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Transthyretin-Associated Amyloidoses Outcome Survey (THAOS) (THAOS)
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Purpose
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.
| Condition | Intervention |
|---|---|
| Transthyretin Mutations Transthyretin Amyloidosis | Other: None. Observational Study. |
| Study Type: | Observational |
| Study Design: | Observational Model: Other Time Perspective: Prospective |
| Official Title: | 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 |
- 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 ]
Biospecimen Retention: Samples With DNA
| Estimated Enrollment: | 1 |
| Actual Study Start Date: | July 27, 2007 |
| Estimated Study Completion Date: | June 16, 2021 |
| Estimated Primary Completion Date: | June 16, 2021 (Final data collection date for primary outcome measure) |
| Groups/Cohorts | Assigned Interventions |
|---|---|
| Observational | Other: None. Observational Study. |
Detailed Description:
Eligibility| Ages Eligible for Study: | 18 Years and older (Adult, Senior) |
| 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 enrollment into THAOS:
- 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.
- Males and females 18 years of age.
-
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):
- 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
- 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
- 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.
Contacts and LocationsPlease refer to this study by its ClinicalTrials.gov identifier: NCT00628745
| Contact: Pfizer CT.gov Call Center | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
Show 97 Study Locations
| Study Director: | Pfizer CT.gov Call Center | Pfizer |
More Information
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Pfizer |
| ClinicalTrials.gov Identifier: | NCT00628745 History of Changes |
| Other Study ID Numbers: |
B3461001 FX-R-001 ( Other Identifier: Alias Study Number ) THAOS ( Other Identifier: Alias Study Number ) |
| Study First Received: | February 25, 2008 |
| Last Updated: | September 6, 2017 |
| Studies a U.S. FDA-regulated Drug Product: | Yes | |
| Studies a U.S. FDA-regulated Device Product: | No | |
Keywords provided by Pfizer:
|
TRANSTHYRETIN AMYLOIDOSIS TRANSTHYRETIN AMYLOIDOSIS |
Additional relevant MeSH terms:
|
Amyloidosis Amyloid Neuropathies, Familial Amyloid Neuropathies Amyloidosis, Familial Proteostasis Deficiencies Metabolic Diseases Heredodegenerative Disorders, Nervous System |
Neurodegenerative Diseases Nervous System Diseases Peripheral Nervous System Diseases Neuromuscular Diseases Genetic Diseases, Inborn Metabolism, Inborn Errors |
ClinicalTrials.gov processed this record on September 21, 2017