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Transthyretin-Associated Amyloidoses Outcome Survey (THAOS) (THAOS)

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2016 by Pfizer
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00628745
First received: February 25, 2008
Last updated: September 6, 2016
Last verified: September 2016
  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
Transthyretin Mutations
Transthyretin Amyloidosis
Other: None. Observational Study.

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:

Primary Outcome Measures:
  • The objectives of THAOS are to describe the population of patients affected with TTR amyloidoses 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 2021 ] [ Designated as safety issue: No ]

Biospecimen Retention:   Samples With DNA
whole blood

Estimated Enrollment: 3000
Study Start Date: July 2007
Estimated Study Completion Date: June 2021
Estimated Primary Completion Date: June 2021 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Observational Other: None. Observational Study.

Detailed Description:
n/a NA
  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
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: Patients must meet all of the following inclusion criteria to be eligible for enrollment into THAOS:

  1. Evidence of a personally signed and dated informed consent document indicating that the patient (or a legally acceptable representative) has been informed of all pertinent aspects of the study.
  2. Males and females 18 years of age.
  3. Confirmed genotyped TTR mutation with or without a diagnosis of ATTR, or wild-type TTR amyloidosis. Confirmation of wild-type TTR 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. Evidence of cardiac involvement by echocardiogram as defined by mean left ventricle wall thickness of >12 mm, and 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 mean 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 mean left ventricle wall thickness of >12 mm, no evidence of primary (light chain) amyloidosis, 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 presenting with any of the following will not be included in THAOS:

1. 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

Contacts
Contact: Pfizer CT.gov Call Center 1-800-718-1021

  Hide Study Locations
Locations
United States, Arizona
Mayo Clinic Arizona Recruiting
Phoenix, Arizona, United States, 85054
United States, California
University of California, Irvine Terminated
Orange, California, United States, 92868
University of California - San Francisco, UCSF Department of Neurology Terminated
San Francisco, California, United States, 94117
Office of Sponsored Research Terminated
San Francisco, California, United States, 94143
Stanford University School of Medicine Active, not recruiting
Stanford, California, United States, 94305-5406
United States, Colorado
UC Denver Recruiting
Aurora, Colorado, United States, 80045
United States, Connecticut
Smilow Cancer Center at Yale New Haven Hospital Recruiting
New Haven, Connecticut, United States, 06510
United States, Florida
University of Miami Hospital & Clinics Recruiting
Miami, Florida, United States, 33136
United States, Illinois
Clinical Trials Unit Recruiting
Chicago, Illinois, United States, 60611
Northwestern University Recruiting
Chicago, Illinois, United States, 60611
University of Chicago Medical Center IRB Recruiting
Chicago, Illinois, United States, 60637
University of Chicago Medical Center Recruiting
Chicago, Illinois, United States, 60637
Advocate Christ Medical Center Recruiting
Oak Lawn, Illinois, United States, 60453
Clinical Research Office Recruiting
Oak Lawn, Illinois, United States, 60453
United States, Louisiana
John Ochsner Heart & Vascular Institute Recruiting
New Orleans, Louisiana, United States, 70121
United States, Maryland
University of Maryland School of Medicine Recruiting
Baltimore, Maryland, United States, 21201
University of Maryland Recruiting
Baltimore, Maryland, United States, 21201
Johns Hopkins Hospital 2 Recruiting
Baltimore, Maryland, United States, 21205
Johns Hopkins Hospital Recruiting
Baltimore, Maryland, United States, 21205
United States, Massachusetts
Harvard Vanguard Medical Associates Terminated
Boston, Massachusetts, United States, 02215
United States, Michigan
University of Michigan Recruiting
Ann Arbor, Michigan, United States, 48109
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
United States, New York
NYU Medical Center - VA Clinic Terminated
New York, New York, United States, 10010
NYU Medical Center, Dysautonomia Center Terminated
New York, New York, United States, 10016
Columbia University Medical Center 1 Recruiting
New York, New York, United States, 10032
Columbia University Medical Center 2 Recruiting
New York, New York, United States, 10034
Columbia University Medical Center 3 Recruiting
New York, New York, United States, 10034
United States, Ohio
Heart Failure and Cardiac Transplantation Medicine,Cleveland Clinic Foundation Terminated
Cleveland, Ohio, United States, 44195
The Ohio University College of Medicine Recruiting
Columbus, Ohio, United States, 43210
United States, Oregon
Oregon Health Sciences University (OHSU) Terminated
Portland, Oregon, United States, 97201
United States, Pennsylvania
University of Pennsylvania, Penn Presbyterian Medical Center Recruiting
Philadelphia, Pennsylvania, United States, 19104
University of Pennsylvania, Presbyterian Medical Center, Philadelphia Heart Institute Recruiting
Philadelphia, Pennsylvania, United States, 19104
University of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19104
Temple University School of Medicine - Admin Office Terminated
Philadelphia, Pennsylvania, United States, 19140
Temple University School of Medicine, Departments of Neurology and Pharmacology Terminated
Philadelphia, Pennsylvania, United States, 19140
Temple University School of Medicine Terminated
Philadelphia, Pennsylvania, United States, 19140
Allegheny General Hospital, Allegheny Singer Research Institute, Cardiology Research Recruiting
Pittsburgh, Pennsylvania, United States, 15212
Office of Sponsored Research Recruiting
Pittsburgh, Pennsylvania, United States, 15212
Admin Terminated
Pittsburgh, Pennsylvania, United States, 15213
University of Pittsburgh Medical Center Terminated
Pittsburgh, Pennsylvania, United States, 15260
United States, Tennessee
IRB Recruiting
Nashville, Tennessee, United States, 37232
Vanderbilt University School of Medicine Recruiting
Nashville, Tennessee, United States, 37232
Argentina
Hospital Italiano de Buenos Aires Recruiting
Buenos Aires, Argentina, C1199ABD
Instituto de Investigaciones Neurológicas Raúl Carrea Recruiting
Buenos Aires, Argentina
Belgium
Afdeling Klinische Cardiologie, O&N I Recruiting
Leuven, Belgium, 3000
Brazil
Hospital Universitario Clementino Fraga Filho Recruiting
Rio de Janeiro, Brazil, 21941-913
Bulgaria
Alexandrovska University Hospital Clinic of Neurology Recruiting
Sofia, Bulgaria, 1431
Canada, Alberta
University of Alberta Foothills Medical Centre Recruiting
Calgary, Alberta, Canada, T2N 4Z6
Cyprus
Cyprus Institute of Neurology and Genetics Terminated
Nicosia, Cyprus, 23462
Denmark
Aarhus University Hospital, Skejby Recruiting
Aarhus N, Denmark, 8200
France
CHU de Bicetre Recruiting
Cedex, France, 94275
CHU Henri Mondor Recruiting
Créteil, France, 94000
Chu De Fort De France Recruiting
Fort De France, France, 97200
Centre National De Reference Maladies Rares Atteintes Vasculaires De La Sclerodermie Systemique Recruiting
Lille Cedex, France, 59037
CHU Henri Mondor-DRCD Recruiting
Paris cedex 10, France, 75475
CHU Henri Mondor- Other Recruiting
Paris, France, 75013
Germany
University Hospital of RWTH Aachen Recruiting
Aachen, Germany, 52074
Medical University of Heidelberg Recruiting
Heidelberg, Germany, D-69120
Johann Gutenberg Universität Mainz Recruiting
Mainz, Germany, 55131
Universitatsklinikum Muenster - Transplant Hepatology Recruiting
Muenster, Germany, 48149
Israel
Wolfson Medical Center, Department of Neurology Terminated
Holon, Israel, 58100
Italy
Comitato Etico Indipendente dell Azienda Recruiting
Bologna, Italy, 40138
Azienda Ospedaliero-Universitaria di Careggi Recruiting
Firenze, Italy, 50134
AOU Policlinico G. Martino - Messina - Comitato Etico Scientifico Recruiting
Messina, Italy, 98125
AOU Policlinico G. Martino - Messina - Dr. Vita Recruiting
Messina, Italy, 98125
Centro per lo Studio e la Cura delle Amiloidosi Sistemiche - Pavia - Prof. Merlini Recruiting
Pavia, Italy, 27100
Comitato di Bioetica della Fondazione IRCCS Policlinico S. Matteo Recruiting
Pavia, Italy, 27100
Japan
Shinshu University School of Medicine Recruiting
Matsumoto, JP, Japan, 390-8261
Chiba University Hospital Recruiting
Chiba, Japan, 260-8677
Kumamoto University Recruiting
Kumamoto, Japan, 860-8556
Korea, Republic of
Konkuk University Medical Center Recruiting
Seoul, Korea, Republic of, 05030
Samsung Medical Center, Sungkyunkwan University School of Medicine Recruiting
Seoul, Korea, Republic of, 135-710
Mexico
Departamento de Neurologia Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran Recruiting
Tlalpan, Mexico, 14000
Netherlands
University Medical Center Groningen Recruiting
Groningen, Netherlands, 9713 GZ
Portugal
Centro Hospitalar Do Alto Ave, Epe Not yet recruiting
Guimaraes, Portugal, 4835-004
Hospital Santa Maria - Centro Estudos Egas Moniz Recruiting
Lisboa, Portugal, 1649-035
Hospital Santa Maria - Comissão de Ética Recruiting
Lisboa, Portugal, 1649-035
Hospital Santa Maria Servico de Neurologia Recruiting
Lisbon, Portugal, 1649-035
Unidade Clinica de Paramiloidose Hospital Geral de Santo Antonio Recruiting
Porto, Portugal, 4099001
Spain
Hospital Universitari de Bellvitge Recruiting
Barcelona, Spain, 0000
Hospital Clinic Villarroel 2 Recruiting
Barcelona, Spain, 8036
Hospital Clinic Villarroel 3 Recruiting
Barcelona, Spain, 8036
Hospital Clinic Villarroel Recruiting
Barcelona, Spain, 8036
Hospital Clinic Villarroel 4 Recruiting
Barcelona, Spain
Hospital Donostia Recruiting
Donostia-San Sebastian, Spain, 20012
Hospital de Rehabilitacion y Traumatologia Virgen de las Nieves Recruiting
Granada, Spain, 18013
Hospital Juan Ramon Jimenez Not yet recruiting
Huelva, Spain, 21005
Hospital Gregorio Marañón Recruiting
Madrid, Spain, 28007
Hospital Universitario Puerta de Hierro Recruiting
Majadahonda, Spain, 28222
Hospital Son Llatzer Recruiting
Palma de Mallorca, Spain, 07198
Sweden
Piteå Älvdals Hospital Recruiting
Piteå, Sweden, 941 50
Karolinska University Hospital, Huddinge Not yet recruiting
Stockholm, Sweden, 141 86
Umeå University Hospital Recruiting
Umeå, Sweden
Taiwan
National Taiwan University Hospital Terminated
Taipei, Taiwan, 10018
Turkey
Istanbul University,Istanbul Faculty of Medicine,Department of Neurology Recruiting
Istanbul, Turkey, 34093
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
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: September 6, 2016
Health Authority: United States: Food and Drug Administration

Keywords provided by Pfizer:
TRANSTHYRETIN
AMYLOIDOSIS
TRANSTHYRETIN AMYLOIDOSIS

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

ClinicalTrials.gov processed this record on September 27, 2016