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

May 21, 2012
July 21, 2014
February 2013
December 2014   (final data collection date for primary outcome measure)
  • 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 ]
Same as current
Complete list of historical versions of study NCT01604122 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
Burden of Disease Study In Patients With Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) orTransthyretin Cardiomyopathy (TTR-CM) And Caregivers
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

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.

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

Observational
Time Perspective: Cross-Sectional
Not Provided
Not Provided
Non-Probability Sample

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

  • Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP)
  • Transthyretin Cardiomyopathy (TTR-CM)
  • Familial Amyloid Cardiomyopathy
  • Senile Systemic Amyloidosis (SSA)
Other: No drug
No drug.
  • Patients
    Patients with TTR-FAP or TTR-CM. No drug will be administered; this is a non-interventional observational study.
    Intervention: Other: 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.
    Intervention: Other: No drug
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Enrolling by invitation
500
December 2014
December 2014   (final data collection date for primary outcome measure)

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.
Both
18 Years to 85 Years
No
Contact information is only displayed when the study is recruiting subjects
Not Provided
 
NCT01604122
B3461036
No
Pfizer
Pfizer
Not Provided
Study Director: Pfizer CT.gov Call Center Pfizer
Pfizer
July 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP