Transthyretin-Associated Amyloidoses Outcome Survey (THAOS)

This study is currently recruiting participants. (see Contacts and Locations)
Verified October 2015 by Pfizer
Information provided by (Responsible Party):
Pfizer Identifier:
First received: February 25, 2008
Last updated: October 6, 2015
Last verified: October 2015

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:

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)
Groups/Cohorts Assigned Interventions
Observational Other: None. Observational Study.

Detailed Description:

n/a NA


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.


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 identifier: NCT00628745

Contact: Pfizer Call Center 1-800-718-1021

  Show 87 Study Locations
Sponsors and Collaborators
Study Director: Pfizer Call Center Pfizer
  More Information

Additional Information:
No publications provided by Pfizer

Additional publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Pfizer Identifier: NCT00628745     History of Changes
Other Study ID Numbers: B3461001, FX-R-001
Study First Received: February 25, 2008
Last Updated: October 6, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by Pfizer:

Additional relevant MeSH terms:
Amyloid Neuropathies, Familial
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 processed this record on October 13, 2015