Transthyretin-Associated Amyloidoses Outcome Survey (THAOS)

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by Pfizer
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00628745
First received: February 25, 2008
Last updated: August 19, 2014
Last verified: August 2014

February 25, 2008
August 19, 2014
December 2007
December 2017   (final data collection date for primary outcome measure)
Not Provided
Not Provided
Complete list of historical versions of study NCT00628745 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
Transthyretin-Associated Amyloidoses Outcome Survey (THAOS)
A Global, Multi-Center, Longitudinal, Observational Survey Of Patients With Documented Transthyretin (TTR) Mutations Or Wild-Type TTR Amyloidosis

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.

n/a NA

Observational
Time Perspective: Prospective
Not Provided
Retention:   Samples With DNA
Description:

whole blood

Non-Probability Sample

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

  • Transthyretin Mutations
  • Transthyretin Amyloidosis
Other: None. Observational Study.
None.
Observational
Intervention: Other: None. Observational Study.
Coelho T, Maurer MS, Suhr OB. THAOS - The Transthyretin Amyloidosis Outcomes Survey: initial report on clinical manifestations in patients with hereditary and wild-type transthyretin amyloidosis. Curr Med Res Opin. 2013 Jan;29(1):63-76. doi: 10.1185/03007995.2012.754348. Epub 2012 Dec 13.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
1000
December 2017
December 2017   (final data collection date for primary outcome measure)

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.
Both
18 Years and older
No
Contact: Pfizer CT.gov Call Center 1-800-718-1021
United States,   Argentina,   Belgium,   Brazil,   Bulgaria,   Cyprus,   Denmark,   France,   Germany,   Italy,   Japan,   Mexico,   Portugal,   Spain,   Sweden,   Turkey
 
NCT00628745
B3461001, FX-R-001
No
Pfizer
Pfizer
Not Provided
Study Director: Pfizer CT.gov Call Center Pfizer
Pfizer
August 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP