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

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2016 by Pfizer
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00628745
First received: February 25, 2008
Last updated: July 11, 2016
Last verified: July 2016

February 25, 2008
July 11, 2016
July 2007
June 2021   (final data collection date for primary outcome measure)
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 ]
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Complete list of historical versions of study NCT00628745 on ClinicalTrials.gov Archive Site
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Transthyretin-Associated Amyloidoses Outcome Survey (THAOS)
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

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
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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.
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
3000
June 2021
June 2021   (final data collection date for primary outcome measure)

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.

Both
18 Years and older   (Adult, Senior)
No
Contact: Pfizer CT.gov Call Center 1-800-718-1021
United States,   Argentina,   Belgium,   Brazil,   Bulgaria,   Canada,   Cyprus,   Denmark,   France,   Germany,   Israel,   Italy,   Japan,   Korea, Republic of,   Mexico,   Netherlands,   Portugal,   Spain,   Sweden,   Taiwan,   Turkey
Australia,   Austria,   Greece,   Switzerland
 
NCT00628745
B3461001, FX-R-001
No
Not Provided
Not Provided
Pfizer
Pfizer
Not Provided
Study Director: Pfizer CT.gov Call Center Pfizer
Pfizer
July 2016

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