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

This study is currently recruiting participants.
Verified October 2017 by Pfizer
Sponsor:
ClinicalTrials.gov Identifier:
NCT00628745
First Posted: March 5, 2008
Last Update Posted: October 3, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Pfizer
February 25, 2008
March 5, 2008
October 3, 2017
July 27, 2007
June 16, 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 ]
Not Provided
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
Observational Model: Other
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.
Observational
Intervention: Other: None. Observational Study.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
1
June 16, 2021
June 16, 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.

Sexes Eligible for Study: All
18 Years and older   (Adult, Senior)
No
Contact: Pfizer CT.gov Call Center 1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com
Argentina,   Belgium,   Brazil,   Bulgaria,   Canada,   Cyprus,   Denmark,   France,   Germany,   Israel,   Italy,   Japan,   Korea, Republic of,   Mexico,   Netherlands,   Portugal,   Spain,   Sweden,   Taiwan,   Turkey,   United States
Australia,   Austria,   Greece,   Switzerland
 
NCT00628745
B3461001
FX-R-001 ( Other Identifier: Alias Study Number )
THAOS ( Other Identifier: Alias Study Number )
No
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Not Provided
Pfizer
Pfizer
Not Provided
Study Director: Pfizer CT.gov Call Center Pfizer
Pfizer
October 2017