NCT00628745

Brief Summary

THAOS is a global, multi-center, longitudinal observational survey open to all patients with transthyretin amyloidosis (ATTR), including ATTR-PN (polyneuropathy), ATTR-CM (cardiomyopathy) and wild-type ATTR-CM. It is open-ended with a minimum duration of 10 years. Patients will be followed as long as they are able to participate. The principal aims of this outcome survey are to better understand and characterize the natural history of the disease by studying a large and heterogenous patient population. Survey data may be used to develop new treatment guidelines and recommendations, and to inform and educate clinicians about the management of this disease.

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network
Enrollment
6,718

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Jan 2008

Longer than P75 for all trials

Geographic Reach
25 countries

113 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 4, 2008

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

February 25, 2008

Completed
9 days until next milestone

First Posted

Study publicly available on registry

March 5, 2008

Completed
15.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 16, 2023

Completed
3 days until next milestone

Study Completion

Last participant's last visit for all outcomes

June 19, 2023

Completed
1.4 years until next milestone

Results Posted

Study results publicly available

November 22, 2024

Completed
Last Updated

November 22, 2024

Status Verified

October 1, 2024

Enrollment Period

15.5 years

First QC Date

February 25, 2008

Results QC Date

April 4, 2024

Last Update Submit

October 1, 2024

Conditions

Keywords

TRANSTHYRETINAMYLOIDOSISTRANSTHYRETIN AMYLOIDOSISTransthyretin amyloid cardiomyopathy

Outcome Measures

Primary Outcomes (3)

  • Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

    An AE was any untoward medical occurrence in a participant who administered a medicinal product without regard to possibility of causal relationship with the study treatment. SAE was defined as one of the following: resulted in death; was life-threatening (immediate risk of death); requires inpatient hospitalization or prolongation of hospitalization; resulted in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); constituted a congenital anomaly/birth defect. Treatment-emergent AE was defined as an AE with onset date occurring during the on-treatment period. AEs included all SAEs and non-SAEs.

    From the start of data collection at Baseline (Day 1) to the end of data collection (Up to 14.4 years)

  • Number of Participants With Treatment Emergent Treatment Related AEs and SAEs

    A treatment-related AE was any untoward medical occurrence attributed to the administered medicinal product in a participant who received study drug. Treatment emergent AEs included both SAEs and all non-SAEs. A treatment-related SAE was a treatment-related AE and was defined as any untoward medical occurrence at any dose that: resulted in death; was life-threatening (immediate risk of death); requires inpatient hospitalization or prolongation of hospitalization; resulted in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); constituted a congenital anomaly/birth defect. Causality was assessed by the investigator.

    From the start of data collection at Baseline (Day 1) to the end of data collection (Up to 14.4 years)

  • Number of All-Cause Deaths

    Number of deaths due to any cause was analyzed as time from enrollment or first treatment of tafamidis.

    From the start of data collection at Baseline (Day 1) to the end of data collection (Up to 14.4 years)

Secondary Outcomes (23)

  • Number of Participants With Modified Polyneuropathy Disability (mPND) Scores at Baseline

    At the start of data collection at Baseline (Day 1)

  • Number of Participants With Coutinho Disease Stages at Baseline

    At the start of data collection at Baseline (Day 1)

  • Number of Participants With Karnofsky Performance Index at Baseline

    At the start of data collection at Baseline (Day 1)

  • Number of Participants With Heart Failure at Baseline

    At the start of data collection at Baseline (Day 1)

  • Number of Participants With New York Heart Association (NYHA) Classifications at Baseline

    At the start of data collection at Baseline (Day 1)

  • +18 more secondary outcomes

Study Arms (1)

Observational

Other: None. Observational Study.

Interventions

Observational

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

The study population includes patients with confirmed hereditary or wild-type ATTR amyloidosis (inclusive of ATTR-PN and ATTR-CM) and those with TTR gene mutations without a diagnosis of ATTR amyloidosis.

You may qualify if:

  • Evidence of a personally signed and dated informed consent document indicating that the participant (or a legally acceptable representative) has been informed of all pertinent aspects of the study.
  • Males and females greater than or equal to 18 years of age.
  • 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):
  • Presence of amyloid in cardiac biopsy tissue confirmed as TTR amyloid by mass spectrometry or immunohistochemistry; or
  • 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
  • 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.

You may not qualify if:

  • Patients meeting any of the following will not be included in the study:
  • \. Patient has evidence of primary (light chain) or secondary amyloidosis.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (113)

University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States

Location

Mayo Clinic Arizona

Phoenix, Arizona, 85054, United States

Location

VA Greater Los Angeles Healthcare System

Los Angeles, California, 90073, United States

Location

University of California, Irvine

Orange, California, 92868, United States

Location

University of California - San Francisco, UCSF Department of Neurology

San Francisco, California, 94117, United States

Location

Office of Sponsored Research

San Francisco, California, 94143, United States

Location

Stanford University School of Medicine

Stanford, California, 94305-5406, United States

Location

UC Denver

Aurora, Colorado, 80045, United States

Location

Smilow Cancer Hospital at Yale-New Haven

New Haven, Connecticut, 06510, United States

Location

UHealth Deerfield Beach

Deerfield Beach, Florida, 33442, United States

Location

University of Miami Hospital & Clinics

Miami, Florida, 33136, United States

Location

UHealth Plantation

Plantation, Florida, 33324, United States

Location

Clinical Trials Unit

Chicago, Illinois, 60611, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

University of Chicago Medical Center IRB

Chicago, Illinois, 60637, United States

Location

University of Chicago Medical Center

Chicago, Illinois, 60637, United States

Location

Advocate Christ Medical Centre

Oak Lawn, Illinois, 60453, United States

Location

John Ochsner Heart & Vascular Institute

New Orleans, Louisiana, 70121, United States

Location

University of Maryland School of Medicine

Baltimore, Maryland, 21201, United States

Location

University of Maryland

Baltimore, Maryland, 21201, United States

Location

Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

Location

Harvard Vanguard Medical Associates

Boston, Massachusetts, 02215, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

NYU Medical Center

New York, New York, 10016, United States

Location

Columbia University Medical Center

New York, New York, 10034, United States

Location

Cardiology Clinic at Montefiore Hutchinson Campus

The Bronx, New York, 10461, United States

Location

Montefiore Medical Center-Jack D. Weiler Hospital

The Bronx, New York, 10461, United States

Location

Congestive Heart Failure Clinic

The Bronx, New York, 10467, United States

Location

Montefiore Moses Division

The Bronx, New York, 10467, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

The Ohio University College of Medicine

Columbus, Ohio, 43210, United States

Location

OSU Wexner Medical Center

Columbus, Ohio, 43221, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Peters Township Health and Wellness Pavillion

McMurray, Pennsylvania, 15317, United States

Location

Penn Presbyterian Medical Center-University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

University of Pennsylvania - Perelman Center for Advanced Medicine

Philadelphia, Pennsylvania, 19104, United States

Location

Temple University School of Medicine

Philadelphia, Pennsylvania, 19140, United States

Location

Allegheny General Hospital

Pittsburgh, Pennsylvania, 15212, United States

Location

Admin

Pittsburgh, Pennsylvania, 15213, United States

Location

University of Pittsburgh Medical Center (UPMC)

Pittsburgh, Pennsylvania, 15213, United States

Location

Wexford Health and Wellness Pavillion

Wexford, Pennsylvania, 15090, United States

Location

IRB

Nashville, Tennessee, 37232, United States

Location

Vanderbilt University School of Medicine

Nashville, Tennessee, 37232, United States

Location

The University of Utah Health Sciences Center

Salt Lake City, Utah, 84132, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Hospital Italiano de Buenos Aires (HIBA)

Buenos Aires, 1199ABB, Argentina

Location

FLENI

Buenos Aires, C1428AQK, Argentina

Location

Instituto De Investigaciones Medicas Dr Alfredo Lanari

Buenos Aires, Argentina

Location

Afdeling Klinische Cardiologie, O&N I

Leuven, 3000, Belgium

Location

Hospital Universitário Clementino Fraga Filho -HUCFF Universidade Federal do Rio de Janeiro

Rio de Janeiro, 21941-913, Brazil

Location

Instituto Dante Pazzanese De Cardiologia

São Paulo, 04012-909, Brazil

Location

Alexandrovska University Hospital Clinic of Neurology

Sofia, 1431, Bulgaria

Location

University of Alberta Foothills Medical Centre

Calgary, Alberta, T2N 4Z6, Canada

Location

Toronto General Hospital-University Health Network

Toronto, Ontario, M5G 2C4, Canada

Location

Toronto General Hospital

Toronto, Ontario, M5G 2N2, Canada

Location

Cyprus Institute of Neurology and Genetics

Nicosia, 23462, Cyprus

Location

Aarhus University Hospital, Skejby

Aarhus, 8200, Denmark

Location

CHU de Bicetre

Cedex, 94275, France

Location

Hopital Louis Pasteur

Colmar, 68024, France

Location

CHU Henri Mondor

Créteil, 94000, France

Location

Chu De Fort De France

Fort de France, 97200, France

Location

CHRU de Lille, Hopital Claude Huriez

Lille, 59037, France

Location

Hopital Salengro - CHRU de Lille

Lille, 59037, France

Location

CHU de Toulouse - Hopital Rangueil

Toulouse, 31059, France

Location

University Hospital of RWTH Aachen

Aachen, 52074, Germany

Location

Charite CAmpus Rudolf-Virchow-Klinikum

Berlin, 13353, Germany

Location

Medical University of Heidelberg

Heidelberg, D-69120, Germany

Location

Johann-Gutenberg-Universität

Mainz, 55131, Germany

Location

University Medical Center, Johannes Gutenberg-University Mainz

Mainz, 55131, Germany

Location

Universitätsmedizin der Johannes Gutenberg Universität Mainz KöR

Mainz, 55131, Germany

Location

Universitatsklinikum Muenster - Transplant Hepatology

Münster, 48149, Germany

Location

Wolfson Medical Center

Holon, 58100, Israel

Location

Sheba Medical Center

Ramat Gan, 52621, Israel

Location

Comitato Etico Indipendente dell Azienda

Bologna, 40138, Italy

Location

Azienda Ospedaliero-Universitaria di Careggi

Florence, 50134, Italy

Location

AOU Policlinico G. Martino - Messina - Comitato Etico Scientifico

Messina, 98125, Italy

Location

AOU Policlinico G. Martino - Messina - Dr. Vita

Messina, 98125, Italy

Location

Centro per lo Studio e la Cura delle Amiloidosi Sistemiche - Pavia - Prof. Merlini

Pavia, 27100, Italy

Location

Comitato di Bioetica della Fondazione IRCCS Policlinico S. Matteo

Pavia, 27100, Italy

Location

Fondazione Toscana Gabriele Monasterio Per La Ricerca Medica E Di Sanita Pubblica (Ftgm)

Pisa, 56126, Italy

Location

Fondazione Policlinico Gemelli - Universita Cattolica del Sacro Cuore

Roma, 00168, Italy

Location

Shinshu University School of Medicine

Matsumoto, JP, 390-8621, Japan

Location

Chiba University Hospital

Chiba, 260-8677, Japan

Location

Kumamoto University

Kumamoto, 860-8556, Japan

Location

University Malaya Medical Centre (UMMC)

Kuala Lumpur, Kuala Lumpur, 59100, Malaysia

Location

Instituto Nacional de Ciencia Medicas y Nutricion Salvador Zubiran

Distrito Federal, 14000, Mexico

Location

University Medical Center Groningen

Groningen, 9713 GZ, Netherlands

Location

Centro Hospitalar Do Alto Ave, Epe

Guimarães, 4835-004, Portugal

Location

Centro Hospitalar Lisboa Norte (CHLN) EPE - Hospital de Santa Maria

Lisbon, 1649-035, Portugal

Location

Hospital Santa Maria

Lisbon, 1649-035, Portugal

Location

Unidade Clinica de Paramiloidose-Centro Hospitalar Porto,EPE-Hospital Geral Santo Antonio

Porto, 4099-001, Portugal

Location

Institutul De Cardiologie Prof. Dr. C. C. Iliescu Spitalului Fundeni

Bucharest, 022328, Romania

Location

King Faisal Specialist Hospital and Research Center

Riyadh, 11564, Saudi Arabia

Location

Samsung Medical Center, Sungkyunkwan University School of Medicine

Seoul, 135-710, South Korea

Location

Konkuk University Medical Center

Seoul, 143-729, South Korea

Location

Hospital Universitario Donostia

Gipuzkoa - SanSebastian, Donostia, 20014, Spain

Location

Hospital Universitario Puerta de Hierro

Majadahonda, Madrid, 28220, Spain

Location

Institut Clinic de Nefrologia i Urologia - ICNU, Hospital Clinic i Provincial de Barcelona

Barcelona, 08036, Spain

Location

Hospital Universitari de Bellvitge

Barcelona, 08907, Spain

Location

Hospital de Rehabilitacion y Traumatologia Virgen de las Nieves

Granada, 18013, Spain

Location

Hospital Juan Ramon Jimenez

Huelva, 21005, Spain

Location

Hospital Gregorio Marañón

Madrid, 28007, Spain

Location

Hospital Clinico San Carlos

Madrid, 28040, Spain

Location

Hospital Son Llàtzer

Palma de Mallorca, 07198, Spain

Location

Piteå Älvdals Hospital

Piteå, 941 50, Sweden

Location

Karolinska University Hospital, Huddinge

Stockholm, 141 86, Sweden

Location

Umeå University Hospital

Umeå, Sweden

Location

National Taiwan University Hospital

Taipei, 10018, Taiwan

Location

National Taiwan University Hospital

Taipei, 100, Taiwan

Location

Istanbul University,Istanbul Faculty of Medicine,Department of Neurology

Istanbul, 34093, Turkey (Türkiye)

Location

Cleveland Clinic Abu Dhabi LLC

Abu Dhabi, United Arab Emirates

Location

Related Publications (26)

  • Wixner J, Dispenzieri A, Amass L, Carlsson M, Riley S, Powers E, Kelly JW. A real-world study of tafamidis in people with transthyretin amyloid cardiomyopathy (ATTR-CM) with heart and nerve symptoms: a plain language summary. Future Cardiol. 2025 Nov 25:1-13. doi: 10.1080/14796678.2025.2583805. Online ahead of print.

  • 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 Dec;14(4):583-599. doi: 10.1007/s40119-025-00421-9. Epub 2025 Jul 9.

  • 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.

Related Links

Biospecimen

Retention: SAMPLES WITH DNA

whole blood

MeSH Terms

Conditions

Amyloidosis, Hereditary, Transthyretin-RelatedAmyloidosis

Condition Hierarchy (Ancestors)

Proteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Limitations and Caveats

A total of 6718 evaluable participants were enrolled in this study. The database has 6752 participants and the discrepancy in the numbers is due to 1 system screen failure and 33 subjects that are either duplicates or were transferred to a different site.

Results Point of Contact

Title
Pfizer ClinicalTrials.gov Call Center
Organization
Pfizer Inc.

Study Officials

  • Pfizer CT.gov Call Center

    Pfizer

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 25, 2008

First Posted

March 5, 2008

Study Start

January 4, 2008

Primary Completion

June 16, 2023

Study Completion

June 19, 2023

Last Updated

November 22, 2024

Results First Posted

November 22, 2024

Record last verified: 2024-10

Data Sharing

IPD Sharing
Will not share

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.

Locations