Status:

COMPLETED

Transthyretin Amyloidosis Outcome Survey (THAOS)

Lead Sponsor:

Pfizer

Conditions:

Transthyretin Gene Mutations

Transthyretin Amyloidosis

Eligibility:

All Genders

18+ years

Brief Summary

THAOS is a global, multi-center, longitudinal observational survey open to all patients with transthyretin amyloidosis (ATTR), including ATTR-PN (polyneuropathy), ATTR-CM (cardiomyopathy) and wild-typ...

Detailed Description

n/a NA

Eligibility Criteria

Inclusion

  • Inclusion Criteria: Patients must meet all of the following inclusion criteria to be eligible for inclusion into THAOS:
  • Evidence of a personally signed and dated informed consent document indicating that the participant (or a legally acceptable representative) has been informed of all pertinent aspects of the study.
  • Males and females greater than or equal to 18 years of age.
  • Confirmed genotyped TTR mutation with or without a diagnosis of hereditary or wild-type ATTR amyloidosis. Confirmation of ATTRwt 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):
  • Presence of amyloid in cardiac biopsy tissue confirmed as TTR amyloid by mass spectrometry or immunohistochemistry; or
  • Evidence of cardiac involvement by echocardiogram as defined by 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
  • Evidence of cardiac involvement by echocardiogram as defined by left ventricle wall thickness of \>12 mm, 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 meeting any of the following will not be included in the study:
  • 1\. Patient has evidence of primary (light chain) or secondary amyloidosis.

Exclusion

    Key Trial Info

    Start Date :

    January 4 2008

    Trial Type :

    OBSERVATIONAL

    Allocation :

    ACTUAL

    End Date :

    June 19 2023

    Estimated Enrollment :

    6718 Patients enrolled

    Trial Details

    Trial ID

    NCT00628745

    Start Date

    January 4 2008

    End Date

    June 19 2023

    Last Update

    November 22 2024

    Active Locations (113)

    Enter a location and click search to find clinical trials sorted by distance.

    Page 1 of 29 (113 locations)

    1

    University of Alabama at Birmingham

    Birmingham, Alabama, United States, 35294

    2

    Mayo Clinic Arizona

    Phoenix, Arizona, United States, 85054

    3

    VA Greater Los Angeles Healthcare System

    Los Angeles, California, United States, 90073

    4

    University of California, Irvine

    Orange, California, United States, 92868