Transthyretin-Associated Amyloidoses Outcome Survey (THAOS)

Trial Purpose and Description

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.

Ages: 18 years and older

Gender: Both


Eligibility Criteria

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):

a. 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

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 mass spectrometry or immunohistochemistry; or

c. 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.

Pfizer Inc., U.S. Pharmaceuticals Group

Dates: 06/04/2014 - 06/30/2021

Last Updated: 03/23/2016

Study HIC#: 1306012239

Get Involved

For more information about this study, contact:
Lynn Marie Wilson
203-737-8871
lynn.wilson@ynhh.org

If you would prefer to contact a member of the Help us Discover team about this trial and other similar trials, please email helpusdiscover@yale.edu or call 1-877-978-8348.

Trial Image

Investigators

Daniel Louis Jacoby

Principal Investigator

Sub-Investigators