Transthyretin Amyloidosis Outcome Survey (THAOS)

Transthyretin Amyloidosis Clinical Trial

— THAOS  

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00628745
• Other study ID #: B3461001
• Secondary ID: FX-R-001THAOS
• Status: Completed
• Start date: January 4, 2008
• Est. completion date: June 16, 2023

Study information

• Verified date: July 2023
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

THAOS is a global, multi-center, longitudinal observational survey open to all patients with transthyretin amyloidosis (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.

Description:

n/a NA

Recruitment information / eligibility

• Status: Completed
• Enrollment: 6752
• Est. completion date: June 16, 2023
• Est. primary completion date: June 16, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria: Patients must meet all of the following inclusion criteria to be

eligible for inclusion into THAOS:

1. 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.

2. Males and females greater than or equal to 18 years of age.

3. 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):

1. 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 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 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. Exclusion Criteria

Patients meeting any of the following will not be included in the study:

1. Patient has evidence of primary (light chain) or secondary amyloidosis.

Related Conditions & MeSH terms

• Amyloid Neuropathies, Familial
• Amyloidosis
• Transthyretin Amyloidosis

Intervention

Other:
• None. Observational Study.

Locations

Country	Name	City	State
Argentina	Hospital Italiano de Buenos Aires (HIBA)	Buenos Aires
Argentina	FLENI	Ciudad Autonoma de Buenos aires
Argentina	Instituto De Investigaciones Medicas Dr Alfredo Lanari	Ciudad Autonoma De Buenos Aires
Belgium	Afdeling Klinische Cardiologie, O&N I	Leuven
Brazil	Hospital Universitário Clementino Fraga Filho -HUCFF Universidade Federal do Rio de Janeiro	Rio de Janeiro
Brazil	Instituto Dante Pazzanese De Cardiologia	Sao Paulo
Bulgaria	Alexandrovska University Hospital Clinic of Neurology	Sofia
Canada	University of Alberta Foothills Medical Centre	Calgary	Alberta
Canada	Toronto General Hospital	Toronto	Ontario
Canada	Toronto General Hospital-University Health Network	Toronto	Ontario
Cyprus	Cyprus Institute of Neurology and Genetics	Nicosia
Denmark	Aarhus University Hospital, Skejby	Aarhus
France	CHU de Bicetre	Cedex
France	Hopital Louis Pasteur	Colmar
France	CHU Henri Mondor	Créteil
France	Chu De Fort De France	Fort De France
France	CHRU de Lille, Hopital Claude Huriez	Lille
France	Hopital Salengro - CHRU de Lille	Lille
France	CHU de Toulouse - Hopital Rangueil	Toulouse cedex 09
Germany	University Hospital of RWTH Aachen	Aachen
Germany	Charite CAmpus Rudolf-Virchow-Klinikum	Berlin
Germany	Medical University of Heidelberg	Heidelberg
Germany	Johann-Gutenberg-Universität	Mainz
Germany	Universitätsmedizin der Johannes Gutenberg Universität Mainz KöR	Mainz
Germany	University Medical Center, Johannes Gutenberg-University Mainz	Mainz
Germany	Universitatsklinikum Muenster - Transplant Hepatology	Muenster
Israel	Wolfson Medical Center	Holon
Israel	Sheba Medical Center	Ramat Gan
Italy	Comitato Etico Indipendente dell Azienda	Bologna
Italy	Azienda Ospedaliero-Universitaria di Careggi	Firenze
Italy	AOU Policlinico G. Martino - Messina - Comitato Etico Scientifico	Messina
Italy	AOU Policlinico G. Martino - Messina - Dr. Vita	Messina
Italy	Centro per lo Studio e la Cura delle Amiloidosi Sistemiche - Pavia - Prof. Merlini	Pavia
Italy	Comitato di Bioetica della Fondazione IRCCS Policlinico S. Matteo	Pavia
Italy	Fondazione Toscana Gabriele Monasterio Per La Ricerca Medica E Di Sanita Pubblica (Ftgm)	Pisa
Italy	Fondazione Policlinico Gemelli - Universita Cattolica del Sacro Cuore	Roma
Japan	Chiba University Hospital	Chiba
Japan	Kumamoto University	Kumamoto
Japan	Shinshu University School of Medicine	Matsumoto	JP
Korea, Republic of	Konkuk University Medical Center	Seoul
Korea, Republic of	Samsung Medical Center, Sungkyunkwan University School of Medicine	Seoul
Malaysia	University Malaya Medical Centre (UMMC)	Kuala Lumpur	Wilayah Persekutuan
Mexico	Instituto Nacional de Ciencia Medicas y Nutricion Salvador Zubiran	Distrito Federal
Netherlands	University Medical Center Groningen	Groningen
Portugal	Centro Hospitalar Do Alto Ave, Epe	Guimaraes
Portugal	Centro Hospitalar Lisboa Norte (CHLN) EPE - Hospital de Santa Maria	Lisboa
Portugal	Hospital Santa Maria	Lisboa
Portugal	Unidade Clinica de Paramiloidose-Centro Hospitalar Porto,EPE-Hospital Geral Santo Antonio	Porto
Romania	Institutul De Cardiologie Prof. Dr. C. C. Iliescu Spitalului Fundeni	Bucuresti
Saudi Arabia	King Faisal Specialist Hospital and Research Center	Riyadh
Spain	Hospital Universitari de Bellvitge	Barcelona
Spain	Institut Clinic de Nefrologia i Urologia - ICNU, Hospital Clinic i Provincial de Barcelona	Barcelona
Spain	Hospital Universitario Donostia	Gipuzkoa - SanSebastian	Donostia
Spain	Hospital de Rehabilitacion y Traumatologia Virgen de las Nieves	Granada
Spain	Hospital Juan Ramon Jimenez	Huelva
Spain	Hospital Clinico San Carlos	Madrid
Spain	Hospital Gregorio Marañón	Madrid
Spain	Hospital Universitario Puerta de Hierro	Majadahonda	Madrid
Spain	Hospital Son Llatzer	Palma de Mallorca
Sweden	Piteå Älvdals Hospital	Piteå
Sweden	Karolinska University Hospital, Huddinge	Stockholm
Sweden	Umeå University Hospital	Umeå
Taiwan	National Taiwan University Hospital	Taipei
Taiwan	National Taiwan University Hospital	Taipei
Turkey	Istanbul University,Istanbul Faculty of Medicine,Department of Neurology	Istanbul
United Arab Emirates	Cleveland Clinic Abu Dhabi LLC	Abu Dhabi
United States	University of Michigan	Ann Arbor	Michigan
United States	UC Denver	Aurora	Colorado
United States	Johns Hopkins Hospital	Baltimore	Maryland
United States	University of Maryland	Baltimore	Maryland
United States	University of Maryland School of Medicine	Baltimore	Maryland
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	Harvard Vanguard Medical Associates	Boston	Massachusetts
United States	Cardiology Clinic at Montefiore Hutchinson Campus	Bronx	New York
United States	Congestive Heart Failure Clinic	Bronx	New York
United States	Montefiore Medical Center-Jack D. Weiler Hospital	Bronx	New York
United States	Montefiore Moses Division	Bronx	New York
United States	Clinical Trials Unit	Chicago	Illinois
United States	Northwestern University	Chicago	Illinois
United States	University of Chicago Medical Center	Chicago	Illinois
United States	University of Chicago Medical Center IRB	Chicago	Illinois
United States	Cleveland Clinic Foundation	Cleveland	Ohio
United States	OSU Wexner Medical Center	Columbus	Ohio
United States	The Ohio University College of Medicine	Columbus	Ohio
United States	UHealth Deerfield Beach	Deerfield Beach	Florida
United States	VA Greater Los Angeles Healthcare System	Los Angeles	California
United States	Peters Township Health and Wellness Pavillion	McMurray	Pennsylvania
United States	University of Miami Hospital & Clinics	Miami	Florida
United States	Medical College of Wisconsin	Milwaukee	Wisconsin
United States	IRB	Nashville	Tennessee
United States	Vanderbilt University School of Medicine	Nashville	Tennessee
United States	Smilow Cancer Hospital at Yale-New Haven	New Haven	Connecticut
United States	John Ochsner Heart & Vascular Institute	New Orleans	Louisiana
United States	Columbia University Medical Center	New York	New York
United States	NYU Medical Center	New York	New York
United States	Advocate Christ Medical Centre	Oak Lawn	Illinois
United States	University of California, Irvine	Orange	California
United States	Penn Presbyterian Medical Center-University of Pennsylvania	Philadelphia	Pennsylvania
United States	Temple University School of Medicine	Philadelphia	Pennsylvania
United States	University of Pennsylvania - Perelman Center for Advanced Medicine	Philadelphia	Pennsylvania
United States	Mayo Clinic Arizona	Phoenix	Arizona
United States	Admin	Pittsburgh	Pennsylvania
United States	Allegheny General Hospital	Pittsburgh	Pennsylvania
United States	University of Pittsburgh Medical Center (UPMC)	Pittsburgh	Pennsylvania
United States	UHealth Plantation	Plantation	Florida
United States	Oregon Health and Science University	Portland	Oregon
United States	Mayo Clinic	Rochester	Minnesota
United States	Washington University School of Medicine	Saint Louis	Missouri
United States	The University of Utah Health Sciences Center	Salt Lake City	Utah
United States	Office of Sponsored Research	San Francisco	California
United States	University of California - San Francisco, UCSF Department of Neurology	San Francisco	California
United States	Stanford University School of Medicine	Stanford	California
United States	Wexford Health and Wellness Pavillion	Wexford	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

United States,  Argentina,  Belgium,  Brazil,  Bulgaria,  Canada,  Cyprus,  Denmark,  France,  Germany,  Israel,  Italy,  Japan,  Korea, Republic of,  Malaysia,  Mexico,  Netherlands,  Portugal,  Romania,  Saudi Arabia,  Spain,  Sweden,  Taiwan,  Turkey,  United Arab Emirates, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	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.	Dec 2007 to June 2023

External Links

•   To obtain contact information for a study center near you, click here.