Trial record 14 of 58 for:    Open Studies | "Amyloidosis"

Transthyretin-Associated Amyloidoses Outcome Survey (THAOS)

This study is currently recruiting participants.
Verified April 2014 by Pfizer
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00628745
First received: February 25, 2008
Last updated: April 11, 2014
Last verified: April 2014
  Purpose

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.


Condition Intervention
Transthyretin Mutations
Transthyretin Amyloidosis
Other: None. Observational Study.

Study Type: Observational
Study Design: Time Perspective: Prospective
Official Title: A Global, Multi-Center, Longitudinal, Observational Survey Of Patients With Documented Transthyretin (TTR) Mutations Or Wild-Type TTR Amyloidosis

Resource links provided by NLM:


Further study details as provided by Pfizer:

Biospecimen Retention:   Samples With DNA

whole blood


Estimated Enrollment: 1000
Study Start Date: December 2007
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: December 2017 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Observational Other: None. Observational Study.
None.

Detailed Description:

n/a NA

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.

Criteria

Inclusion Criteria:

  • Written informed consent.
  • Patient has confirmed: genotyped TTR mutation with or without a diagnosis of TTR-associated amyloidosis (e.g., ATTR-PN, ATTR-CM), or Wild-type TTR-associated amyloidosis with cardiomyopathy (wild-type ATTR-CM).
  • Confirmation of wild-type ATTR-CM will be determined by one of the following set of criteria (A or B):

A. Presence of amyloid in cardiac biopsy tissue confirmed as TTR amyloid by immunohistochemistry and genotyped confirmation that patient does not possess a known mutation in TTR gene (i.e., is a carrier of wild-type allele only) via genetic testing; or B. 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 immunohistochemistry, and genotyped confirmation that patient does not possess a known mutation in TTR gene (i.e., is a carrier of wild-type allele only) via genetic testing.

Exclusion Criteria:

  • Patient has primary or secondary amyloidosis.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00628745

Contacts
Contact: Pfizer CT.gov Call Center 1-800-718-1021

  Show 64 Study Locations
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
No publications provided by Pfizer

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00628745     History of Changes
Other Study ID Numbers: B3461001, FX-R-001
Study First Received: February 25, 2008
Last Updated: April 11, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Pfizer:
TRANSTHYRETIN
AMYLOIDOSIS
TRANSTHYRETIN AMYLOIDOSIS

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases

ClinicalTrials.gov processed this record on April 20, 2014