Transthyretin Amyloidosis Outcome Survey (THAOS) (THAOS)

July 19, 2023 updated by: Pfizer

Transthyretin Amyloidosis Outcomes Survey (THAOS): A Global, Multi-Center, Longitudinal, Observational Survey of Patients With Documented Transthyretin Gene Mutations or Wild-Type Transthyretin Amyloidosis.

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

n/a NA

Study Type

Observational

Enrollment (Actual)

6752

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
      • Buenos Aires, Argentina, 1199ABB
        • Hospital Italiano de Buenos Aires (HIBA)
      • Ciudad Autonoma De Buenos Aires, Argentina
        • Instituto De Investigaciones Medicas Dr Alfredo Lanari
      • Ciudad Autonoma de Buenos aires, Argentina, C1428AQK
        • FLENI
  • Belgium
      • Leuven, Belgium, 3000
        • Afdeling Klinische Cardiologie, O&N I
  • Brazil
      • Rio de Janeiro, Brazil, 21941-913
        • Hospital Universitário Clementino Fraga Filho -HUCFF Universidade Federal do Rio de Janeiro
      • Sao Paulo, Brazil, 04012-909
        • Instituto Dante Pazzanese de Cardiologia
  • Bulgaria
      • Sofia, Bulgaria, 1431
        • Alexandrovska University Hospital Clinic of Neurology
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T2N 4Z6
        • University of Alberta Foothills Medical Centre
    • Ontario
      • Toronto, Ontario, Canada, M5G 2N2
        • Toronto General Hospital
      • Toronto, Ontario, Canada, M5G 2C4
        • Toronto General Hospital-University Health Network
  • Cyprus
      • Nicosia, Cyprus, 23462
        • Cyprus Institute of Neurology and Genetics
  • Denmark
      • Aarhus, Denmark, 8200
        • Aarhus University Hospital, Skejby
  • France
      • Cedex, France, 94275
        • CHU de Bicêtre
      • Colmar, France, 68024
        • Hôpital Louis Pasteur
      • Créteil, France, 94000
        • CHU Henri Mondor
      • Fort De France, France, 97200
        • CHU de Fort de France
      • Lille, France, 59037
        • CHRU de Lille, Hopital Claude Huriez
      • Lille, France, 59037
        • Hopital Salengro - CHRU de Lille
      • Toulouse cedex 09, France, 31059
        • CHU de Toulouse - Hôpital Rangueil
  • Germany
      • Aachen, Germany, 52074
        • University Hospital of RWTH Aachen
      • Berlin, Germany, 13353
        • Charite CAmpus Rudolf-Virchow-Klinikum
      • Heidelberg, Germany, D-69120
        • Medical University of Heidelberg
      • Mainz, Germany, 55131
        • Johann-Gutenberg-Universität
      • Mainz, Germany, 55131
        • University Medical Center, Johannes Gutenberg-University Mainz
      • Mainz, Germany, 55131
        • Universitätsmedizin der Johannes Gutenberg Universität Mainz KöR
      • Muenster, Germany, 48149
        • Universitatsklinikum Muenster - Transplant Hepatology
  • Israel
      • Holon, Israel, 58100
        • Wolfson Medical Center
      • Ramat Gan, Israel, 52621
        • Sheba Medical Center
  • Italy
      • Bologna, Italy, 40138
        • Comitato Etico Indipendente dell Azienda
      • Firenze, Italy, 50134
        • Azienda Ospedaliero-Universitaria di Careggi
      • Messina, Italy, 98125
        • AOU Policlinico G. Martino - Messina - Comitato Etico Scientifico
      • Messina, Italy, 98125
        • AOU Policlinico G. Martino - Messina - Dr. Vita
      • Pavia, Italy, 27100
        • Centro per lo Studio e la Cura delle Amiloidosi Sistemiche - Pavia - Prof. Merlini
      • Pavia, Italy, 27100
        • Comitato di Bioetica della Fondazione IRCCS Policlinico S. Matteo
      • Pisa, Italy, 56126
        • Fondazione Toscana Gabriele Monasterio Per La Ricerca Medica E Di Sanita Pubblica (Ftgm)
      • Roma, Italy, 00168
        • Fondazione Policlinico Gemelli - Universita Cattolica del Sacro Cuore
  • Japan
      • Chiba, Japan, 260-8677
        • Chiba University Hospital
      • Kumamoto, Japan, 860-8556
        • Kumamoto University
    • JP
      • Matsumoto, JP, Japan, 390-8621
        • Shinshu University School of Medicine
  • Korea, Republic of
      • Seoul, Korea, Republic of, 143-729
        • Konkuk University Medical Center
      • Seoul, Korea, Republic of, 135-710
        • Samsung Medical Center, Sungkyunkwan University School of Medicine
  • Malaysia
    • Wilayah Persekutuan
      • Kuala Lumpur, Wilayah Persekutuan, Malaysia, 59100
        • University Malaya Medical Centre (UMMC)
  • Mexico
      • Distrito Federal, Mexico, 14000
        • Instituto Nacional de Ciencia Medicas y Nutricion Salvador Zubiran
  • Netherlands
      • Groningen, Netherlands, 9713 GZ
        • University Medical Center Groningen
  • Portugal
      • Guimaraes, Portugal, 4835-004
        • Centro Hospitalar Do Alto Ave, Epe
      • Lisboa, Portugal, 1649-035
        • Hospital Santa Maria
      • Lisboa, Portugal, 1649-035
        • Centro Hospitalar Lisboa Norte (CHLN) EPE - Hospital de Santa Maria
      • Porto, Portugal, 4099-001
        • Unidade Clinica de Paramiloidose-Centro Hospitalar Porto,EPE-Hospital Geral Santo Antonio
  • Romania
      • Bucuresti, Romania, 022328
        • Institutul De Cardiologie Prof. Dr. C. C. Iliescu Spitalului Fundeni
  • Saudi Arabia
      • Riyadh, Saudi Arabia, 11564
        • King Faisal Specialist Hospital and Research Center
  • Spain
      • Barcelona, Spain, 08907
        • Hospital Universitari de Bellvitge
      • Barcelona, Spain, 08036
        • Institut Clinic de Nefrologia i Urologia - ICNU, Hospital Clinic i Provincial de Barcelona
      • Granada, Spain, 18013
        • Hospital de Rehabilitacion y Traumatologia Virgen de las Nieves
      • Huelva, Spain, 21005
        • Hospital Juan Ramón Jimenez
      • Madrid, Spain, 28040
        • Hospital Clinico San Carlos
      • Madrid, Spain, 28007
        • Hospital Gregorio Marañón
      • Palma de Mallorca, Spain, 07198
        • Hospital Son Llatzer
    • Donostia
      • Gipuzkoa - SanSebastian, Donostia, Spain, 20014
        • Hospital Universitario Donostia
    • Madrid
      • Majadahonda, Madrid, Spain, 28220
        • Hospital Universitario Puerta de Hierro
  • Sweden
      • Piteå, Sweden, 941 50
        • Piteå Älvdals Hospital
      • Stockholm, Sweden, 141 86
        • Karolinska University Hospital, Huddinge
      • Umeå, Sweden
        • Umeå University Hospital
  • Taiwan
      • Taipei, Taiwan, 100
        • National Taiwan University Hospital
      • Taipei, Taiwan, 10018
        • National Taiwan University Hospital
  • Turkey
      • Istanbul, Turkey, 34093
        • Istanbul University,Istanbul Faculty of Medicine,Department of Neurology
  • United Arab Emirates
      • Abu Dhabi, United Arab Emirates
        • Cleveland Clinic Abu Dhabi LLC
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • University of Alabama at Birmingham
    • Arizona
      • Phoenix, Arizona, United States, 85054
        • Mayo Clinic Arizona
    • California
      • Los Angeles, California, United States, 90073
        • VA Greater Los Angeles Healthcare System
      • Orange, California, United States, 92868
        • University of California, Irvine
      • San Francisco, California, United States, 94117
        • University of California - San Francisco, UCSF Department of Neurology
      • San Francisco, California, United States, 94143
        • Office of Sponsored Research
      • Stanford, California, United States, 94305-5406
        • Stanford University School of Medicine
    • Colorado
      • Aurora, Colorado, United States, 80045
        • UC Denver
    • Connecticut
      • New Haven, Connecticut, United States, 06510
        • Smilow Cancer Hospital at Yale-New Haven
    • Florida
      • Deerfield Beach, Florida, United States, 33442
        • UHealth Deerfield Beach
      • Miami, Florida, United States, 33136
        • University of Miami Hospital & Clinics
      • Plantation, Florida, United States, 33324
        • UHealth Plantation
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern University
      • Chicago, Illinois, United States, 60637
        • University of Chicago Medical Center
      • Chicago, Illinois, United States, 60611
        • Clinical Trials Unit
      • Chicago, Illinois, United States, 60637
        • University of Chicago Medical Center IRB
      • Oak Lawn, Illinois, United States, 60453
        • Advocate Christ Medical Centre
    • Louisiana
      • New Orleans, Louisiana, United States, 70121
        • John Ochsner Heart & Vascular Institute
    • Maryland
      • Baltimore, Maryland, United States, 21201
        • University of Maryland
      • Baltimore, Maryland, United States, 21287
        • Johns Hopkins Hospital
      • Baltimore, Maryland, United States, 21201
        • University of Maryland School of Medicine
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Harvard Vanguard Medical Associates
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University School of Medicine
    • New York
      • Bronx, New York, United States, 10461
        • Cardiology Clinic at Montefiore Hutchinson Campus
      • Bronx, New York, United States, 10461
        • Montefiore Medical Center-Jack D. Weiler Hospital
      • Bronx, New York, United States, 10467
        • Congestive Heart Failure Clinic
      • Bronx, New York, United States, 10467
        • Montefiore Moses Division
      • New York, New York, United States, 10016
        • NYU Medical Center
      • New York, New York, United States, 10034
        • Columbia University Medical Center
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic Foundation
      • Columbus, Ohio, United States, 43210
        • The Ohio University College of Medicine
      • Columbus, Ohio, United States, 43221
        • OSU Wexner Medical Center
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health and Science University
    • Pennsylvania
      • McMurray, Pennsylvania, United States, 15317
        • Peters Township Health and Wellness Pavillion
      • Philadelphia, Pennsylvania, United States, 19140
        • Temple University School of Medicine
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania - Perelman Center for Advanced Medicine
      • Philadelphia, Pennsylvania, United States, 19104
        • Penn Presbyterian Medical Center-University of Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15212
        • Allegheny General Hospital
      • Pittsburgh, Pennsylvania, United States, 15213
        • University of Pittsburgh Medical Center (UPMC)
      • Pittsburgh, Pennsylvania, United States, 15213
        • Admin
      • Wexford, Pennsylvania, United States, 15090
        • Wexford Health and Wellness Pavillion
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University School of Medicine
      • Nashville, Tennessee, United States, 37232
        • IRB
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • The University of Utah Health Sciences Center
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-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.

Description

Inclusion Criteria: Patients must meet all of the following inclusion criteria to be eligible for inclusion into THAOS:

  1. 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.
  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

Patients meeting any of the following will not be included in the study:

1. Patient has evidence of primary (light chain) or secondary amyloidosis.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Other
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Observational
Other: None. Observational Study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The objectives of THAOS are to describe the population of patients affected with TTR amyloidosis and to enhance the understanding of the disease natural history, including the variability and progression of the hereditary and acquired forms of disease.
Time Frame: Dec 2007 to June 2023
Cardiovascular and Neurological efficacy endpoints for analysis of clinical outcomes on all enrolled patients with available data. Outcomes will be examined for the entire patient group, as well as through subgroups based on important variables.
Dec 2007 to June 2023

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Pfizer

Investigators

  • Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 4, 2008

Primary Completion (Actual)

June 16, 2023

Study Completion (Actual)

June 16, 2023

Study Registration Dates

First Submitted

February 25, 2008

First Submitted That Met QC Criteria

March 4, 2008

First Posted (Estimated)

March 5, 2008

Study Record Updates

Last Update Posted (Actual)

July 21, 2023

Last Update Submitted That Met QC Criteria

July 19, 2023

Last Verified

July 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • B3461001
  • FX-R-001 (Other Identifier: Alias Study Number)
  • THAOS (Other Identifier: Alias Study Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

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.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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