Transthyretin-Associated Amyloidoses Outcome Survey (THAOS)

This study is currently recruiting participants.
Verified April 2014 by Pfizer
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00628745
First received: February 25, 2008
Last updated: April 11, 2014
Last verified: April 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
Transthyretin Mutations
Transthyretin Amyloidosis
Other: None. Observational Study.

Study Type: Observational
Study Design: Time Perspective: Prospective
Official Title: 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: 1000
Study Start Date: December 2007
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: December 2017 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Observational 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
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
Pfizer Investigational Site Recruiting
Phoenix, Arizona, United States, 85054
United States, California
Pfizer Investigational Site Recruiting
Orange, California, United States, 92868
Pfizer Investigational Site Recruiting
San Francisco, California, United States, 94117
Pfizer Investigational Site Recruiting
San Francisco, California, United States, 94143
Pfizer Investigational Site Recruiting
Stanford, California, United States, 94305-5406
United States, Colorado
Pfizer Investigational Site Recruiting
Aurora, Colorado, United States, 80045
United States, Illinois
Pfizer Investigational Site Recruiting
Chicago, Illinois, United States, 60611
Pfizer Investigational Site Recruiting
Chicago, Illinois, United States, 60637
Pfizer Investigational Site Recruiting
Oak Lawn, Illinois, United States, 60453
United States, Maryland
Pfizer Investigational Site Recruiting
Baltimore, Maryland, United States, 21201
Pfizer Investigational Site Recruiting
Baltimore, Maryland, United States, 21205
United States, Massachusetts
Pfizer Investigational Site Recruiting
Boston, Massachusetts, United States, 02215
United States, Michigan
Pfizer Investigational Site Recruiting
Ann Arbor, Michigan, United States, 48109
United States, Minnesota
Pfizer Investigational Site Recruiting
Rochester, Minnesota, United States, 55905
United States, Missouri
Pfizer Investigational Site Not yet recruiting
Saint Louis, Missouri, United States, 63104
United States, New York
Pfizer Investigational Site Recruiting
New York, New York, United States, 10034
Pfizer Investigational Site Terminated
New York, New York, United States, 10010
Pfizer Investigational Site Recruiting
New York, New York, United States, 10032
Pfizer Investigational Site Terminated
New York, New York, United States, 10016
United States, Ohio
Pfizer Investigational Site Recruiting
Cleveland, Ohio, United States, 44195
Pfizer Investigational Site Recruiting
Columbus, Ohio, United States, 43210
United States, Oklahoma
Pfizer Investigational Site Not yet recruiting
Oklahoma City, Oklahoma, United States, 73104
United States, Oregon
Pfizer Investigational Site Recruiting
Portland, Oregon, United States, 97201
United States, Pennsylvania
Pfizer Investigational Site Recruiting
Philadelphia, Pennsylvania, United States, 19140
Pfizer Investigational Site Recruiting
Philadelphia, Pennsylvania, United States, 19104
Pfizer Investigational Site Recruiting
Pittsburgh, Pennsylvania, United States, 15212
Pfizer Investigational Site Terminated
Pittsburgh, Pennsylvania, United States, 15213
Pfizer Investigational Site Terminated
Pittsburgh, Pennsylvania, United States, 15260
United States, Tennessee
Pfizer Investigational Site Recruiting
Nashville, Tennessee, United States, 37232
Argentina
Pfizer Investigational Site Recruiting
Buenos Aires, Argentina, C1428AQK
Pfizer Investigational Site Not yet recruiting
Buenos Aires, Argentina, C1181ACH
Belgium
Pfizer Investigational Site Recruiting
Leuven, Belgium, 3000
Brazil
Pfizer Investigational Site Recruiting
Ilha do Fundao, Rio de Janeiro, Brazil, 21941-913
Pfizer Investigational Site Not yet recruiting
Monte Alegre, Ribeirão Preto, Brazil, 14048-900
Bulgaria
Pfizer Investigational Site Not yet recruiting
Sofia, Bulgaria, 1431
Denmark
Pfizer Investigational Site Recruiting
Aarhus N, Denmark, 8200
France
Pfizer Investigational Site Recruiting
Créteil, France, 94000
Pfizer Investigational Site Recruiting
Fort de France, France, 97200
Pfizer Investigational Site Recruiting
Lille Cedex, France, 59037
Pfizer Investigational Site Recruiting
Paris, France, 75013
Pfizer Investigational Site Recruiting
Paris cedex 10, France, 75475
Germany
Pfizer Investigational Site Recruiting
Heidelberg, Germany, D-69120
Pfizer Investigational Site Not yet recruiting
Mainz, Germany, 55131
Pfizer Investigational Site Recruiting
Muenster, Germany, 48149
Italy
Pfizer Investigational Site Recruiting
Bologna, Italy, 40138
Pfizer Investigational Site Not yet recruiting
Firenze, Italy, 50134
Pfizer Investigational Site Recruiting
Messina, Italy, 98125
Pfizer Investigational Site Recruiting
Pavia, Italy, 27100
Japan
Pfizer Investigational Site Recruiting
Matsumoto, JP, Japan
Pfizer Investigational Site Recruiting
Kumamoto, Japan
Mexico
Pfizer Investigational Site Recruiting
Tlalpan, Mexico, 14000
Portugal
Pfizer Investigational Site Recruiting
Lisboa, Portugal, 1649-035
Pfizer Investigational Site Recruiting
Lisbon, Portugal, 1649-035
Pfizer Investigational Site Recruiting
Porto, Portugal, 4099001
Spain
Pfizer Investigational Site Recruiting
Madrid, Spain., Spain, 28007
Pfizer Investigational Site Recruiting
Barcelona, Spain, 8036
Pfizer Investigational Site Recruiting
Barcelona, Spain
Pfizer Investigational Site Recruiting
Donostia-San Sebastian, Spain, 20012
Pfizer Investigational Site Recruiting
Majadahonda, Spain, 28222
Pfizer Investigational Site Recruiting
Palma de Mallorca, Spain, 07198
Sweden
Pfizer Investigational Site Recruiting
Piteå, Sweden, 941 50
Pfizer Investigational Site Not yet recruiting
Stockholm, Sweden, 141 86
Pfizer Investigational Site Recruiting
Umeå, Sweden
Turkey
Pfizer Investigational Site Recruiting
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: April 11, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Pfizer:
TRANSTHYRETIN
AMYLOIDOSIS
TRANSTHYRETIN AMYLOIDOSIS

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases

ClinicalTrials.gov processed this record on April 17, 2014