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Transthyretin Amyloidosis Outcome Survey (THAOS) (THAOS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00628745
Recruitment Status : Active, not recruiting
First Posted : March 5, 2008
Last Update Posted : April 27, 2023
Sponsor:
Information provided by (Responsible Party):
Pfizer

Tracking Information
First Submitted Date February 25, 2008
First Posted Date March 5, 2008
Last Update Posted Date April 27, 2023
Actual Study Start Date January 4, 2008
Estimated Primary Completion Date June 16, 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: April 19, 2021)		 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. [ Time Frame: Dec 2007 to June 2023 ]
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.
Original Primary Outcome Measures Not Provided
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Transthyretin 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 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 The study population includes patients with confirmed hereditary or wild-type ATTR amyloidosis (inclusive of ATTR-PN and ATTR-CM) and those with TTR gene mutations without a diagnosis of ATTR amyloidosis.
Condition
  • Transthyretin Gene 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 Active, not recruiting
Actual Enrollment
 (submitted: April 25, 2023)		 6224
Original Estimated Enrollment
 (submitted: March 4, 2008)		 1000
Estimated Study Completion Date June 16, 2023
Estimated Primary Completion Date June 16, 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 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.
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries 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,   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/data_requests.
Current Responsible Party Pfizer
Original Responsible Party Linda Robertson, FoldRx Pharmaceuticals, Inc.
Current Study Sponsor Pfizer
Original Study Sponsor FoldRx Pharmaceuticals
Collaborators Not Provided
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
PRS Account Pfizer
Verification Date April 2023