Transthyretin-Associated Amyloidoses Outcome Survey (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: | Time Perspective: Prospective |
| Official Title: | A Global, Multi-Center, Longitudinal, Observational Survey Of Patients With Documented Transthyretin (TTR) Mutations Or Wild-Type TTR Amyloidosis |
whole blood
| Estimated Enrollment: | 1000 |
| Study Start Date: | December 2007 |
| Estimated Study Completion Date: | December 2017 |
| Estimated Primary Completion Date: | December 2017 (Final data collection date for primary outcome measure) |
| Groups/Cohorts | Assigned Interventions |
|---|---|
| Observational |
Other: None. Observational Study.
None.
|
Detailed Description:
n/a NA
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
Participants with transthyretin-associated amyloidoses (ATTR), including ATTR-PN (polyneuropathy), ATTR-CM (cardiomyopathy) and wild-type ATTR-CM.
Inclusion Criteria:
- Written informed consent.
- Patient has confirmed: genotyped TTR mutation with or without a diagnosis of TTR-associated amyloidosis (e.g., ATTR-PN, ATTR-CM), or Wild-type TTR-associated amyloidosis with cardiomyopathy (wild-type ATTR-CM).
- Confirmation of wild-type ATTR-CM will be determined by one of the following set of criteria (A or B):
A. Presence of amyloid in cardiac biopsy tissue confirmed as TTR amyloid by immunohistochemistry and genotyped confirmation that patient does not possess a known mutation in TTR gene (i.e., is a carrier of wild-type allele only) via genetic testing; 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 immunohistochemistry, and genotyped confirmation that patient does not possess a known mutation in TTR gene (i.e., is a carrier of wild-type allele only) via genetic testing.
Exclusion Criteria:
- Patient has primary or secondary amyloidosis.
Contacts and Locations| Contact: Pfizer CT.gov Call Center | 1-800-718-1021 |
Hide Study Locations| United States, California | |
| Pfizer Investigational Site | Recruiting |
| Orange, California, United States, 92868 | |
| Pfizer Investigational Site | Recruiting |
| San Francisco, California, United States, 94117 | |
| Pfizer Investigational Site | Recruiting |
| Stanford, California, United States, 94305-5406 | |
| United States, Colorado | |
| Pfizer Investigational Site | Recruiting |
| Aurora, Colorado, United States, 80045 | |
| United States, Illinois | |
| Pfizer Investigational Site | Recruiting |
| Chicago, Illinois, United States, 60637 | |
| Pfizer Investigational Site | Recruiting |
| Chicago, Illinois, United States, 60611 | |
| Pfizer Investigational Site | Recruiting |
| Oak Lawn, Illinois, United States, 60453 | |
| United States, Maryland | |
| Pfizer Investigational Site | Recruiting |
| Baltimore, Maryland, United States, 21201 | |
| Pfizer Investigational Site | Recruiting |
| Baltimore, Maryland, United States, 21205 | |
| United States, Massachusetts | |
| Pfizer Investigational Site | Recruiting |
| Boston, Massachusetts, United States, 02215 | |
| United States, Michigan | |
| Pfizer Investigational Site | Not yet recruiting |
| Ann Arbor, Michigan, United States, 48109 | |
| United States, New York | |
| Pfizer Investigational Site | Recruiting |
| New York, New York, United States, 10032 | |
| Pfizer Investigational Site | Terminated |
| New York, New York, United States, 10016 | |
| United States, Ohio | |
| Pfizer Investigational Site | Recruiting |
| Cleveland, Ohio, United States, 44195 | |
| Pfizer Investigational Site | Recruiting |
| Columbus, Ohio, United States, 43210 | |
| United States, Oregon | |
| Pfizer Investigational Site | Recruiting |
| Portland, Oregon, United States, 97239-3098 | |
| United States, Pennsylvania | |
| Pfizer Investigational Site | Recruiting |
| Philadelphia, Pennsylvania, United States, 19104 | |
| Pfizer Investigational Site | Recruiting |
| Philadelphia, Pennsylvania, United States, 19140 | |
| Pfizer Investigational Site | Terminated |
| Pittsburgh, Pennsylvania, United States, 15213 | |
| Pfizer Investigational Site | Recruiting |
| Pittsburgh, Pennsylvania, United States, 15212-4772 | |
| United States, Tennessee | |
| Pfizer Investigational Site | Not yet recruiting |
| Nashville, Tennessee, United States, 37232 | |
| Argentina | |
| Pfizer Investigational Site | Recruiting |
| Ciudad de Buenos Aires, Argentina, C1428AQK | |
| Belgium | |
| Pfizer Investigational Site | Recruiting |
| Leuven, Belgium, 3000 | |
| Brazil | |
| Pfizer Investigational Site | Recruiting |
| Ilha do Fundao, Rio de Janeiro, Brazil, 21941-913 | |
| Pfizer Investigational Site | Not yet recruiting |
| Conceicao de Monte Alegre, Brazil, 14048-900 | |
| Cyprus | |
| Pfizer Investigational Site | Recruiting |
| Nicosia, Cyprus, 23462, 1683 | |
| Denmark | |
| Pfizer Investigational Site | Recruiting |
| Arhus N, Denmark, 8200 | |
| France | |
| Pfizer Investigational Site | Recruiting |
| Creil, France, 94000 | |
| Germany | |
| Pfizer Investigational Site | Recruiting |
| Heidelberg, Germany, D-69120 | |
| Pfizer Investigational Site | Recruiting |
| Munster, Germany, 48149 | |
| Israel | |
| Pfizer Investigational Site | Recruiting |
| Holon, Israel, 58100 | |
| Italy | |
| Pfizer Investigational Site | Recruiting |
| Bologna, Italy, 40138 | |
| Pfizer Investigational Site | Not yet recruiting |
| Firenze, Italy, 50134 | |
| Pfizer Investigational Site | Recruiting |
| Messina, Italy, 98125 | |
| Pfizer Investigational Site | Recruiting |
| Pavia, Italy, 27100 | |
| Japan | |
| Pfizer Investigational Site | Recruiting |
| Matsumoto, JP, Japan | |
| Pfizer Investigational Site | Recruiting |
| Kumamoto, Japan | |
| Mexico | |
| Pfizer Investigational Site | Recruiting |
| Tlalpan, Mexico | |
| Portugal | |
| Pfizer Investigational Site | Recruiting |
| Lisbon, Portugal, 1649-035 | |
| Pfizer Investigational Site | Recruiting |
| Porto, Portugal, 4099-001 | |
| Spain | |
| Pfizer Investigational Site | Not yet recruiting |
| Barcelona, Spain | |
| Pfizer Investigational Site | Recruiting |
| Barcelona, Spain, 08036 | |
| Pfizer Investigational Site | Recruiting |
| Donostia-San Sebastian, Spain, 20012 | |
| Pfizer Investigational Site | Not yet recruiting |
| Majadahonda, Spain, 28222 | |
| Pfizer Investigational Site | Recruiting |
| Palma de Mallorca, Spain, 07198 | |
| Sweden | |
| Pfizer Investigational Site | Recruiting |
| Umea, Sweden | |
| Taiwan | |
| Pfizer Investigational Site | Recruiting |
| Taipei, Taiwan, 10018 | |
| Pfizer Investigational Site | Recruiting |
| Taipei City, Taiwan, 11217 | |
| Turkey | |
| Pfizer Investigational Site | Recruiting |
| Istanbul, Turkey, 34093 | |
| Study Director: | Pfizer CT.gov Call Center | Pfizer |
More Information
Additional Information:
No publications provided by Pfizer
Additional 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 |
| Study First Received: | February 25, 2008 |
| Last Updated: | April 29, 2013 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Pfizer:
|
TRANSTHYRETIN AMYLOIDOSIS TRANSTHYRETIN AMYLOIDOSIS |
Additional relevant MeSH terms:
|
Amyloidosis Proteostasis Deficiencies Metabolic Diseases |
ClinicalTrials.gov processed this record on May 19, 2013