Clinical trial NCT01604122: Burden of Disease Study In Patients With Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) orTransthyretin Cardiomyopathy (TTR-CM) And Caregivers
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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP)
Transthyretin Cardiomyopathy (TTR-CM)
Familial Amyloid Cardiomyopathy
Senile Systemic Amyloidosis (SSA)
Keywords: Survey
burden of illness
caregivers
TTR-FAP
TTR-CM
familial amyloid polyneuropathy
observational
transthyretin familial amyloidosis
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2012-05-23
Enrollment: 1
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: CROSS_SECTIONAL
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

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

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.

Primary outcomes:
measure: Demographical Characteristics of Participants
timeFrame: Baseline (Day 1)
measure: Disease Characteristics of Participants: Disease Duration
timeFrame: Baseline (Day 1)
measure: Disease Characteristics of Participants: Mutation Type
timeFrame: Baseline (Day 1)
measure: Disease Characteristics of Participants: Liver Transplantation Status
timeFrame: Baseline (Day 1)
measure: Disease Characteristics of Participants: Number of Participants With Family History of ATTR
timeFrame: Baseline (Day 1)
measure: Disease Characteristics of Participants: Mobility Status
timeFrame: Baseline (Day 1)
measure: 12-Item Short-Form Health Survey (SF-12) Scores
timeFrame: Baseline (Day 1)
measure: Hospital Anxiety and Depression Scale (HADS): Depression and Anxiety Subscale Scores
timeFrame: Baseline (Day 1)
measure: Euro Quality of Life (EQ-5D-3L)- Health State Profile Utility Score
timeFrame: Baseline (Day 1)
measure: Euro Quality of Life (EQ-5D-3L)- Visual Analog Scale (VAS) Score
timeFrame: Baseline (Day 1)
measure: Work Productivity and Activity Impairment- Specific Health Version (WPAI-SH): Percent of Work Time Missed
timeFrame: Baseline (Day 1)
measure: Work Productivity and Activity Impairment- Specific Health Version: Percent Impairment While Working
timeFrame: Baseline (Day 1)
measure: Work Productivity and Activity Impairment- Specific Health Version: Percent Overall Work Impairment
timeFrame: Baseline (Day 1)
measure: Work Productivity and Activity Impairment- Specific Health Version: Percent Activity Impairment
timeFrame: Baseline (Day 1)
measure: Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers
timeFrame: Baseline (Day 1)
measure: Healthcare Resource Use Survey: Number of Hospitalizations
timeFrame: Baseline (Day 1)
measure: Healthcare Resource Use Survey: Number of Emergency Care Visits
timeFrame: Baseline (Day 1)
measure: Healthcare and Resource Use Survey: Symptomatic Treatment of Participants
timeFrame: Baseline (Day 1)
measure: Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits
timeFrame: Baseline (Day 1)
measure: Healthcare Resource Use Survey: Out-of-Pocket Costs
timeFrame: Baseline (Day 1)
measure: Participants Pain Score
timeFrame: Baseline (Day 1)
measure: Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) Total Quality of Life (TQOL): Total Scores
timeFrame: Baseline (Day 1)
measure: Norfolk Quality of Life-Diabetic Neuropathy Total Quality of Life: Subscale Scores
timeFrame: Baseline (Day 1)
measure: Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores
timeFrame: Baseline (Day 1)
measure: Zarit Burden Interview (ZBI): Total Scores
timeFrame: Baseline (Day 1)
measure: Zarit Burden Interview: Subscale Scores
timeFrame: Baseline (Day 1)
measure: Caregiver Burden Items Assessment: Number of Hours Per Week Spent in Care of the Participants With ATTR
timeFrame: Baseline (Day 1)
measure: Caregiver Burden Items Assessment: Work Time Lost
timeFrame: Baseline (Day 1)
measure: Caregiver Burden Items Assessment: Total Cost
timeFrame: Baseline (Day 1)
Secondary outcomes:
Other outcomes:
Locations:
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: Stewart M, Shaffer S, Murphy B, Loftus J, Alvir J, Cicchetti M, Lenderking WR. Characterizing the High Disease Burden of Transthyretin Amyloidosis for Patients and Caregivers. Neurol Ther. 2018 Dec;7(2):349-364. doi: 10.1007/s40120-018-0106-z. Epub 2018 Aug 2.