della Fondazione IRCCS Policlinico S. Matteo
Pavia, , Italy
Fondazione Toscana Gabriele Monasterio Per La Ricerca Medica E Di Sanita Pubblica (Ftgm)
Pisa, , Italy
Fondazione Policlinico Gemelli - Universita Cattolica del Sacro Cuore
Roma, , Italy
Shinshu University School of Medicine
Matsumoto, JP, Japan
Chiba University Hospital
Chiba, , Japan
Kumamoto University
Kumamoto, , Japan
University Malaya Medical Centre (UMMC)
Kuala Lumpur, Kuala Lumpur, Malaysia
Instituto Nacional de Ciencia Medicas y Nutricion Salvador Zubiran
Distrito Federal, , Mexico
University Medical Center Groningen
Groningen, , Netherlands
Centro Hospitalar Do Alto Ave, Epe
Guimarães, , Portugal
Centro Hospitalar Lisboa Norte (CHLN) EPE - Hospital de Santa Maria
Lisbon, , Portugal
Hospital Santa Maria
Lisbon, , Portugal
Unidade Clinica de Paramiloidose-Centro Hospitalar Porto,EPE-Hospital Geral Santo Antonio
Porto, , Portugal
Institutul De Cardiologie Prof. Dr. C. C. Iliescu Spitalului Fundeni
Bucharest, , Romania
King Faisal Specialist Hospital and Research Center
Riyadh, , Saudi Arabia
Samsung Medical Center, Sungkyunkwan University School of Medicine
Seoul, , South Korea
Konkuk University Medical Center
Seoul, , South Korea
Hospital Universitario Donostia
Gipuzkoa - SanSebastian, Donostia, Spain
Hospital Universitario Puerta de Hierro
Majadahonda, Madrid, Spain
Institut Clinic de Nefrologia i Urologia - ICNU, Hospital Clinic i Provincial de Barcelona
Barcelona, , Spain
Hospital Universitari de Bellvitge
Barcelona, , Spain
Hospital de Rehabilitacion y Traumatologia Virgen de las Nieves
Granada, , Spain
Hospital Juan Ramon Jimenez
Huelva, , Spain
Hospital Gregorio Marañón
Madrid, , Spain
Hospital Clinico San Carlos
Madrid, , Spain
Hospital Son Llà tzer
Palma de Mallorca, , Spain
Piteå Älvdals Hospital
Piteå, , Sweden
Karolinska University Hospital, Huddinge
Stockholm, , Sweden
Umeå University Hospital
Umeå, , Sweden
National Taiwan University Hospital
Taipei, , Taiwan
National Taiwan University Hospital
Taipei, , Taiwan
Istanbul University,Istanbul Faculty of Medicine,Department of Neurology
Istanbul, , Turkey (Türkiye)
Cleveland Clinic Abu Dhabi LLC
Abu Dhabi, , United Arab Emirates
Countries
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References
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Wixner J, Dispenzieri A, Amass L, Carlsson M, Riley S, Powers E, Kelly JW; THAOS investigators. Survival in a Contemporary, Real-World Cohort of Patients with Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis: An Analysis from THAOS. Cardiol Ther. 2025 Jul 9. doi: 10.1007/s40119-025-00421-9. Online ahead of print.
Mora-Ayestaran N, Dispenzieri A, Kristen AV, Maurer MS, Diemberger I, Drachman BM, Grogan M, Gupta P, Glass O, Amass L, Garcia-Pavia P; THAOS Investigators. Age- and Sex-Related Differences in Patients With Wild-Type Transthyretin Amyloidosis: Insights From THAOS. JACC Adv. 2024 Jul 12;3(8):101086. doi: 10.1016/j.jacadv.2024.101086. eCollection 2024 Aug.
Garcia-Pavia P, Kristen AV, Drachman B, Carlsson M, Amass L, Angeli FS, Maurer MS; THAOS investigators. Survival in a Real-World Cohort of Patients With Transthyretin Amyloid Cardiomyopathy Treated With Tafamidis: An Analysis From the Transthyretin Amyloidosis Outcomes Survey (THAOS). J Card Fail. 2025 Mar;31(3):525-533. doi: 10.1016/j.cardfail.2024.06.003. Epub 2024 Jun 21.
Gentile L, Diemberger I, Plante-Bordeneuve V, Mazzeo A, Dori A, Luigetti M, Di Paolantonio A, Dispenzieri A, Grogan M, Waddington Cruz M, Adams D, Inamo J, Kristen AV, Lino Cirami C, Chapman D, Gupta P, Glass O, Amass L. Phenotypic characteristics of F64L, I68L, I107V, and S77Y ATTRv genotypes from the Transthyretin Amyloidosis Outcomes Survey (THAOS). PLoS One. 2024 Jan 19;19(1):e0292435. doi: 10.1371/journal.pone.0292435. eCollection 2024.
Gonzalez-Moreno J, Dispenzieri A, Grogan M, Coelho T, Tournev I, Waddington-Cruz M, Wixner J, Diemberger I, Garcia-Pavia P, Chapman D, Gupta P, Glass O, Amass L; THAOS investigators. Clinical and Genotype Characteristics and Symptom Migration in Patients With Mixed Phenotype Transthyretin Amyloidosis from the Transthyretin Amyloidosis Outcomes Survey. Cardiol Ther. 2024 Mar;13(1):117-135. doi: 10.1007/s40119-023-00344-3. Epub 2023 Dec 20.
Gentile L, Coelho T, Dispenzieri A, Conceicao I, Waddington-Cruz M, Kristen A, Wixner J, Diemberger I, Gonzalez-Moreno J, Cariou E, Maurer MS, Plante-Bordeneuve V, Garcia-Pavia P, Tournev I, Gonzalez-Costello J, Duarte AG, Grogan M, Mazzeo A, Chapman D, Gupta P, Glass O, Amass L; THAOS investigators. A 15-year consolidated overview of data in over 6000 patients from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Orphanet J Rare Dis. 2023 Nov 10;18(1):350. doi: 10.1186/s13023-023-02962-5.
Coelho T, Conceicao I, Waddington-Cruz M, Keohane D, Sultan MB, Chapman D, Amass L; THAOS investigators. A natural history analysis of asymptomatic TTR gene carriers as they develop symptomatic transthyretin amyloidosis in the Transthyretin Amyloidosis Outcomes Survey (THAOS). Amyloid. 2022 Dec;29(4):228-236. doi: 10.1080/13506129.2022.2070470. Epub 2022 Jun 22.
Dispenzieri A, Coelho T, Conceicao I, Waddington-Cruz M, Wixner J, Kristen AV, Rapezzi C, Plante-Bordeneuve V, Gonzalez-Moreno J, Maurer MS, Grogan M, Chapman D, Amass L; THAOS investigators. Clinical and genetic profile of patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS): 14-year update. Orphanet J Rare Dis. 2022 Jun 18;17(1):236. doi: 10.1186/s13023-022-02359-w.
Campbell CM, LoRusso S, Dispenzieri A, Kristen AV, Maurer MS, Rapezzi C, Lairez O, Drachman B, Garcia-Pavia P, Grogan M, Chapman D, Amass L; THAOS investigators. Sex Differences in Wild-Type Transthyretin Amyloidosis: An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Cardiol Ther. 2022 Sep;11(3):393-405. doi: 10.1007/s40119-022-00265-7. Epub 2022 May 18.
Barroso FA, Coelho T, Dispenzieri A, Conceicao I, Waddington-Cruz M, Wixner J, Maurer MS, Rapezzi C, Plante-Bordeneuve V, Kristen AV, Gonzalez-Duarte A, Chapman D, Stewart M, Amass L; THAOS investigators. Characteristics of patients with autonomic dysfunction in the Transthyretin Amyloidosis Outcomes Survey (THAOS). Amyloid. 2022 Sep;29(3):175-183. doi: 10.1080/13506129.2022.2043270. Epub 2022 Apr 22.
Nativi-Nicolau J, Siu A, Dispenzieri A, Maurer MS, Rapezzi C, Kristen AV, Garcia-Pavia P, LoRusso S, Waddington-Cruz M, Lairez O, Witteles R, Chapman D, Amass L, Grogan M; THAOS Investigators. Temporal Trends of Wild-Type Transthyretin Amyloid Cardiomyopathy in the Transthyretin Amyloidosis Outcomes Survey. JACC CardioOncol. 2021 Oct 19;3(4):537-546. doi: 10.1016/j.jaccao.2021.08.009. eCollection 2021 Oct.
Gonzalez-Moreno J, Losada-Lopez I, Cisneros-Barroso E, Garcia-Pavia P, Gonzalez-Costello J, Munoz-Beamud F, Campistol JM, Fernandez-Torron R, Chapman D, Amass L. A Descriptive Analysis of ATTR Amyloidosis in Spain from the Transthyretin Amyloidosis Outcomes Survey. Neurol Ther. 2021 Dec;10(2):833-845. doi: 10.1007/s40120-021-00267-y. Epub 2021 Jul 30.
Gentile L, Tournev I, Amass L, Chapman D, Mazzeo A; THAOS investigators. Phenotypic Differences of Glu89Gln Genotype in ATTR Amyloidosis From Endemic Loci: Update From THAOS. Cardiol Ther. 2021 Dec;10(2):481-490. doi: 10.1007/s40119-021-00226-6. Epub 2021 Jun 19.
Waddington-Cruz M, Wixner J, Amass L, Kiszko J, Chapman D, Ando Y; THAOS investigators. Characteristics of Patients with Late- vs. Early-Onset Val30Met Transthyretin Amyloidosis from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Neurol Ther. 2021 Dec;10(2):753-766. doi: 10.1007/s40120-021-00258-z. Epub 2021 May 22.
Gonzalez-Duarte A, Barroso F, Mundayat R, Shapiro B. Blood pressure and orthostatic hypotension as measures of autonomic dysfunction in patients from the transthyretin amyloidosis outcomes survey (THAOS). Auton Neurosci. 2019 Dec;222:102590. doi: 10.1016/j.autneu.2019.102590. Epub 2019 Oct 23.
Huber P, Flynn A, Sultan MB, Li H, Rill D, Ebede B, Gundapaneni B, Schwartz JH. A comprehensive safety profile of tafamidis in patients with transthyretin amyloid polyneuropathy. Amyloid. 2019 Dec;26(4):203-209. doi: 10.1080/13506129.2019.1643714. Epub 2019 Jul 27.
Pinto MV, Pinto LF, Dias M, Rosa RS, Mundayat R, Pedrosa RC, Waddington-Cruz M. Late-onset hereditary ATTR V30M amyloidosis with polyneuropathy: Characterization of Brazilian subjects from the THAOS registry. J Neurol Sci. 2019 Aug 15;403:1-6. doi: 10.1016/j.jns.2019.05.030. Epub 2019 May 28.
Damy T, Kristen AV, Suhr OB, Maurer MS, Plante-Bordeneuve V, Yu CR, Ong ML, Coelho T, Rapezzi C; THAOS Investigators. Transthyretin cardiac amyloidosis in continental Western Europe: an insight through the Transthyretin Amyloidosis Outcomes Survey (THAOS). Eur Heart J. 2022 Feb 3;43(5):391-400. doi: 10.1093/eurheartj/ehz173.
Cruz MW, Pinto MV, Pinto LF, Gervais R, Dias M, Perez C, Mundayat R, Ong ML, Pedrosa RC, Foguel D. Baseline disease characteristics in Brazilian patients enrolled in Transthyretin Amyloidosis Outcome Survey (THAOS). Arq Neuropsiquiatr. 2019 Feb;77(2):96-100. doi: 10.1590/0004-282X20180156.
Rubin J, Steidley DE, Carlsson M, Ong ML, Maurer MS. Myocardial Contraction Fraction by M-Mode Echocardiography Is Superior to Ejection Fraction in Predicting Mortality in Transthyretin Amyloidosis. J Card Fail. 2018 Aug;24(8):504-511. doi: 10.1016/j.cardfail.2018.07.001. Epub 2018 Aug 17.
Mundayat R, Stewart M, Alvir J, Short S, Ong ML, Keohane D, Rill D, Sultan MB. Positive Effectiveness of Tafamidis in Delaying Disease Progression in Transthyretin Familial Amyloid Polyneuropathy up to 2 Years: An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Neurol Ther. 2018 Jun;7(1):87-101. doi: 10.1007/s40120-018-0097-9. Epub 2018 Apr 9.
Kristen AV, Maurer MS, Rapezzi C, Mundayat R, Suhr OB, Damy T; THAOS investigators. Impact of genotype and phenotype on cardiac biomarkers in patients with transthyretin amyloidosis - Report from the Transthyretin Amyloidosis Outcome Survey (THAOS). PLoS One. 2017 Apr 6;12(4):e0173086. doi: 10.1371/journal.pone.0173086. eCollection 2017.
Maurer MS, Hanna M, Grogan M, Dispenzieri A, Witteles R, Drachman B, Judge DP, Lenihan DJ, Gottlieb SS, Shah SJ, Steidley DE, Ventura H, Murali S, Silver MA, Jacoby D, Fedson S, Hummel SL, Kristen AV, Damy T, Plante-Bordeneuve V, Coelho T, Mundayat R, Suhr OB, Waddington Cruz M, Rapezzi C; THAOS Investigators. Genotype and Phenotype of Transthyretin Cardiac Amyloidosis: THAOS (Transthyretin Amyloid Outcome Survey). J Am Coll Cardiol. 2016 Jul 12;68(2):161-72. doi: 10.1016/j.jacc.2016.03.596.
Damy T, Maurer MS, Rapezzi C, Plante-Bordeneuve V, Karayal ON, Mundayat R, Suhr OB, Kristen AV. Clinical, ECG and echocardiographic clues to the diagnosis of TTR-related cardiomyopathy. Open Heart. 2016 Feb 8;3(1):e000289. doi: 10.1136/openhrt-2015-000289. eCollection 2016.
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 Jan;29(1):63-76. doi: 10.1185/03007995.2012.754348. Epub 2012 Dec 13.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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To obtain contact information for a study center near you, click here.
Other Identifiers
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FX-R-001
Identifier Type: OTHER
Identifier Source: secondary_id
THAOS
Identifier Type: OTHER
Identifier Source: secondary_id
B3461001
Identifier Type: -
Identifier Source: org_study_id
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