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.
Age range
18 Years – 85 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Demographical Characteristics of Participants
Timeframe: Baseline (Day 1)
Disease Characteristics of Participants: Disease Duration
Timeframe: Baseline (Day 1)
Disease Characteristics of Participants: Mutation Type
Timeframe: Baseline (Day 1)
Disease Characteristics of Participants: Liver Transplantation Status
Timeframe: Baseline (Day 1)
Disease Characteristics of Participants: Number of Participants With Family History of ATTR
Timeframe: Baseline (Day 1)
Disease Characteristics of Participants: Mobility Status
Timeframe: Baseline (Day 1)
12-Item Short-Form Health Survey (SF-12) Scores
Timeframe: Baseline (Day 1)
Hospital Anxiety and Depression Scale (HADS): Depression and Anxiety Subscale Scores
Timeframe: Baseline (Day 1)
Euro Quality of Life (EQ-5D-3L)- Health State Profile Utility Score
Timeframe: Baseline (Day 1)
Euro Quality of Life (EQ-5D-3L)- Visual Analog Scale (VAS) Score
Timeframe: Baseline (Day 1)
Work Productivity and Activity Impairment- Specific Health Version (WPAI-SH): Percent of Work Time Missed
Timeframe: Baseline (Day 1)
Work Productivity and Activity Impairment- Specific Health Version: Percent Impairment While Working
Timeframe: Baseline (Day 1)
Work Productivity and Activity Impairment- Specific Health Version: Percent Overall Work Impairment
Timeframe: Baseline (Day 1)
Work Productivity and Activity Impairment- Specific Health Version: Percent Activity Impairment
Timeframe: Baseline (Day 1)
Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers
Timeframe: Baseline (Day 1)
Healthcare Resource Use Survey: Number of Hospitalizations
Timeframe: Baseline (Day 1)
Healthcare Resource Use Survey: Number of Emergency Care Visits
Timeframe: Baseline (Day 1)
Healthcare and Resource Use Survey: Symptomatic Treatment of Participants
Timeframe: Baseline (Day 1)
Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits
Timeframe: Baseline (Day 1)
Healthcare Resource Use Survey: Out-of-Pocket Costs
Timeframe: Baseline (Day 1)
Participants Pain Score
Timeframe: Baseline (Day 1)
Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) Total Quality of Life (TQOL): Total Scores
Timeframe: Baseline (Day 1)
Norfolk Quality of Life-Diabetic Neuropathy Total Quality of Life: Subscale Scores
Timeframe: Baseline (Day 1)
Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores
Timeframe: Baseline (Day 1)
Zarit Burden Interview (ZBI): Total Scores
Timeframe: Baseline (Day 1)
Zarit Burden Interview: Subscale Scores
Timeframe: Baseline (Day 1)
Caregiver Burden Items Assessment: Number of Hours Per Week Spent in Care of the Participants With ATTR
Timeframe: Baseline (Day 1)
Caregiver Burden Items Assessment: Work Time Lost
Timeframe: Baseline (Day 1)
Caregiver Burden Items Assessment: Total Cost
Timeframe: Baseline (Day 1)