Transthyretin Amyloidosis Outcome Survey (THAOS) (THAOS)
|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. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00628745|
Recruitment Status : Active, not recruiting
First Posted : March 5, 2008
Last Update Posted : April 27, 2023
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|Condition or disease||Intervention/treatment|
|Transthyretin Gene Mutations Transthyretin Amyloidosis||Other: None. Observational Study.|
|Study Type :||Observational|
|Actual Enrollment :||6224 participants|
|Official Title:||Transthyretin Amyloidosis Outcomes Survey (THAOS): A Global, Multi-Center, Longitudinal, Observational Survey of Patients With Documented Transthyretin Gene Mutations or Wild-Type Transthyretin Amyloidosis.|
|Actual Study Start Date :||January 4, 2008|
|Estimated Primary Completion Date :||June 16, 2023|
|Estimated Study Completion Date :||June 16, 2023|
Other: None. Observational Study.
- The objectives of THAOS are to describe the population of patients affected with TTR amyloidosis 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 2023 ]Cardiovascular and Neurological efficacy endpoints for analysis of clinical outcomes on all enrolled patients with available data. Outcomes will be examined for the entire patient group, as well as through subgroups based on important variables.
Biospecimen Retention: Samples With DNA
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|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
|Sampling Method:||Non-Probability Sample|
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.
Patients meeting any of the following will not be included in the study:
1. Patient has evidence of primary (light chain) or secondary amyloidosis.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00628745
|Study Director:||Pfizer CT.gov Call Center||Pfizer|
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
|Other Study ID Numbers:||
FX-R-001 ( Other Identifier: Alias Study Number )
THAOS ( Other Identifier: Alias Study Number )
|First Posted:||March 5, 2008 Key Record Dates|
|Last Update Posted:||April 27, 2023|
|Last Verified:||April 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Plan Description:||Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Transthyretin amyloid cardiomyopathy
Amyloid Neuropathies, Familial
Heredodegenerative Disorders, Nervous System
Nervous System Diseases
Peripheral Nervous System Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors