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Transthyretin-Associated Amyloidoses Outcome Survey (THAOS)

This study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00628745
First received: February 25, 2008
Last updated: October 31, 2014
Last verified: October 2014
  Purpose

THAOS is a global, multi-center, longitudinal observational survey open to all patients with transthyretin-associated amyloidoses (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.


Condition Intervention Phase
Transthyretin Mutations
Transthyretin Amyloidosis
Other: None. Observational Study.
Phase 4

Study Type: Observational
Study Design: Time Perspective: Prospective
Official Title: Transthyretin-associated Amyloidosis Outcomes Survey (Thaos): A Global, Multi-center, Longitudinal, Observational Survey Of Patients With Documented Transthyretin (Ttr) Mutations Or Wild-type Ttr Amyloidosis

Resource links provided by NLM:


Further study details as provided by Pfizer:

Biospecimen Retention:   Samples With DNA

whole blood


Estimated Enrollment: 3000
Study Start Date: December 2007
Estimated Study Completion Date: June 2021
Estimated Primary Completion Date: June 2021 (Final data collection date for primary outcome measure)
Intervention Details:
    Other: None. Observational Study.
    None.
Detailed Description:

n/a NA

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Participants with transthyretin-associated amyloidoses (ATTR), including ATTR-PN (polyneuropathy), ATTR-CM (cardiomyopathy) and wild-type ATTR-CM.

Criteria

Inclusion Criteria:

  • Written informed consent.
  • Patient has confirmed: genotyped TTR mutation with or without a diagnosis of TTR-associated amyloidosis (e.g., ATTR-PN, ATTR-CM), or Wild-type TTR-associated amyloidosis with cardiomyopathy (wild-type ATTR-CM).
  • Confirmation of wild-type ATTR-CM will be determined by one of the following set of criteria (A or B):

A. Presence of amyloid in cardiac biopsy tissue confirmed as TTR amyloid by immunohistochemistry and genotyped confirmation that patient does not possess a known mutation in TTR gene (i.e., is a carrier of wild-type allele only) via genetic testing; or B. Evidence of cardiac involvement by echocardiogram as defined by mean left ventricle wall thickness of > 12 mm, and presence of amyloid in non-cardiac tissue confirmed as TTR amyloid by immunohistochemistry, and genotyped confirmation that patient does not possess a known mutation in TTR gene (i.e., is a carrier of wild-type allele only) via genetic testing.

Exclusion Criteria:

  • Patient has primary or secondary amyloidosis.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00628745

  Hide Study Locations
Locations
United States, Arizona
Mayo Clinic
Phoenix, Arizona, United States, 85054
United States, California
University of California, Irvine
Orange, California, United States, 92868
Office of Sponsored Research
San Francisco, California, United States, 94143
University of California - San Francisco, UCSF Department of Neurology
San Francisco, California, United States, 94117
Falk Cardiovascular Research Center, Stanford University School of Medicine
Stanford, California, United States, 94305-5406
United States, Colorado
UC Denver;Department of Neurology
Aurora, Colorado, United States, 80045
United States, Connecticut
Yale School of Medicine
New Haven, Connecticut, United States, 06510
United States, Illinois
Clinical Trials Unit
Chicago, Illinois, United States, 60611
Northwestern University
Chicago, Illinois, United States, 60611
University of Chicago Medical Center
Chicago, Illinois, United States, 60637
University of Chicago Medical Center IRB
Chicago, Illinois, United States, 60637
Advocate Christ Medical Center
Oak Lawn, Illinois, United States, 60453
Clinical Research Office
Oak Lawn, Illinois, United States, 60453
United States, Louisiana
John Ochsner Heart & Vascular Institute
New Orleans, Louisiana, United States, 70121
United States, Maryland
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21205
Johns Hopkins Hospital 2
Baltimore, Maryland, United States, 21205
University of Maryland
Baltimore, Maryland, United States, 21201
University of Maryland School of Medicine
Baltimore, Maryland, United States, 21201
United States, Massachusetts
Harvard Vanguard Medical Associates
Boston, Massachusetts, United States, 02215
United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48109
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
United States, New York
Columbia University Medical Center 1
New York, New York, United States, 10032
Columbia University Medical Center 2
New York, New York, United States, 10034
Columbia University Medical Center 3
New York, New York, United States, 10034
NYU Medical Center - VA Clinic
New York, New York, United States, 10010
NYU Medical Center, Dysautonomia Center
New York, New York, United States, 10016
United States, Ohio
Heart Failure and Cardiac Transplantation Medicine,Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44195
Ohio State University College of Medicine, Neuromuscular Division
Columbus, Ohio, United States, 43210
United States, Oklahoma
The University of Oklahoma Health Sciences Center - Outpatient Office
Oklahoma City, Oklahoma, United States, 73104
United States, Oregon
Oregon Health Sciences University (OHSU)
Portland, Oregon, United States, 97201
United States, Pennsylvania
Temple University School of Medicine
Philadelphia, Pennsylvania, United States, 19140
Temple University School of Medicine - Admin Office
Philadelphia, Pennsylvania, United States, 19140
University of Pennsylvania, Presbyterian Medical Center, Philadelphia Heart Institute
Philadelphia, Pennsylvania, United States, 19104
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
University of Pennsylvania, Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, United States, 19104
Temple University School of Medicine, Departments of Neurology and Pharmacology
Philadelphia, Pennsylvania, United States, 19140
Admin
Pittsburgh, Pennsylvania, United States, 15213
Allegheny General Hospital, Allegheny Singer Research Institute, Cardiology Research
Pittsburgh, Pennsylvania, United States, 15212
Office of Sponsored Research
Pittsburgh, Pennsylvania, United States, 15212
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States, 15260
United States, Tennessee
IRB
Nashville, Tennessee, United States, 37232
Vanderbilt University School of Medicine
Nashville, Tennessee, United States, 37232
Argentina
Hospital Italiano de Buenos Aires
Buenos Aires, Argentina, C1181ACH
Instituto de Investigaciones Neurológicas Raúl Carrea
Buenos Aires, Argentina, C1428AQK
Belgium
Afdeling Klinische Cardiologie, O&N I
Leuven, Belgium, 3000
Brazil
Hospital Universitario Clementino Fraga FilhoRua Rodolpho Paulo Rocco
Ilha do Fundao, Rio de Janeiro, Brazil, 21941-913
Hospital das Clinicas de Ribeirão Preto, Unidade de Pesquisa Clinica - UPC
Monte Alegre, Ribeirão Preto, Brazil, 14048-900
Bulgaria
Alexandrovska University Hospital Clinic of Neurology
Sofia, Bulgaria, 1431
Cyprus
Cyprus Institute of Neurology and Genetics
Nicosia, Cyprus, 23462
Denmark
Aarhus University Hospital, Skejby
Aarhus N, Denmark, 8200
France
CHU Henri Mondor
Créteil, France, 94000
Hopital Pierre Zobda-Quitman - CHU de Fort de France
Fort de France, France, 97200
Centre National De Reference Maladies Rares Atteintes Vasculaires De La Sclerodermie Systemique
Lille Cedex, France, 59037
CHU Henri Mondor- Other
Paris, France, 75013
CHU Henri Mondor-DRCD
Paris cedex 10, France, 75475
Germany
Medical University of Heidelberg, Department of Cardiology, Angiology, Respiratory Medicine
Heidelberg, Germany, D-69120
Johann Gutenberg Universität Mainz
Mainz, Germany, 55131
Universitatsklinikum Muenster - Transplant Hepatology
Muenster, Germany, 48149
Italy
Comitato Etico Indipendente dell Azienda Ospedaliero-Universitaria di Bologna Policlinico
Bologna, Italy, 40138
Nefrologia, Dialisi e Medicina dei Trapianti
Firenze, Italy, 50134
AOU Policlinico G. Martino - Messina - Comitato Etico Scientifico
Messina, Italy, 98125
AOU Policlinico G. Martino - Messina - Dr. Vita
Messina, Italy, 98125
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
Pavia, Italy, 27100
Japan
Shinshu University School of Medicine
Matsumoto, JP, Japan, 390-8621
Kumamoto University, Department of Diagnostic Medicine
Kumamoto, Japan, 860-8556
Mexico
Departamento de Neurologia Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran
Tlalpan, Mexico, 14000
Portugal
Hospital Santa Maria - Comissão de Ética
Lisboa, Portugal, 1649-035
Hospital Santa Maria - Centro Estudos Egas Moniz
Lisboa, Portugal, 1649-035
Hospital Santa Maria Servico de Neurologia
Lisbon, Portugal, 1649-035
Unidade Clinica de Paramiloidose Hospital Geral de Santo Antonio
Porto, Portugal, 4099001
Spain
Hospital Gregorio Marañón
Madrid, Spain., Spain, 28007
Hospital Clinic Villarroel
Barcelona, Spain, 8036
Hospital Clinic Villarroel 2
Barcelona, Spain, 8036
Hospital Universitari de Bellvitge
Barcelona, Spain
Hospital Clinic Villarroel 3
Barcelona, Spain, 8036
Hospital Donostia
Donostia-San Sebastian, Spain, 20012
Hospital Universitario Puerta de Hierro
Majadahonda, Spain, 28222
Hospital Son Llatzer
Palma de Mallorca, Spain, 07198
Sweden
Piteå Älvdals Hospital
Piteå, Sweden, 941 50
Karolinska University Hospital
Stockholm, Sweden, 141 86
Umeå University Hospital
Umeå, Sweden
Turkey
Istanbul University,Istanbul Faculty of Medicine,Department of Neurology
Istanbul, Turkey, 34093
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
No publications provided by Pfizer

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00628745     History of Changes
Other Study ID Numbers: B3461001, FX-R-001
Study First Received: February 25, 2008
Last Updated: October 31, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Pfizer:
TRANSTHYRETIN
AMYLOIDOSIS
TRANSTHYRETIN AMYLOIDOSIS

Additional relevant MeSH terms:
Amyloid Neuropathies, Familial
Amyloidosis
Amyloid Neuropathies
Amyloidosis, Familial
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases
Neurodegenerative Diseases
Neuromuscular Diseases
Peripheral Nervous System Diseases
Proteostasis Deficiencies

ClinicalTrials.gov processed this record on November 25, 2014