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Transthyretin-Associated Amyloidosis Outcome Survey (THAOS)

Last updated on December 10, 2019

FOR MORE INFORMATION
Study Location
Sheba Medical Center
Ramat Gan, , 52621 Israel
Contact
1-800-718-1021
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

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.

NCT00628745
Pfizer
Recruiting
Transthyretin-Associated Amyloidosis Outcome Survey (THAOS)

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Descriptive Information
Brief TitleTransthyretin-Associated Amyloidosis Outcome Survey (THAOS)
Official TitleTRANSTHYRETIN-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 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 Descriptionn/a NA
Study TypeObservational
Study DesignObservational Model: Other
Time Perspective: Prospective
Target Follow-Up DurationNot Provided
BiospecimenRetention:   Samples With DNA
Description:
whole blood
Sampling MethodNon-Probability Sample
Study PopulationParticipants with transthyretin-associated amyloidoses (ATTR), including ATTR-PN (polyneuropathy), ATTR-CM (cardiomyopathy) and wild-type ATTR-CM.
Condition
  • Transthyretin Mutations
  • Transthyretin Amyloidosis
InterventionOther: None. Observational Study.
Study Groups/CohortsObservational
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 StatusRecruiting
Estimated Enrollment
 (submitted: November 22, 2016)
1
Original Estimated Enrollment
 (submitted: March 4, 2008)
1000
Estimated Study Completion DateJune 16, 2021
Estimated Primary Completion DateJune 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:

  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
Ages18 Years and older   (Adult, Older Adult)
Accepts Healthy VolunteersNo
Contacts
Contact: Pfizer CT.gov Call Center1-800-718-1021[email protected]
Listed Location CountriesArgentina,   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 CountriesAustralia,   Austria,   Greece,   Switzerland
 
Administrative Information
NCT NumberNCT00628745
Other Study ID NumbersB3461001
FX-R-001 ( Other Identifier: Alias Study Number )
THAOS ( Other Identifier: Alias Study Number )
Has Data Monitoring CommitteeNo
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/da....
Responsible PartyPfizer
Study SponsorPfizer
CollaboratorsNot Provided
Investigators
Study Director:Pfizer CT.gov Call CenterPfizer
PRS AccountPfizer
Verification DateNovember 2019

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Contact a representative by phone, email, or visiting the study website. Please see the references below:

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Pfizer Clinical Trials Contact Center

1-800-718-1021

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[email protected]

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