Transthyretin-Associated Amyloidosis Outcome Survey (THAOS)

NCT00628745

Last updated date
Study Location
VA Greater Los Angeles Healthcare System
Los Angeles, California, 90073, United States
Contact
1-800-718-1021

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1-800-718-1021

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Eligibility Criteria
condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Transthyretin Mutations, Transthyretin Amyloidosis
Sex
Females and Males
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
18 + years
Inclusion Criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

Patients must meet all of the following inclusion criteria to be eligible for enrollment 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 18 years of age.

3. 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):

1. 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

2. 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

3. 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
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details


Participant presenting with any of the following will not be included in THAOS:


1. Participant has primary or secondary amyloidosis.

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Transthyretin Mutations, Transthyretin AmyloidosisTransthyretin-Associated Amyloidosis Outcome Survey (THAOS)
NCT00628745
  1. Los Angeles, California
  2. Toronto, Ontario
  3. Toronto, Ontario
  4. Birmingham, Alabama
  5. Aurora, Colorado
  6. New Haven, Connecticut
  7. Deerfield Beach, Florida
  8. Miami, Florida
  9. Plantation, Florida
  10. Chicago, Illinois
  11. Chicago, Illinois
  12. Chicago, Illinois
  13. Chicago, Illinois
  14. Oak Lawn, Illinois
  15. New Orleans, Louisiana
  16. Baltimore, Maryland
  17. Baltimore, Maryland
  18. Baltimore, Maryland
  19. Ann Arbor, Michigan
  20. Rochester, Minnesota
  21. Saint Louis, Missouri
  22. Bronx, New York
  23. Bronx, New York
  24. Bronx, New York
  25. Bronx, New York
  26. New York, New York
  27. Columbus, Ohio
  28. Columbus, Ohio
  29. McMurray, Pennsylvania
  30. Philadelphia, Pennsylvania
  31. Philadelphia, Pennsylvania
  32. Pittsburgh, Pennsylvania
  33. Pittsburgh, Pennsylvania
  34. Wexford, Pennsylvania
  35. Nashville, Tennessee
  36. Nashville, Tennessee
  37. Salt Lake City, Utah
  38. Milwaukee, Wisconsin
  39. Buenos Aires,
  40. Ciudad Autonoma de Buenos aires,
  41. Ciudad Autonoma De Buenos Aires,
  42. Leuven,
  43. Rio de Janeiro,
  44. Sao Paulo,
  45. Sofia,
  46. Calgary, Alberta
  47. Aarhus,
  48. Cedex,
  49. Colmar,
  50. Créteil,
  51. Fort De France,
  52. Lille,
  53. Lille,
  54. Toulouse cedex 09,
  55. Aachen,
  56. Berlin,
  57. Heidelberg,
  58. Mainz,
  59. Mainz,
  60. Mainz,
  61. Muenster,
  62. Ramat Gan,
  63. Bologna,
  64. Firenze,
  65. Messina,
  66. Messina,
  67. Pavia,
  68. Pavia,
  69. Pisa,
  70. Roma,
  71. Matsumoto, JP
  72. Chiba,
  73. Kumamoto,
  74. Seoul,
  75. Seoul,
  76. Kuala Lumpur, Wilayah Persekutuan
  77. Distrito Federal,
  78. Groningen,
  79. Guimaraes,
  80. Lisboa,
  81. Lisboa,
  82. Porto,
  83. Bucuresti,
  84. Gipuzkoa - SanSebastian, Donostia
  85. Majadahonda, Madrid
  86. Barcelona,
  87. Barcelona,
  88. Granada,
  89. Huelva,
  90. Madrid,
  91. Palma de Mallorca,
  92. Piteå,
  93. Stockholm,
  94. Umeå,
  95. Taipei,
ALL GENDERS
18 Years+
years
MULTIPLE SITES
Advanced Information
Descriptive Information
Brief Title Transthyretin-Associated Amyloidosis Outcome Survey (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.
Brief Summary

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

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 *

*   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: January 19, 2021)
8000
Original Estimated Enrollment
 (submitted: March 4, 2008)
1000
Estimated Study Completion Date June 18, 2023
Estimated Primary Completion Date June 18, 2023   (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:

  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 18 years of age.
  3. 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):

    1. 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
    2. 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
    3. 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

Participant presenting with any of the following will not be included in THAOS:

1. Participant has primary or secondary amyloidosis.

Sex/Gender
Sexes Eligible for Study:All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Pfizer CT.gov Call Center1-800-718-1021[email protected]
Listed Location Countries Argentina,   Belgium,   Brazil,   Bulgaria,   Canada,   Cyprus,   Denmark,   France,   Germany,   Israel,   Italy,   Japan,   Korea, Republic of,   Malaysia,   Mexico,   Netherlands,   Portugal,   Romania,   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
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/d….
Responsible Party Pfizer
Study Sponsor Pfizer
Collaborators Not Provided
Investigators
Study Director:Pfizer CT.gov Call CenterPfizer
PRS Account Pfizer
Verification Date March 2021