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Transthyretin-Associated Amyloidoses Outcome Survey (THAOS)

Last updated on June 16, 2018

FOR MORE INFORMATION
Study Location
University of Alabama at Birmingham
Birmingham, Alabama, 35294 United States
Contact
1-800-718-1021
Eligibility criteria
Condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Transthyretin Mutations, Transthyretin Amyloidosis
Sex
Females and Males
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
18+ years
Inclusion criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

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
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details

Patients presenting with any of the following will not be included in THAOS:

1. Patient has primary or secondary amyloidosis.

NCT00628745
Pfizer
Recruiting
Transthyretin-Associated Amyloidoses Outcome Survey (THAOS)

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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 [email protected]
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
November 2017

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