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Burden of Disease Study In Patients With Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) orTransthyretin Cardiomyopathy (TTR-CM) And Caregivers

This study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT01604122
First received: May 21, 2012
Last updated: July 21, 2014
Last verified: July 2014
  Purpose

This study is an online (web-based) or paper-based survey for patients with transthyretin familial amyloidosis polyneuropathy (TTR-FAP) and caregivers. The results will be used to describe the emotional, physical, and financial impact of having TTR-FAP or caring for someone who has the disease.


Condition Intervention
Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP)
Transthyretin Cardiomyopathy (TTR-CM)
Familial Amyloid Cardiomyopathy
Senile Systemic Amyloidosis (SSA)
Other: No drug

Study Type: Observational
Study Design: Time Perspective: Cross-Sectional
Official Title: Cross-Sectional, Non-Interventional Burden of Disease (BOD) Study in Patients With Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) or Transthyretin Cardiomyopathy (TTR-CM) and Caregivers

Resource links provided by NLM:


Further study details as provided by Pfizer:

Primary Outcome Measures:
  • SF-12 physical and mental health summary scores and subscale scores (Physical functioning, Role-physical, Bodily pain, General health, Vitality, Social functioning, Role-emotional, and Mental health) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • Hospital Anxiety and Depression Scale (HADS) depression and anxiety subscale scores [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • EuroQoL-5 Dimensions (EQ-5D-3L) utility (index) score and visual analog scale (EQ-VAS) score [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • Work Productivity and Activities Impairment: Specific Health Version (WPAI-SH) - Four component score for absenteeism, presenteeism (reduced productivity while at work), overall work impairment and impairment in activities performed outside of work [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • Caregiver burden items as assessed by number of hours spent caring for TTR-FAP patient, work time lost, impact on work productivity, and costs. [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) total score (TQOL) and subscale scores (Symptoms, ADLs, Small fiber, Large fiber, Autonomic) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • Pain items (3 items: right now, average in past week, and worst in past week) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • Zarit Burden Interview (ZBI) total score and subscale scores (Burden in the relationship, Emotional Well-being, Social and family life, Finances, Loss of control over one's life) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • Healthcare resource use (HCRU): Visits to doctors, hospitals, and other treatments. Healthcare resource use for caregivers will be assessed using an abbreviated version. [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • Kansas City Cardiomyopathy Questionnaire (KCCQ) subscale scores [ Time Frame: Day 1 ] [ Designated as safety issue: No ]

Estimated Enrollment: 500
Study Start Date: February 2013
Estimated Study Completion Date: December 2014
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Patients
Patients with TTR-FAP or TTR-CM. No drug will be administered; this is a non-interventional observational study.
Other: No drug
No drug.
Caregivers
Caregivers who are taking care of patients with TTR-FAP or TTR-CM. No drug will be administered; this is a non-interventional observational study.
Other: No drug
No drug.

Detailed Description:

Convenience sample of patients and caregivers recruited through patient advocacy groups. Convenience sample.

  Eligibility

Ages Eligible for Study:   18 Years to 85 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Adults (ages 18-85 years inclusive) diagnosed with TTR-FAP or TTR-CM and experiencing symptoms or currently providing care for a patient with TTR-FAP or TTR-CM

Criteria

Inclusion Criteria:

  • Adults (ages 18-85 years inclusive) diagnosed with TTR-FAP or TTR-CM and experiencing symptoms or currently providing care for a patient with TTR-FAP or TTR-FAP

Exclusion Criteria:

  • Caregivers who formally care for a TTR-FAP or TTR-CM patient as part of their job description and receive payment for their services.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01604122

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

Additional Information:
No publications provided

Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT01604122     History of Changes
Other Study ID Numbers: B3461036
Study First Received: May 21, 2012
Last Updated: July 21, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Pfizer:
Survey
burden of illness
caregivers
TTR-FAP
TTR-CM
familial amyloid polyneuropathy
observational
transthyretin familial amyloidosis

Additional relevant MeSH terms:
Amyloidosis
Amyloidosis, Familial
Polyneuropathies
Amyloid Neuropathies
Cardiomyopathies
Proteostasis Deficiencies
Metabolic Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Heart Diseases
Cardiovascular Diseases

ClinicalTrials.gov processed this record on July 28, 2014