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

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: Cross-sectional, Non-interventional Burden Of Disease (Bod) Study In Patients With Transthyretin Familial Amyloidosis Polyneuropathy (Ttr-fap) Or Transthyretin Cardiomyopa

Brief Summary: 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.

Detailed Summary: Convenience sample of patients and caregivers recruited through patient advocacy groups. Convenience sample.
Sponsor: Pfizer

Current Primary Outcome:

  • Demographical Characteristics of Participants [ Time Frame: Baseline (Day 1) ]
    Main characteristics included were education level and employment status which were asked from all participants and caregivers. Type of job (full-time, part-time) was asked only from those participants and caregivers who provided their employment status as employed. Those who were unemployed reported their cause of unemployment, whether it was due to ATTR or not.
  • Disease Characteristics of Participants: Disease Duration [ Time Frame: Baseline (Day 1) ]
    Duration of disease was defined as the time from diagnosis of disease until baseline visit. This outcome measure was planned to be assessed for reporting arm of participants diagnosed with ATTR.
  • Disease Characteristics of Participants: Mutation Type [ Time Frame: Baseline (Day 1) ]
    Genetic mutation leads to misfolding of protein transthyretin (TTR) which results in ATTR. In this outcome, number of participants with each type of resulted mutation type (Val30Met, wild type TTR, Phe64Leu, Ser77Tyr, Thr60Ala or other than these) were reported. This outcome was planned to be assessed for reporting arm of participants diagnosed with ATTR.
  • Disease Characteristics of Participants: Liver Transplantation Status [ Time Frame: Baseline (Day 1) ]
    TTR protein is primarily synthesized in the liver. Liver transplantation was considered as one of the measure to eliminate the main source of variant TTR. In the study, participants who were diagnosed with ATTR were asked for their liver transplantation status (whether

    Original Primary Outcome:

    • 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 ]
    • Hospital Anxiety and Depression Scale (HADS) depression and anxiety subscale scores [ Time Frame: Day 1 ]
    • EuroQoL-5 Dimensions (EQ-5D-3L) utility (index) score and visual analog scale (EQ-VAS) score [ Time Frame: Day 1 ]
    • 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 ]
    • 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 ]
    • 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 ]
    • Pain items (3 items: right now, average in past week, and worst in past week) [ Time Frame: Day 1 ]
    • 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 ]
    • Healthcare resource use (HCRU): Visits to doctors, hospitals, and other treatments. Healthcare resource use for caregivers will be a

      Current Secondary Outcome:

      Original Secondary Outcome:

      Information By: Pfizer

      Dates:
      Date Received: May 21, 2012
      Date Started: April 18, 2012
      Date Completion:
      Last Updated: March 1, 2017
      Last Verified: March 2017