N/A
N=92
Burden of Disease Study In Patients With Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) orTransthyretin Cardiomyopathy (TTR-CM) And Caregivers
Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) · Transthyretin Cardiomyopathy (TTR-CM) · Familial Amyloid Cardiomyopathy · Senile Systemic Amyloidosis (SSA)
Bottom Line
View on ClinicalTrials.gov: NCT01604122 ↗Enrolled (actual)
92
Serious AEs
—
Results posted
Apr 2017
Primary outcome: Primary: Demographical Characteristics of Participants — 20; 11; 39; 19 participants
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- No drug (Other)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Pfizer
- Primary completion
- Jul 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Demographical Characteristics of Participants |
20; 11; 39; 19; 1; 1 | — |
| PRIMARY Disease Characteristics of Participants: Disease Duration |
5.6 | — |
| PRIMARY Disease Characteristics of Participants: Mutation Type |
18; 2; 4; 3; 11; 5 | — |
| PRIMARY Disease Characteristics of Participants: Liver Transplantation Status |
31; 29 | — |
| PRIMARY Disease Characteristics of Participants: Number of Participants With Family History of ATTR |
51 | — |
| PRIMARY Disease Characteristics of Participants: Mobility Status |
15; 16; 14; 6; 9; 0 | — |
| PRIMARY 12-Item Short-Form Health Survey (SF-12) Scores |
33.6; 42.2; 47.1; 39.3; 32.2; 43.1 | — |
| PRIMARY Hospital Anxiety and Depression Scale (HADS): Depression and Anxiety Subscale Scores |
5.9; 8.1; 5.9; 10.8 | — |
| PRIMARY Euro Quality of Life (EQ-5D-3L)- Health State Profile Utility Score |
0.74; 0.65 | — |
| PRIMARY Euro Quality of Life (EQ-5D-3L)- Visual Analog Scale (VAS) Score |
58.7; 64.2 | — |
| PRIMARY Work Productivity and Activity Impairment- Specific Health Version (WPAI-SH): Percent of Work Time Missed |
2; 0 | — |
| PRIMARY Work Productivity and Activity Impairment- Specific Health Version: Percent Impairment While Working |
32.8; 36.3 | — |
| PRIMARY Work Productivity and Activity Impairment- Specific Health Version: Percent Overall Work Impairment |
37.3; 40.4 | — |
| PRIMARY Work Productivity and Activity Impairment- Specific Health Version: Percent Activity Impairment |
49.8; 41.2 | — |
| PRIMARY Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers |
2.0; 1.8; 1.4; 1.2; 2.2; 1.1 | — |
| PRIMARY Healthcare Resource Use Survey: Number of Hospitalizations |
2.1; 3.5 | — |
| PRIMARY Healthcare Resource Use Survey: Number of Emergency Care Visits |
1.3; 2.8 | — |
| PRIMARY Healthcare and Resource Use Survey: Symptomatic Treatment of Participants |
12 | — |
| PRIMARY Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits |
4.0; 1.0; 3.7; 3.0; 3.5; 2.0 | — |
| PRIMARY Healthcare Resource Use Survey: Out-of-Pocket Costs |
150; 28; 30 | — |
| PRIMARY Participants Pain Score |
3.1; 3.4; 4.3 | — |
| PRIMARY Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) Total Quality of Life (TQOL): Total Scores |
54.6 | — |
| PRIMARY Norfolk Quality of Life-Diabetic Neuropathy Total Quality of Life: Subscale Scores |
11.5; 5.7; 6.0; 26.5; 4.2 | — |
| PRIMARY Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores |
41.7; 48.5; 47.7; 51.0; 49.3; 78.7 | — |
| PRIMARY Zarit Burden Interview (ZBI): Total Scores |
28.8 | — |
| PRIMARY Zarit Burden Interview: Subscale Scores |
7.6; 8.3; 5.1; 1.9; 6.5 | — |
| PRIMARY Caregiver Burden Items Assessment: Number of Hours Per Week Spent in Care of the Participants With ATTR |
72 | — |
| PRIMARY Caregiver Burden Items Assessment: Work Time Lost |
22 | — |
| PRIMARY Caregiver Burden Items Assessment: Total Cost |
9313.0 | — |
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.
Eligibility 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.
Data sourced from ClinicalTrials.gov (NCT01604122). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.