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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT01604122
First received: May 21, 2012
Last updated: March 1, 2017
Last verified: March 2017
Results First Received: July 14, 2016  
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

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Participants Diagnosed With ATTR 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 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.

Participant Flow:   Overall Study
    Participants Diagnosed With ATTR   Caregivers
STARTED   60   32 
COMPLETED   60   32 
NOT COMPLETED   0   0 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
All participants who were enrolled in the study and completed the survey.

Reporting Groups
  Description
Participants Diagnosed With ATTR 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 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 Total of all reporting groups

Baseline Measures
   Participants Diagnosed With ATTR   Caregivers   Total 
Overall Participants Analyzed 
[Units: Participants]
 60   32   92 
Age 
[Units: Years]
Mean (Standard Deviation)
 58.4  (12.0)   55.9  (12.8)   57.5  (12.2) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      13  21.7%      22  68.8%      35  38.0% 
Male      47  78.3%      10  31.3%      57  62.0% 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Demographical Characteristics of Participants   [ Time Frame: Baseline (Day 1) ]

2.  Primary:   Disease Characteristics of Participants: Disease Duration   [ Time Frame: Baseline (Day 1) ]

3.  Primary:   Disease Characteristics of Participants: Mutation Type   [ Time Frame: Baseline (Day 1) ]

4.  Primary:   Disease Characteristics of Participants: Liver Transplantation Status   [ Time Frame: Baseline (Day 1) ]

5.  Primary:   Disease Characteristics of Participants: Number of Participants With Family History of ATTR   [ Time Frame: Baseline (Day 1) ]

6.  Primary:   Disease Characteristics of Participants: Mobility Status   [ Time Frame: Baseline (Day 1) ]

7.  Primary:   12-Item Short-Form Health Survey (SF-12) Scores   [ Time Frame: Baseline (Day 1) ]

8.  Primary:   Hospital Anxiety and Depression Scale (HADS): Depression and Anxiety Subscale Scores   [ Time Frame: Baseline (Day 1) ]

9.  Primary:   Euro Quality of Life (EQ-5D-3L)- Health State Profile Utility Score   [ Time Frame: Baseline (Day 1) ]

10.  Primary:   Euro Quality of Life (EQ-5D-3L)- Visual Analog Scale (VAS) Score   [ Time Frame: Baseline (Day 1) ]

11.  Primary:   Work Productivity and Activity Impairment- Specific Health Version (WPAI-SH): Percent of Work Time Missed   [ Time Frame: Baseline (Day 1) ]

12.  Primary:   Work Productivity and Activity Impairment- Specific Health Version: Percent Impairment While Working   [ Time Frame: Baseline (Day 1) ]

13.  Primary:   Work Productivity and Activity Impairment- Specific Health Version: Percent Overall Work Impairment   [ Time Frame: Baseline (Day 1) ]

14.  Primary:   Work Productivity and Activity Impairment- Specific Health Version: Percent Activity Impairment   [ Time Frame: Baseline (Day 1) ]

15.  Primary:   Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers   [ Time Frame: Baseline (Day 1) ]

16.  Primary:   Healthcare Resource Use Survey: Number of Hospitalizations   [ Time Frame: Baseline (Day 1) ]

17.  Primary:   Healthcare Resource Use Survey: Number of Emergency Care Visits   [ Time Frame: Baseline (Day 1) ]

18.  Primary:   Healthcare and Resource Use Survey: Symptomatic Treatment of Participants   [ Time Frame: Baseline (Day 1) ]

19.  Primary:   Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits   [ Time Frame: Baseline (Day 1) ]

20.  Primary:   Healthcare Resource Use Survey: Out-of-Pocket Costs   [ Time Frame: Baseline (Day 1) ]

21.  Primary:   Participants Pain Score   [ Time Frame: Baseline (Day 1) ]

22.  Primary:   Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) Total Quality of Life (TQOL): Total Scores   [ Time Frame: Baseline (Day 1) ]

23.  Primary:   Norfolk Quality of Life-Diabetic Neuropathy Total Quality of Life: Subscale Scores   [ Time Frame: Baseline (Day 1) ]

24.  Primary:   Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores   [ Time Frame: Baseline (Day 1) ]

25.  Primary:   Zarit Burden Interview (ZBI): Total Scores   [ Time Frame: Baseline (Day 1) ]

26.  Primary:   Zarit Burden Interview: Subscale Scores   [ Time Frame: Baseline (Day 1) ]

27.  Primary:   Caregiver Burden Items Assessment: Number of Hours Per Week Spent in Care of the Participants With ATTR   [ Time Frame: Baseline (Day 1) ]

28.  Primary:   Caregiver Burden Items Assessment: Work Time Lost   [ Time Frame: Baseline (Day 1) ]

29.  Primary:   Caregiver Burden Items Assessment: Total Cost   [ Time Frame: Baseline (Day 1) ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Pfizer ClinicalTrials.gov Call Center
Organization: Pfizer, Inc.
phone: 1-800-718-1021
e-mail: ClinicalTrials.gov_Inquiries@pfizer.com



Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT01604122     History of Changes
Other Study ID Numbers: B3461036
Study First Received: May 21, 2012
Results First Received: July 14, 2016
Last Updated: March 1, 2017