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Trial record 19 of 359 for:    transthyretin

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

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ClinicalTrials.gov Identifier: NCT01604122
Recruitment Status : Completed
First Posted : May 23, 2012
Results First Posted : April 12, 2017
Last Update Posted : April 12, 2017
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Type Observational
Study Design Observational Model: Other;   Time Perspective: Cross-Sectional
Conditions Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP)
Transthyretin Cardiomyopathy (TTR-CM)
Familial Amyloid Cardiomyopathy
Senile Systemic Amyloidosis (SSA)
Intervention Other: No drug
Enrollment 1
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Participants Diagnosed With ATTR Caregivers
Hide Arm/Group Description Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey. Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Period Title: Overall Study
Started 60 32
Completed 60 32
Not Completed 0 0
Arm/Group Title Participants Diagnosed With ATTR Caregivers Total
Hide Arm/Group Description Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey. Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not. Total of all reporting groups
Overall Number of Baseline Participants 60 32 92
Hide Baseline Analysis Population Description
All participants who were enrolled in the study and completed the survey.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 60 participants 32 participants 92 participants
58.4  (12.0) 55.9  (12.8) 57.5  (12.2)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 60 participants 32 participants 92 participants
Female
13
  21.7%
22
  68.8%
35
  38.0%
Male
47
  78.3%
10
  31.3%
57
  62.0%
1.Primary Outcome
Title Demographical Characteristics of Participants
Hide Description 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.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, 'n' signifies those participants who were evaluable for specific category for each arm respectively.
Arm/Group Title Participants Diagnosed With ATTR Caregivers
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Overall Number of Participants Analyzed 60 32
Measure Type: Number
Unit of Measure: participants
Employment Status: Currently Employed (n= 60, 32) 20 11
Employment Status:Not Currently Employed (n=60,32) 39 19
Employment Status: Skipped (n= 60, 32) 1 1
Employment Status: Not Asked (n= 60, 32) 0 1
Employment Type: Full-Time (n= 20, 11) 11 9
Employment Type: Part-Time (n= 20, 11) 9 2
Unemployed: Due to ATTR (n= 39, 19) 25 0
Unemployed: Not due to ATTR (n= 39, 19) 13 0
Unemployed: Skipped (n= 39, 19) 1 0
Unemployed: Not Asked (n= 39, 19) 0 19
Education Level: Lower Than High School (n=60,32) 6 3
Education Level: High School/Equivalent(n=60,32) 8 6
Education Level: Associate Degree(n=60,32) 12 3
Education Level: College Degree (n= 60, 32) 14 10
Education Level: Professional or Graduate(n=60,32) 20 7
Education Level: Skipped (n= 60, 32) 0 2
Education Level: Not Asked (n= 60, 32) 0 1
2.Primary Outcome
Title Disease Characteristics of Participants: Disease Duration
Hide Description 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.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey.
Arm/Group Title Participants Diagnosed With ATTR
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Overall Number of Participants Analyzed 60
Median (Full Range)
Unit of Measure: years
5.6
(0.0 to 16.1)
3.Primary Outcome
Title Disease Characteristics of Participants: Mutation Type
Hide Description 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.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey.
Arm/Group Title Participants Diagnosed With ATTR
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Overall Number of Participants Analyzed 60
Measure Type: Number
Unit of Measure: participants
Val30Met 18
Wild Type TTR 2
Phe64Leu 4
Ser77Tyr 3
Thr60Ala 11
Other 5
Unknown 17
4.Primary Outcome
Title Disease Characteristics of Participants: Liver Transplantation Status
Hide Description 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 they had transplantation or not). In this outcome measure, number of participants with liver transplant status were reported. This outcome was planned to be assessed for reporting arm of participants diagnosed with ATTR.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey.
Arm/Group Title Participants Diagnosed With ATTR
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Overall Number of Participants Analyzed 60
Measure Type: Number
Unit of Measure: participants
With Liver Transplantation 31
No Liver Transplantation 29
5.Primary Outcome
Title Disease Characteristics of Participants: Number of Participants With Family History of ATTR
Hide Description Family history of participants diagnosed with ATTR was assessed to determine whether family history of ATTR was a significant risk factor for ATTR or not. This outcome was planned to be assessed for reporting arm of participants diagnosed with ATTR.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey.
Arm/Group Title Participants Diagnosed With ATTR
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Overall Number of Participants Analyzed 60
Measure Type: Number
Unit of Measure: participants
51
6.Primary Outcome
Title Disease Characteristics of Participants: Mobility Status
Hide Description Mobility, i.e., ability to walk was assessed as a part of loss of functioning in the participants diagnosed with ATTR. In this outcome, number of participants with their different mobility status along with the use of mobility aids (able to walk normally, some problems with feet but able to walk without difficulty, some difficulty walking but can walk without help, confined to bed all the time, need 1 cane or crutch to walk, need 2 canes/crutches or a walker to walk) were reported.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were included in the study and completed the survey.
Arm/Group Title Participants Diagnosed With ATTR
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Overall Number of Participants Analyzed 60
Measure Type: Number
Unit of Measure: participants
Able to Walk Normally 15
Some Problems: Able to Walk Without Difficulty 16
Some Difficulty: Can Walk Without Help 14
Need 1 Cane or Crutch to Walk 6
Need 2 Canes/Crutches or a Walker to Walk 9
Confined to bed all the Time 0
7.Primary Outcome
Title 12-Item Short-Form Health Survey (SF-12) Scores
Hide Description SF-12 was a patient reported outcome survey that represented overall health status by measuring 8 health-related aspects of an individual: Body pain, general mental health, perception of general health, physical functioning, role limitations caused by mental condition, role limitations caused by a physical condition, social functioning, and vitality. The score range for each of the 8 health aspects was from 0 (poor health) to 100 (better health), higher scores indicating good health condition. Responses on the SF-12 were also used to calculate 2 summary scores: Physical component score (PCS) and mental component score (MCS). The score range for each of these 2 summary scores was from 0 (poor health) to 100 (better health), where 100 indicated good health condition.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, 'n' signifies those participants who were evaluable for specific component for each arm respectively.
Arm/Group Title Participants Diagnosed With ATTR Caregivers
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Overall Number of Participants Analyzed 60 32
Mean (Standard Deviation)
Unit of Measure: units on a scale
Physical Health Summary (n= 56, 28) 33.6  (11.5) 42.2  (13.7)
Mental Health Summary (n= 56, 28) 47.1  (10.8) 39.3  (11.1)
Physical Functioning (n= 58, 29) 32.2  (11.0) 43.1  (13.7)
Role-Physical (n= 56, 28) 33.7  (10.7) 39.1  (13.1)
Bodily Pain (n= 57, 29) 43.0  (13.3) 39.5  (15.3)
General Health (n= 59, 29) 37.2  (12.0) 39.8  (13.5)
Vitality (n= 56, 29) 41.1  (10.6) 45.7  (11.5)
Social Functioning (n= 57, 29) 41.0  (11.6) 39.5  (13.8)
Role-Emotional (n= 57, 29) 39.5  (13.9) 34.3  (13.6)
Mental Health (n= 57, 29) 48.2  (10.2) 41.8  (11.4)
8.Primary Outcome
Title Hospital Anxiety and Depression Scale (HADS): Depression and Anxiety Subscale Scores
Hide Description HADS: participant rated 14-item questionnaire with 2 subscales; HADS-anxiety scale (HADS-A) and HADS-depression scale (HADS-D). HADS-A assesses state of generalized anxiety (anxious mood, restlessness, anxious thoughts, panic attacks); HADS-D assesses state of lost interest and diminished pleasure response (lowering of hedonic tone). Each subscale comprised of 7 items and the participant responds as to how each item applies to him/her over the past week prior to baseline visit, on 4-point response scale. Separate scores were calculated for anxiety and depression with score ranges from 0 (no presence of anxiety or depression) to 3 (severe feeling of anxiety or depression). Total score range was from 0 to 21 for each subscale; higher score indicating greater severity of anxiety and depression symptoms.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, ‘N’ (number of participants analyzed) signifies those participants who were evaluable for this measure.
Arm/Group Title Participants Diagnosed With ATTR Caregivers
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Overall Number of Participants Analyzed 55 29
Mean (Standard Deviation)
Unit of Measure: units on a scale
Depression Sub-Scale 5.9  (3.4) 8.1  (5.3)
Anxiety Sub-Scale 5.9  (3.9) 10.8  (4.5)
9.Primary Outcome
Title Euro Quality of Life (EQ-5D-3L)- Health State Profile Utility Score
Hide Description EQ-5D-3L: participant rated questionnaire to assess generic health status in two parts: single utility score and visual analog scale. For utility score, participants rated their current health state on 5 dimensions: mobility, self-care, usual activities, pain and discomfort, and anxiety and depression with each dimension having three levels of function: 1 indicates no problem; 2 indicates some problem; 3 indicates extreme problem. Scoring formula developed by EuroQol Group assigns a utility value for each domain in the profile. Score was transformed and results in a total score range of 0.05 to 1.00; higher scores indicating a better health state.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, ‘N’ signifies those participants who were evaluable for this measure.
Arm/Group Title Participants Diagnosed With ATTR Caregivers
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Overall Number of Participants Analyzed 57 28
Mean (Standard Deviation)
Unit of Measure: units on a scale
0.74  (0.20) 0.65  (0.33)
10.Primary Outcome
Title Euro Quality of Life (EQ-5D-3L)- Visual Analog Scale (VAS) Score
Hide Description EQ-5D: participant rated questionnaire to assess generic health status in two parts: single utility score and visual analog scale. The VAS component rated the current health state on a scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state); higher scores indicating a better health state.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, ‘N’ signifies those participants who were evaluable for this measure.
Arm/Group Title Participants Diagnosed With ATTR Caregivers
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Overall Number of Participants Analyzed 55 28
Mean (Standard Deviation)
Unit of Measure: units on a scale
58.7  (19.8) 64.2  (28.1)
11.Primary Outcome
Title Work Productivity and Activity Impairment- Specific Health Version (WPAI-SH): Percent of Work Time Missed
Hide Description The WPAI assesses work productivity and impairment. It was a 6-item questionnaire used to assess the degree to which a specified health problem affected work productivity and regular activities over the past 7 days prior to baseline visit. The questionnaire asked about current employment status, hours worked, hours missed from work and degree to which a specified health problem (ATTR) or caregiving affected work productivity and regular activities. Percentage of work time missed of participants were recorded and reported.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, ‘N’ signifies those participants who were evaluable for this measure.
Arm/Group Title Participants Diagnosed With ATTR Caregivers
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Overall Number of Participants Analyzed 18 9
Median (Full Range)
Unit of Measure: percentage of work time missed
2
(0 to 79)
0
(0 to 57)
12.Primary Outcome
Title Work Productivity and Activity Impairment- Specific Health Version: Percent Impairment While Working
Hide Description The WPAI assesses work productivity and impairment. It was a 6-item questionnaire used to assess the degree to which a specified health problem affected work productivity and regular activities over the past 7 days prior to baseline visit. The questionnaire asks about current employment status, hours worked, hours missed from work and degree to which a specified health problem (ATTR) or caregiving affected work productivity and regular activities. Component scores included percent work time missed due to the health problem; percent impairment while working due to problem; percent overall work impairment due to problem; and percent activity impairment due to problem. The computed percentage range for each sub-scale was from 0-100, where higher numbers indicating greater impairment and less productivity.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, ‘N’ signifies those participants who were evaluable for this measure.
Arm/Group Title Participants Diagnosed With ATTR Caregivers
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Overall Number of Participants Analyzed 18 8
Mean (Standard Deviation)
Unit of Measure: units on a scale
32.8  (32.7) 36.3  (31.6)
13.Primary Outcome
Title Work Productivity and Activity Impairment- Specific Health Version: Percent Overall Work Impairment
Hide Description The WPAI assesses work productivity and impairment. It was a 6-item questionnaire used to assess the degree to which a specified health problem affected work productivity and regular activities over the past 7 days prior to baseline visit. The questionnaire asked about current employment status, hours worked, hours missed from work and degree to which a specified health problem (ATTR) or caregiving affected work productivity and regular activities. Component scores included percent work time missed due to the health problem; percent impairment while working due to problem; percent overall work impairment due to problem; and percent activity impairment due to problem. The computed percentage range for each sub-scale was from 0-100, where higher numbers indicating greater impairment and less productivity.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, ‘N’ signifies those participants who were evaluable for this measure.
Arm/Group Title Participants Diagnosed With ATTR Caregivers
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Overall Number of Participants Analyzed 18 8
Mean (Standard Deviation)
Unit of Measure: units on a scale
37.3  (36.1) 40.4  (35.6)
14.Primary Outcome
Title Work Productivity and Activity Impairment- Specific Health Version: Percent Activity Impairment
Hide Description The WPAI assesses work productivity and impairment. It was a 6-item questionnaire used to assess the degree to which a specified health problem affected work productivity and regular activities over the past 7 days prior to baseline visit. The questionnaire asks about current employment status, hours worked, hours missed from work and degree to which a specified health problem (ATTR) or caregiving affected work productivity and regular activities. Component scores included percent work time missed due to the health problem; percent impairment while working due to problem; percent overall work impairment due to problem; and percent activity impairment due to problem. The computed percentage range for each sub-scale was from 0-100, where higher numbers indicating greater impairment and less productivity.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, ‘N’ signifies those participants who were evaluable for this measure.
Arm/Group Title Participants Diagnosed With ATTR Caregivers
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Overall Number of Participants Analyzed 55 17
Mean (Standard Deviation)
Unit of Measure: units on a scale
49.8  (30.5) 41.2  (30.4)
15.Primary Outcome
Title Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers
Hide Description Healthcare resources use survey of participants diagnosed with ATTR and caregivers was assessed by questions concerning a variety of different types of treatment and resources including outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs (for example, costs of travel to receive care).
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, 'n' signifies those participants who were evaluable for specific category for each arm respectively.
Arm/Group Title Participants Diagnosed With ATTR Caregivers
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Overall Number of Participants Analyzed 60 32
Mean (Standard Deviation)
Unit of Measure: outpatient visits
Primary Care Provider/Family Doctor (n=27, 12) 2.0  (1.5) 1.8  (1.5)
Neurologist (n=12, 6) 1.4  (0.8) 1.2  (0.4)
Cardiologist (n=17, 9) 2.2  (2.7) 1.1  (0.3)
Gastroenterologist (n=9, 3) 1.3  (0.5) 1.3  (0.6)
Hematologist (n=5, 4) 2.8  (0.8) 1.8  (1.5)
Liver Transplant Surgeon (n=6, 5) 1.2  (0.4) 1.0  (0.0)
Nurse/Nurse Practitioner (n=9, 5) 4.4  (5.6) 2.6  (2.2)
Urologist (n=9, 1) 1.9  (1.3) 2.0 [1]   (NA)
Ophthalmology (n=5, 1) 1.0  (0.0) 2.0 [1]   (NA)
Other (n=18, 6) 2.2  (2.8) 1.0  (0.0)
[1]
Data for standard deviation was not estimable as only 1 participant was analyzed for this measure.
16.Primary Outcome
Title Healthcare Resource Use Survey: Number of Hospitalizations
Hide Description Healthcare resources use survey of participants diagnosed with ATTR and caregivers was assessed by questions concerning a variety of different types of treatment and resources including outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs (for example, costs of travel to receive care).
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, ‘N’ signifies those participants who were evaluable for this measure.
Arm/Group Title Participants Diagnosed With ATTR Caregivers
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Overall Number of Participants Analyzed 9 6
Mean (Standard Deviation)
Unit of Measure: hospitalization visits
2.1  (2.0) 3.5  (1.9)
17.Primary Outcome
Title Healthcare Resource Use Survey: Number of Emergency Care Visits
Hide Description Healthcare resources use survey of participants diagnosed with ATTR and caregivers was assessed by questions concerning a variety of different types of treatment and resources including outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs (for example, costs of travel to receive care).
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, ‘N’ signifies those participants who were evaluable for this measure.
Arm/Group Title Participants Diagnosed With ATTR Caregivers
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Overall Number of Participants Analyzed 13 6
Mean (Standard Deviation)
Unit of Measure: emergency care visits
1.3  (0.9) 2.8  (1.3)
18.Primary Outcome
Title Healthcare and Resource Use Survey: Symptomatic Treatment of Participants
Hide Description Healthcare resources use survey of participants diagnosed with ATTR was assessed by questions concerning a variety of treatments and resources included outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs. Number of participants (diagnosed with ATTR) who visited non-medical practitioners (nutrition consultant/dietician, chiropractor, acupuncturist, massage therapist, occupational therapist or other than these) for symptomatic treatments were reported.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey.
Arm/Group Title Participants Diagnosed With ATTR
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Overall Number of Participants Analyzed 60
Measure Type: Number
Unit of Measure: participants
12
19.Primary Outcome
Title Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits
Hide Description Healthcare resources use survey of participants diagnosed with ATTR was assessed by questions concerning a variety of treatments and resources included outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs. Number of visits of participants (diagnosed with ATTR) who visited non-medical practitioners (nutrition consultant/dietician, chiropractor, acupuncturist, massage therapist, occupational therapist or other than these) for symptomatic treatments were reported.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, ‘N’ signifies those participants who were evaluable for this measure and 'n' signifies those participants who were evaluable for specific category.
Arm/Group Title Participants Diagnosed With ATTR
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Overall Number of Participants Analyzed 12
Mean (Standard Deviation)
Unit of Measure: symptomatic treatment visits
Nutrition Consultant/Dietitian (n=2) 4.0  (2.8)
Chiropractor (n=1) 1.0 [1]   (NA)
Acupuncturist (n=3) 3.7  (2.5)
Massage Therapist (n=5) 3.0  (1.9)
Occupational Therapist (n=2) 3.5  (3.5)
Other (n=3) 2.0  (1.0)
[1]
Data for standard deviation was not estimable as only 1 participant was analyzed for this measure.
20.Primary Outcome
Title Healthcare Resource Use Survey: Out-of-Pocket Costs
Hide Description Healthcare resources use survey of participants diagnosed with ATTR was assessed by questions concerning a variety of treatments and resources included outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs (expenditure on nutritional supplements, non-prescription medications and travel to receive medical care).
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, 'n' signifies those participants who were evaluable for specific category.
Arm/Group Title Participants Diagnosed With ATTR
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Overall Number of Participants Analyzed 60
Median (Full Range)
Unit of Measure: dollars
Nutritional Supplements (n=13)
150
(30 to 450)
Non-Prescription Medications (n=46)
28
(0 to 300)
Travel to Receive Medical Care (n=45)
30
(0 to 4000)
21.Primary Outcome
Title Participants Pain Score
Hide Description Participants diagnosed with ATTR rated their pain due to the health condition based on 3 items: pain right now, average pain in the past week, and worst pain in the past week prior to baseline visit. All 3 items were rated on an 11-point numeric rating scale ranging from 0=none to 10=severe pain, where higher scores indicated severe pain.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, ‘N’ signifies those participants who were evaluable for this measure.
Arm/Group Title Participants Diagnosed With ATTR
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Overall Number of Participants Analyzed 39
Mean (Standard Deviation)
Unit of Measure: units on a scale
Pain Right now 3.1  (3.1)
Average Pain Past Week 3.4  (3.0)
Worst Pain Past Week 4.3  (3.4)
22.Primary Outcome
Title Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) Total Quality of Life (TQOL): Total Scores
Hide Description Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of neuropathy on the quality of life of participants diagnosed with ATTR. Scoring was based on 35 questions that yield a TQOL as well as 5 subscale scores: activities of daily living, large fiber neuropathy/physical functioning, small fiber neuropathy, autonomic neuropathy, and symptoms. TQOL= sum of all the items, total possible score range= -2 to 138, where higher score=worse quality of life. This outcome measure was planned to be analyzed only for the reporting arm of participants diagnosed with ATTR.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, ‘N’ signifies those participants who were evaluable for this measure.
Arm/Group Title Participants Diagnosed With ATTR
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Overall Number of Participants Analyzed 47
Mean (Standard Deviation)
Unit of Measure: units on a scale
54.6  (32.8)
23.Primary Outcome
Title Norfolk Quality of Life-Diabetic Neuropathy Total Quality of Life: Subscale Scores
Hide Description Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of neuropathy on the quality of life of participants diagnosed with ATTR. It was summarized in 5 domains: (1) Activities of daily living (score ranges from 0 to 20, where higher score=worse quality of life); (2) Large fiber neuropathy/physical functioning (score ranges from -2 to 58, where higher score=worse condition); (3) Small fiber neuropathy (score ranges from 0 to 16, where higher score=worse condition); (4) Autonomic neuropathy (score ranges from 0 to 12, where higher score=worse condition) and (5) Symptoms (score ranges from 0 to 32, where higher score=less symptoms of disease). Total possible score range= -2 to 138, where higher score=worse quality of life. This outcome measure was analyzed only for the participants diagnosed with ATTR.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, ‘N’ signifies those participants who were evaluable for this measure and 'n' signifies those participants who were evaluable for specific category.
Arm/Group Title Participants Diagnosed With ATTR
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Overall Number of Participants Analyzed 54
Mean (Standard Deviation)
Unit of Measure: units on a scale
Symptoms (n= 50) 11.5  (7.4)
Activities of Daily Living (n= 50) 5.7  (6.1)
Small Fiber Neuropathy (n= 49) 6.0  (4.2)
Large Fiber Neuropathy (n= 47) 26.5  (16.8)
Autonomic Neuropathy (n= 50) 4.2  (3.1)
24.Primary Outcome
Title Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores
Hide Description KCCQ was a 23-item participant-completed questionnaire that assessed health status and health-related quality of life (HRQoL) in participants with heart failure. It was quantified in to following 10 summary scores: physical limitation, symptom frequency, symptom severity, and symptom stability, total symptoms, quality of life, social interference, self-efficacy, overall summary and clinical summary. Each summary score was scaled to range from 0 (minimum) to 100 (maximum), with higher scores representing greater disability. Total score ranged from 0 to 100, where higher scores indicated better functioning, fewer symptoms, and better disease specific quality of life.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, ‘N’ signifies those participants who were evaluable for this measure.
Arm/Group Title Participants Diagnosed With ATTR
Hide Arm/Group Description:
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
Overall Number of Participants Analyzed 17
Mean (Standard Deviation)
Unit of Measure: units on a scale
Physical Limitation Score 41.7  (29.2)
Symptom Stability Score 48.5  (10.7)
Symptom Frequency Score 47.7  (25.1)
Symptom Burden Score 51.0  (24.5)
Total Symptom Score 49.3  (23.8)
Self-Efficacy Score 78.7  (21.1)
Quality of Life Score 50.0  (18.6)
Social Limitation Score 31.8  (25.0)
Overall Summary Score 43.2  (20.0)
Clinical Summary Score 45.5  (25.2)
25.Primary Outcome
Title Zarit Burden Interview (ZBI): Total Scores
Hide Description ZBI was a 22-item questionnaire designed to evaluate five broad aspects of caregiver burden in terms of personal and role strain associated with caregiving. Five broad aspects were: burden in the relationship, emotional well-being, social and family life, finances, loss of control over one's life. Each item rated on a 5 point scale anchored at 0 for "never" and 4 for "nearly always." Total score ranges from 0-88 with higher scores indicating increased burden of care.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were included in the study and completed the survey.
Arm/Group Title Caregivers
Hide Arm/Group Description:
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Overall Number of Participants Analyzed 32
Mean (Standard Deviation)
Unit of Measure: units on a scale
28.8  (17.5)
26.Primary Outcome
Title Zarit Burden Interview: Subscale Scores
Hide Description A questionnaire designed to evaluate aspects of caregiver burden in terms of personal and role strain associated with caregiving. Total score of ZBI scale ranges from 0-88 with higher scores indicating increased burden of care. Five subscale scores were also calculated: (1) Burden in the relationship (consist of 6-items, ranging from 0 to 24 where higher scores indicating increased burden in relationship); (2) Emotional well-being (consisting of 7-items, ranging from 0 to 28 where higher scores indicating worse condition; (3) Social and family life (consisting of 4-items, ranging from 0 to 16 where higher scores indicating worse life condition); (4) Finances (consisting of a single item, scored from 0 to 4 where higher scores indicating worse financial condition); and (5) Loss of control over one's life (consisting of 4-items, ranging from 0 to 16 where higher scores indicating worse control over life).
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were included in the study and completed the survey. Here, 'n' signifies those participants who were evaluable for specific category.
Arm/Group Title Caregivers
Hide Arm/Group Description:
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Overall Number of Participants Analyzed 32
Mean (Standard Deviation)
Unit of Measure: units on a scale
Burden in Relationship (n=28) 7.6  (5.0)
Emotional Well-Being (n=29) 8.3  (5.9)
Social and Family Life (n=28) 5.1  (3.4)
Finances (n=29) 1.9  (1.4)
Loss of Control Over Life (n=29) 6.5  (3.5)
27.Primary Outcome
Title Caregiver Burden Items Assessment: Number of Hours Per Week Spent in Care of the Participants With ATTR
Hide Description Caregivers completed a series of questions related to the number of hours per week spent on providing care and support to the participants diagnosed with ATTR.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, ‘N’ signifies those participants who were evaluable for this measure.
Arm/Group Title Caregivers
Hide Arm/Group Description:
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Overall Number of Participants Analyzed 29
Median (Full Range)
Unit of Measure: hours per week
72
(2 to 168)
28.Primary Outcome
Title Caregiver Burden Items Assessment: Work Time Lost
Hide Description Caregivers completed a series of questions related to the loss in their working time while providing care and support to the participants diagnosed with ATTR.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, ‘N’ signifies those participants who were evaluable for this measure.
Arm/Group Title Caregivers
Hide Arm/Group Description:
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Overall Number of Participants Analyzed 26
Median (Full Range)
Unit of Measure: hours
22
(0 to 168)
29.Primary Outcome
Title Caregiver Burden Items Assessment: Total Cost
Hide Description Caregivers completed a series of questions related to the total cost spent on providing healthcare support to participants diagnosed with ATTR.
Time Frame Baseline (Day 1)
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who were enrolled in the study and completed the survey. Here, ‘N’ signifies those participants who were evaluable for this measure.
Arm/Group Title Caregivers
Hide Arm/Group Description:
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: dollars
9313.0  (33226.1)
Time Frame [Not Specified]
Adverse Event Reporting Description Due to the non-interventional nature of the study, adverse events were not collected during the study.
 
Arm/Group Title Participants Diagnosed With ATTR Caregivers
Hide Arm/Group Description Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey. Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
All-Cause Mortality
Participants Diagnosed With ATTR Caregivers
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Participants Diagnosed With ATTR Caregivers
Affected / at Risk (%) Affected / at Risk (%)
Total   0/0   0/0 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Participants Diagnosed With ATTR Caregivers
Affected / at Risk (%) Affected / at Risk (%)
Total   0/0   0/0 
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
Results Point of Contact
Name/Title: Pfizer ClinicalTrials.gov Call Center
Organization: Pfizer, Inc.
Phone: 1-800-718-1021
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT01604122     History of Changes
Other Study ID Numbers: B3461036
First Submitted: May 21, 2012
First Posted: May 23, 2012
Results First Submitted: July 14, 2016
Results First Posted: April 12, 2017
Last Update Posted: April 12, 2017