Transthyretin-Associated Amyloidoses Outcome Survey (THAOS) (THAOS)

Tracking Information
First Submitted Date	February 25, 2008
First Posted Date	March 5, 2008
Last Update Posted Date	January 23, 2018
Actual Study Start Date	July 27, 2007
Estimated Primary Completion Date	June 16, 2021 (Final data collection date for primary outcome measure)
Current Primary Outcome Measures; (submitted: October 16, 2015)	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 ]
Original Primary Outcome Measures	Not Provided
Change History	Complete list of historical versions of study NCT00628745 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures	Not Provided
Original Secondary Outcome Measures	Not Provided
Current Other Outcome Measures	Not Provided
Original Other Outcome Measures	Not Provided
Descriptive Information
Brief Title	Transthyretin-Associated Amyloidoses Outcome Survey (THAOS)
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
Brief Summary	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.
Detailed Description	n/a NA
Study Type	Observational
Study Design	Observational Model: Other; Time Perspective: Prospective
Target Follow-Up Duration	Not Provided
Biospecimen	Retention: Samples With DNA; Description: whole blood
Sampling Method	Non-Probability Sample
Study Population	Participants with transthyretin-associated amyloidoses (ATTR), including ATTR-PN (polyneuropathy), ATTR-CM (cardiomyopathy) and wild-type ATTR-CM.
Condition	Transthyretin Mutations; Transthyretin Amyloidosis
Intervention	Other: None. Observational Study.
Study Groups/Cohorts	Observational; Intervention: Other: None. Observational Study.
Publications *	Mundayat R, Stewart M, Alvir J, Short S, Ong ML, Keohane D, Rill D, Sultan MB. Positive Effectiveness of Tafamidis in Delaying Disease Progression in Transthyretin Familial Amyloid Polyneuropathy up to 2 Years: An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Neurol Ther. 2018 Apr 9. doi: 10.1007/s40120-018-0097-9. [Epub ahead of print]; Kristen AV, Maurer MS, Rapezzi C, Mundayat R, Suhr OB, Damy T; THAOS investigators. Impact of genotype and phenotype on cardiac biomarkers in patients with transthyretin amyloidosis - Report from the Transthyretin Amyloidosis Outcome Survey (THAOS). PLoS One. 2017 Apr 6;12(4):e0173086. doi: 10.1371/journal.pone.0173086. eCollection 2017.; Maurer MS, Hanna M, Grogan M, Dispenzieri A, Witteles R, Drachman B, Judge DP, Lenihan DJ, Gottlieb SS, Shah SJ, Steidley DE, Ventura H, Murali S, Silver MA, Jacoby D, Fedson S, Hummel SL, Kristen AV, Damy T, Planté-Bordeneuve V, Coelho T, Mundayat R, Suhr OB, Waddington Cruz M, Rapezzi C; THAOS Investigators. Genotype and Phenotype of Transthyretin Cardiac Amyloidosis: THAOS (Transthyretin Amyloid Outcome Survey). J Am Coll Cardiol. 2016 Jul 12;68(2):161-72. doi: 10.1016/j.jacc.2016.03.596.; Damy T, Maurer MS, Rapezzi C, Planté-Bordeneuve V, Karayal ON, Mundayat R, Suhr OB, Kristen AV. Clinical, ECG and echocardiographic clues to the diagnosis of TTR-related cardiomyopathy. Open Heart. 2016 Feb 8;3(1):e000289. doi: 10.1136/openhrt-2015-000289. eCollection 2016.; Coelho T, Maurer MS, Suhr OB. THAOS - The Transthyretin Amyloidosis Outcomes Survey: initial report on clinical manifestations in patients with hereditary and wild-type transthyretin amyloidosis. Curr Med Res Opin. 2013 Jan;29(1):63-76. doi: 10.1185/03007995.2012.754348. Epub 2012 Dec 13.
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status	Recruiting
Estimated Enrollment; (submitted: November 22, 2016)	1
Original Estimated Enrollment; (submitted: March 4, 2008)	1000
Estimated Study Completion Date	June 16, 2021
Estimated Primary Completion Date	June 16, 2021 (Final data collection date for primary outcome measure)
Eligibility Criteria	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.
Sex/Gender	Sexes Eligible for Study: All
Ages	18 Years and older (Adult, Senior)
Accepts Healthy Volunteers	No
Contacts	Contact: Pfizer CT.gov Call Center 1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com
Listed Location Countries	Argentina, Belgium, Brazil, Bulgaria, Canada, Cyprus, Denmark, France, Germany, Israel, Italy, Japan, Korea, Republic of, Mexico, Netherlands, Portugal, Spain, Sweden, Taiwan, Turkey, United States
Removed Location Countries	Australia, Austria, Greece, Switzerland
Administrative Information
NCT Number	NCT00628745
Other Study ID Numbers	B3461001; FX-R-001 ( Other Identifier: Alias Study Number ); THAOS ( Other Identifier: Alias Study Number )
Has Data Monitoring Committee	No
U.S. FDA-regulated Product	Studies a U.S. FDA-regulated Drug Product: Yes Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement	Not Provided
Responsible Party	Pfizer
Study Sponsor	Pfizer
Collaborators	Not Provided
Investigators	Study Director: Pfizer CT.gov Call Center Pfizer
PRS Account	Pfizer
Verification Date	January 2018