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Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy (ATTR-ACT)

Primary Purpose

Transthyretin (TTR) Amyloid Cardiomyopathy

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Tafamidis
Tafamidis
Placebo
Sponsored by
Pfizer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Transthyretin (TTR) Amyloid Cardiomyopathy focused on measuring amyloidosis, amyloid, transthyretin, TTR

Eligibility Criteria

18 Years - 90 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Medical history of Heart Failure (HF) with at least 1 prior hospitalization for HF or clinical evidence of HF (without hospitalization) manifested by signs or symptoms of volume overload or elevated intracardiac pressures (e.g., elevated jugular venous pressure, shortness of breath or signs of pulmonary congestion on x-ray or auscultation, peripheral edema) that required/requires treatment with a diuretic for improvement,
  • Evidence of cardiac involvement by echocardiography with an end-diastolic interventricular septal wall thickness > 12 mm,
  • Presence of amyloid deposits in biopsy tissue and presence of a variant TTR genotype and/or TTR precursor protein identification by immunohistochemistry, scintigraphy or mass spectrometry

Exclusion Criteria:

  • A New York Heart Association (NYHA) classification of IV.
  • Presence of primary (light chain) amyloidosis.
  • Prior liver or heart transplantation or implanted cardiac mechanical assist device.

Sites / Locations

  • The Kirklin Clinic
  • UAB Hospital Department of Pharmacy IDS
  • University Hospital, University of Alabama at Birmingham
  • Mayo Clinic Arizona - Mayo Clinic Speciality Building
  • Mayo Clinic Hospital
  • Mayo Clinic Arizona - Mayo Clinic Building
  • UCSD Clinical and Translational Research Institute, Investigational Drug Service
  • Altman Clinical Translational Research institute
  • Altman Clinical Translational Research Institute
  • UCSD Sulpizio Cardiovascular Center
  • Cedars-Sinai Medical Center, Investigational Drug Pharmacy Services
  • Cedars-Sinai Medical Center
  • Cedars-Sinai Medical Care Foundation
  • UCSF Cardiovascular Care and Prevention Center
  • Stanford University Hospital and Clinics
  • UM Sylvester at Deerfield Beach
  • University of Miami Hospital & Clinics
  • Northwestern Medical Group
  • Northwestern Memorial Hospital
  • Advocate Christ Medical Centre
  • Ochsner Medical Center
  • University of Maryland, School of Medicine
  • Johns Hopkins University
  • The Johns Hopkins Hospital IDS Pharmacy
  • Boston Medical Center - Cardiovascular Center, Outpatient Clinic
  • Boston Medical Center Investigational Pharmacy Services
  • Boston Medical Center
  • Boston University Medical Center General Clinical Research Unit
  • Michigan Medicine,University of Michigan
  • Mayo Clinic Pharmacy Research Supp
  • Mayo Clinic
  • Newark Beth Israel Medical Center
  • New York University Hospitals
  • Icahn School of Medicine at Mount Sinai and Mount Sinai Hospital
  • Mount Sinai Doctors Faculty Practice
  • The Mount Sinai Hospital Department of Pharmacy
  • Columbia University Medical Center Research Pharmacy
  • Columbia University Medical Center/Clinical Cardiovascular Research Laboratory for the Elderly
  • Duke University Health System
  • Cleveland Clinic
  • OHSU Center for Health and Healing
  • OHSU Research Pharmacy Services
  • Oregon Health and Science University (OHSU)
  • Penn Presbyterian Medical Center
  • Vanderbilt University Medical Center
  • Houston Methodist Hospital
  • University of Utah,Health Sciences Center
  • VCU Medical Center - Ambulatory Care Clinic
  • VCU Medical Center - Main Hospital
  • Virginia Mason Medical Center
  • Universitaire Ziekenhuizen Leuven
  • Unidade de Pesquisa Clinica / Hospital Universitario Clementino Fraga Filho / UFRJ
  • University of Calgary/Foothills Medical Centre
  • Toronto General Hospital
  • Fakultni nemocnice u sv. Anny, Nemocnicni lekarna
  • Nemocnice u sv. Anny v Brne
  • Vseobecna fakultni nemocnice v Praze
  • Vseobecna fakultni nemocnice v Praze, Nemocnicni lekarna
  • IKEM, Ustavni lekarna
  • IKEM
  • Hopital Antoine Beclere
  • CHU Henri Mondor
  • Medical University of Heidelberg
  • Universitaetsklinikum Muenster
  • U.O. Cardiologia- Prof. Rapezzi
  • Unita Operativa di Cardiologia
  • S.O.D. Cardiologia Generale 1 - D.A.I. del Cuore e dei Vasi
  • Centro per lo Studio e la Cura Delle Amiloidosi Sistemiche - Fondazione IRCCS Policlinico S. Matteo
  • Kurume University Hospital
  • Kumamoto University Hospital
  • Shinshu University Hospital
  • University Medical Center Groningen
  • Hospital Universitario Puerta De Hierro - Majadahonda
  • Hospital Clinico Universitario Virgen de la Arrixaca
  • Hospital Universitario A Coruna
  • Medicin-Geriatrik kliniken-Skelleftea lasarett
  • Sjukhusvägen, Akademiska sjukhuset
  • St Bartholomew's Hospital-Barts Health NHS Trust
  • St. George's Hospital (St George's Healthcare NHS Trust)
  • St. George's University of London

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

Tafamidis - 20 mg

Tafamidis - 80 mg

Placebo

Arm Description

Active Treatment-Low dose

Active Treatment-High Dose

Placebo control

Outcomes

Primary Outcome Measures

Hierarchical Combination of All-Cause Mortality and Frequency of Cardiovascular-Related Hospitalizations
All-cause mortality and frequency of cardiovascular hospitalization were analyzed using Finkelstein-Schoenfeld method. The method combines all-cause mortality and frequency of CV-related hospitalizations in a hierarchical fashion using all-cause mortality first. The method compares every participant with every other participant within strata, assigning a +1 to the "better" participant and a -1 to the "worse" participant and 0 if they are "tied". Participants who discontinued for transplantation (heart transplantation and combined heart and liver transplantation) or for implantation of a cardiac mechanical assist device, were handled in the same manner as death. 'Win' represents a participant doing better based on hierarchical comparison. The reported unit is the total "wins" for each treatment group from performing such a hierarchical comparison across all 4 strata in the study.

Secondary Outcome Measures

All-Cause Mortality
Number of deaths due to any cause was analyzed. Participants who discontinued for transplantation (heart transplantation and combined heart and liver transplantation) or for implantation of a cardiac mechanical assist device were handled in the same manner as death.
Frequency of Cardiovascular-Related Hospitalizations
CV related hospitalizations per year is calculated as participant's number of CV related hospitalizations upon duration on study in years.
Change From Baseline in the Total Distance Walked During 6 Minute Walk Test (6MWT) at Month 30
6MWT is the total distance that a participant could walk in 6 minutes. Participants were asked to perform the test at a pace that was comfortable to them, with as many breaks as they needed.
Change From Baseline in Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS) at Month 30
KCCQ is a 23-item participant-completed questionnaire that assesses health status and health-related quality of life in participants with heart failure. Eight domain scores were calculated for the KCCQ: Physical limitation, Social limitation, Quality of life, Self-efficacy, Symptom stability, Symptom frequency, Symptom burden, and Total symptoms (calculated as the mean of Symptom frequency and Symptom burden scores). Two summary scores were also calculated: Clinical Summary (calculated as mean of Physical limitation and Total symptom scores) and Overall Summary (calculated as mean of Physical limitation, Social limitation, Total symptoms, and Quality of life scores). Domain and summary scores were scaled to range from 0 (minimum) to 100 (maximum); higher scores represent better health status.
Number of Participants With Cardiovascular-Related Mortality
Deaths adjudicated as CV-related and indeterminate are reported. Participants who discontinued for transplantation (heart transplantation and combined heart and liver transplantation) or for implantation of a cardiac mechanical assist device, were handled in the same manner as death.
Percentage of Participants With Stabilized Transthyretin (TTR) at Month 1
TTR stabilization is a measure of the degree of stabilization afforded the TTR molecule by tafamidis.

Full Information

First Posted
November 14, 2013
Last Updated
April 15, 2019
Sponsor
Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT01994889
Brief Title
Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy
Acronym
ATTR-ACT
Official Title
A MULTICENTER, INTERNATIONAL, PHASE 3, DOUBLE-BLIND, PLACEBO-CONTROLLED, RANDOMIZED STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF DAILY ORAL DOSING OF TAFAMIDIS MEGLUMINE (PF-06291826) 20 MG OR 80 MG IN COMPARISON TO PLACEBO IN SUBJECTS DIAGNOSED WITH TRANSTHYRETIN CARDIOMYOPATHY (TTR-CM)
Study Type
Interventional

2. Study Status

Record Verification Date
April 2019
Overall Recruitment Status
Completed
Study Start Date
December 9, 2013 (Actual)
Primary Completion Date
February 7, 2018 (Actual)
Study Completion Date
February 7, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This Phase 3 study will investigate the efficacy, safety and tolerability of an oral daily dose of 20 mg or 80 mg tafamidis meglumine capsules compared to placebo in subjects with either transthyretin genetic variants or wild-type transthyretin resulting in amyloid cardiomyopathy.
Detailed Description
Phase 3, multicenter, global, three-arm, parallel design, placebo-controlled, double-blind, randomized study to determine efficacy, safety and tolerability of tafamidis on clinical outcomes (all-cause mortality and frequency of cardiovascular-related hospitalizations) in subjects with either transthyretin genetic variants or wild-type transthyretin resulting in amyloid cardiomyopathy.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Transthyretin (TTR) Amyloid Cardiomyopathy
Keywords
amyloidosis, amyloid, transthyretin, TTR

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
441 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Tafamidis - 20 mg
Arm Type
Experimental
Arm Description
Active Treatment-Low dose
Arm Title
Tafamidis - 80 mg
Arm Type
Experimental
Arm Description
Active Treatment-High Dose
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo control
Intervention Type
Drug
Intervention Name(s)
Tafamidis
Intervention Description
Tafamidis 20 mg in soft gel capsules administered once a day for 30 months
Intervention Type
Drug
Intervention Name(s)
Tafamidis
Intervention Description
Tafamidis 80 mg in soft gel capsules administered once a day for 30 months
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo in soft gel capsules administered once a day for 30 months
Primary Outcome Measure Information:
Title
Hierarchical Combination of All-Cause Mortality and Frequency of Cardiovascular-Related Hospitalizations
Description
All-cause mortality and frequency of cardiovascular hospitalization were analyzed using Finkelstein-Schoenfeld method. The method combines all-cause mortality and frequency of CV-related hospitalizations in a hierarchical fashion using all-cause mortality first. The method compares every participant with every other participant within strata, assigning a +1 to the "better" participant and a -1 to the "worse" participant and 0 if they are "tied". Participants who discontinued for transplantation (heart transplantation and combined heart and liver transplantation) or for implantation of a cardiac mechanical assist device, were handled in the same manner as death. 'Win' represents a participant doing better based on hierarchical comparison. The reported unit is the total "wins" for each treatment group from performing such a hierarchical comparison across all 4 strata in the study.
Time Frame
Baseline up to Month 30
Secondary Outcome Measure Information:
Title
All-Cause Mortality
Description
Number of deaths due to any cause was analyzed. Participants who discontinued for transplantation (heart transplantation and combined heart and liver transplantation) or for implantation of a cardiac mechanical assist device were handled in the same manner as death.
Time Frame
Baseline up to Month 30
Title
Frequency of Cardiovascular-Related Hospitalizations
Description
CV related hospitalizations per year is calculated as participant's number of CV related hospitalizations upon duration on study in years.
Time Frame
Baseline to Month 30
Title
Change From Baseline in the Total Distance Walked During 6 Minute Walk Test (6MWT) at Month 30
Description
6MWT is the total distance that a participant could walk in 6 minutes. Participants were asked to perform the test at a pace that was comfortable to them, with as many breaks as they needed.
Time Frame
Baseline, Month 30
Title
Change From Baseline in Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS) at Month 30
Description
KCCQ is a 23-item participant-completed questionnaire that assesses health status and health-related quality of life in participants with heart failure. Eight domain scores were calculated for the KCCQ: Physical limitation, Social limitation, Quality of life, Self-efficacy, Symptom stability, Symptom frequency, Symptom burden, and Total symptoms (calculated as the mean of Symptom frequency and Symptom burden scores). Two summary scores were also calculated: Clinical Summary (calculated as mean of Physical limitation and Total symptom scores) and Overall Summary (calculated as mean of Physical limitation, Social limitation, Total symptoms, and Quality of life scores). Domain and summary scores were scaled to range from 0 (minimum) to 100 (maximum); higher scores represent better health status.
Time Frame
Baseline, Month 30
Title
Number of Participants With Cardiovascular-Related Mortality
Description
Deaths adjudicated as CV-related and indeterminate are reported. Participants who discontinued for transplantation (heart transplantation and combined heart and liver transplantation) or for implantation of a cardiac mechanical assist device, were handled in the same manner as death.
Time Frame
Baseline up to Month 30
Title
Percentage of Participants With Stabilized Transthyretin (TTR) at Month 1
Description
TTR stabilization is a measure of the degree of stabilization afforded the TTR molecule by tafamidis.
Time Frame
Month 1

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
90 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Medical history of Heart Failure (HF) with at least 1 prior hospitalization for HF or clinical evidence of HF (without hospitalization) manifested by signs or symptoms of volume overload or elevated intracardiac pressures (e.g., elevated jugular venous pressure, shortness of breath or signs of pulmonary congestion on x-ray or auscultation, peripheral edema) that required/requires treatment with a diuretic for improvement, Evidence of cardiac involvement by echocardiography with an end-diastolic interventricular septal wall thickness > 12 mm, Presence of amyloid deposits in biopsy tissue and presence of a variant TTR genotype and/or TTR precursor protein identification by immunohistochemistry, scintigraphy or mass spectrometry Exclusion Criteria: A New York Heart Association (NYHA) classification of IV. Presence of primary (light chain) amyloidosis. Prior liver or heart transplantation or implanted cardiac mechanical assist device.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pfizer CT.gov Call Center
Organizational Affiliation
Pfizer
Official's Role
Study Director
Facility Information:
Facility Name
The Kirklin Clinic
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35249
Country
United States
Facility Name
UAB Hospital Department of Pharmacy IDS
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35249
Country
United States
Facility Name
University Hospital, University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35249
Country
United States
Facility Name
Mayo Clinic Arizona - Mayo Clinic Speciality Building
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85054
Country
United States
Facility Name
Mayo Clinic Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85054
Country
United States
Facility Name
Mayo Clinic Arizona - Mayo Clinic Building
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85259
Country
United States
Facility Name
UCSD Clinical and Translational Research Institute, Investigational Drug Service
City
La Jolla
State/Province
California
ZIP/Postal Code
92037-0620
Country
United States
Facility Name
Altman Clinical Translational Research institute
City
La Jolla
State/Province
California
ZIP/Postal Code
92037-0990
Country
United States
Facility Name
Altman Clinical Translational Research Institute
City
La Jolla
State/Province
California
ZIP/Postal Code
92037
Country
United States
Facility Name
UCSD Sulpizio Cardiovascular Center
City
La Jolla
State/Province
California
ZIP/Postal Code
92037
Country
United States
Facility Name
Cedars-Sinai Medical Center, Investigational Drug Pharmacy Services
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Facility Name
Cedars-Sinai Medical Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Facility Name
Cedars-Sinai Medical Care Foundation
City
Los Angeles
State/Province
California
ZIP/Postal Code
90211
Country
United States
Facility Name
UCSF Cardiovascular Care and Prevention Center
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Stanford University Hospital and Clinics
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
UM Sylvester at Deerfield Beach
City
Deerfield Beach
State/Province
Florida
ZIP/Postal Code
33442
Country
United States
Facility Name
University of Miami Hospital & Clinics
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Northwestern Medical Group
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Northwestern Memorial Hospital
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Advocate Christ Medical Centre
City
Oak Lawn
State/Province
Illinois
ZIP/Postal Code
60453
Country
United States
Facility Name
Ochsner Medical Center
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70121
Country
United States
Facility Name
University of Maryland, School of Medicine
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21201
Country
United States
Facility Name
Johns Hopkins University
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
The Johns Hopkins Hospital IDS Pharmacy
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Boston Medical Center - Cardiovascular Center, Outpatient Clinic
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States
Facility Name
Boston Medical Center Investigational Pharmacy Services
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States
Facility Name
Boston Medical Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States
Facility Name
Boston University Medical Center General Clinical Research Unit
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States
Facility Name
Michigan Medicine,University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Mayo Clinic Pharmacy Research Supp
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Newark Beth Israel Medical Center
City
Newark
State/Province
New Jersey
ZIP/Postal Code
07112
Country
United States
Facility Name
New York University Hospitals
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
Icahn School of Medicine at Mount Sinai and Mount Sinai Hospital
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Mount Sinai Doctors Faculty Practice
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
The Mount Sinai Hospital Department of Pharmacy
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Columbia University Medical Center Research Pharmacy
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Columbia University Medical Center/Clinical Cardiovascular Research Laboratory for the Elderly
City
New York
State/Province
New York
ZIP/Postal Code
10034
Country
United States
Facility Name
Duke University Health System
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Cleveland Clinic
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
OHSU Center for Health and Healing
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
OHSU Research Pharmacy Services
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Oregon Health and Science University (OHSU)
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Penn Presbyterian Medical Center
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Houston Methodist Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Utah,Health Sciences Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
VCU Medical Center - Ambulatory Care Clinic
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
Facility Name
VCU Medical Center - Main Hospital
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
Facility Name
Virginia Mason Medical Center
City
Seattle
State/Province
Washington
ZIP/Postal Code
98101
Country
United States
Facility Name
Universitaire Ziekenhuizen Leuven
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
Unidade de Pesquisa Clinica / Hospital Universitario Clementino Fraga Filho / UFRJ
City
Rio de Janeiro
State/Province
RJ
ZIP/Postal Code
21941-913
Country
Brazil
Facility Name
University of Calgary/Foothills Medical Centre
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T2N 4Z6
Country
Canada
Facility Name
Toronto General Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 2N2
Country
Canada
Facility Name
Fakultni nemocnice u sv. Anny, Nemocnicni lekarna
City
Brno
ZIP/Postal Code
656 91
Country
Czechia
Facility Name
Nemocnice u sv. Anny v Brne
City
Brno
ZIP/Postal Code
656 91
Country
Czechia
Facility Name
Vseobecna fakultni nemocnice v Praze
City
Praha 2
ZIP/Postal Code
128 08
Country
Czechia
Facility Name
Vseobecna fakultni nemocnice v Praze, Nemocnicni lekarna
City
Praha 2
ZIP/Postal Code
12808
Country
Czechia
Facility Name
IKEM, Ustavni lekarna
City
Praha 4
ZIP/Postal Code
140 21
Country
Czechia
Facility Name
IKEM
City
Praha 4
ZIP/Postal Code
140 21
Country
Czechia
Facility Name
Hopital Antoine Beclere
City
Clamart
ZIP/Postal Code
92140
Country
France
Facility Name
CHU Henri Mondor
City
Creteil
ZIP/Postal Code
94010
Country
France
Facility Name
Medical University of Heidelberg
City
Heidelberg
ZIP/Postal Code
69120
Country
Germany
Facility Name
Universitaetsklinikum Muenster
City
Muenster
ZIP/Postal Code
48149
Country
Germany
Facility Name
U.O. Cardiologia- Prof. Rapezzi
City
Bologna
ZIP/Postal Code
40138
Country
Italy
Facility Name
Unita Operativa di Cardiologia
City
Brescia
ZIP/Postal Code
25123
Country
Italy
Facility Name
S.O.D. Cardiologia Generale 1 - D.A.I. del Cuore e dei Vasi
City
Firenze
ZIP/Postal Code
50134
Country
Italy
Facility Name
Centro per lo Studio e la Cura Delle Amiloidosi Sistemiche - Fondazione IRCCS Policlinico S. Matteo
City
Pavia
ZIP/Postal Code
27100
Country
Italy
Facility Name
Kurume University Hospital
City
Kurume-shi
State/Province
Fukuoka
ZIP/Postal Code
830-0011
Country
Japan
Facility Name
Kumamoto University Hospital
City
Kumamoto-city
State/Province
Kumamoto
ZIP/Postal Code
8608556
Country
Japan
Facility Name
Shinshu University Hospital
City
Nagano
ZIP/Postal Code
390-8621
Country
Japan
Facility Name
University Medical Center Groningen
City
Groningen
ZIP/Postal Code
9713 GZ
Country
Netherlands
Facility Name
Hospital Universitario Puerta De Hierro - Majadahonda
City
Majadahonda
State/Province
Madrid
ZIP/Postal Code
28222
Country
Spain
Facility Name
Hospital Clinico Universitario Virgen de la Arrixaca
City
El Palmar
State/Province
Murcia
ZIP/Postal Code
30120
Country
Spain
Facility Name
Hospital Universitario A Coruna
City
A Coruna
ZIP/Postal Code
15006
Country
Spain
Facility Name
Medicin-Geriatrik kliniken-Skelleftea lasarett
City
Skelleftea
ZIP/Postal Code
93186
Country
Sweden
Facility Name
Sjukhusvägen, Akademiska sjukhuset
City
Uppsala
ZIP/Postal Code
751 85
Country
Sweden
Facility Name
St Bartholomew's Hospital-Barts Health NHS Trust
City
London
ZIP/Postal Code
EC1A 7BE
Country
United Kingdom
Facility Name
St. George's Hospital (St George's Healthcare NHS Trust)
City
London
ZIP/Postal Code
SW17 0QT
Country
United Kingdom
Facility Name
St. George's University of London
City
London
ZIP/Postal Code
SW17 0RE
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
IPD Sharing URL
https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Citations:
PubMed Identifier
36399070
Citation
Tess DA, Maurer TS, Li Z, Bulawa C, Fleming J, Moody AT. Relationship of binding-site occupancy, transthyretin stabilisation and disease modification in patients with tafamidis-treated transthyretin amyloid cardiomyopathy. Amyloid. 2023 Jun;30(2):208-219. doi: 10.1080/13506129.2022.2145876. Epub 2022 Nov 18.
Results Reference
derived
PubMed Identifier
35353352
Citation
Rozenbaum MH, Tran D, Bhambri R, Nativi-Nicolau J. Annual Cardiovascular-Related Hospitalization Days Avoided with Tafamidis in Patients with Transthyretin Amyloid Cardiomyopathy. Am J Cardiovasc Drugs. 2022 Jul;22(4):445-450. doi: 10.1007/s40256-022-00526-9. Epub 2022 Mar 30.
Results Reference
derived
PubMed Identifier
35297655
Citation
Nativi-Nicolau J, Judge DP, Hoffman JE, Gundapaneni B, Keohane D, Sultan MB, Grogan M. How did transthyretin amyloid cardiomyopathy progress in patients who took placebo in the study ATTR-ACT? A plain language summary. Future Cardiol. 2022 Mar 17. doi: 10.2217/fca-2021-0150. Online ahead of print.
Results Reference
derived
PubMed Identifier
34923848
Citation
Elliott P, Drachman BM, Gottlieb SS, Hoffman JE, Hummel SL, Lenihan DJ, Ebede B, Gundapaneni B, Li B, Sultan MB, Shah SJ. Long-Term Survival With Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy. Circ Heart Fail. 2022 Jan;15(1):e008193. doi: 10.1161/CIRCHEARTFAILURE.120.008193. Epub 2021 Dec 20.
Results Reference
derived
PubMed Identifier
34779246
Citation
Hanna M, Damy T, Grogan M, Stewart M, Gundapaneni B, Sultan MB, Maurer MS. Tafamidis and quality of life in people with transthyretin amyloid cardiomyopathy in the study ATTR-ACT: A plain language summary. Future Cardiol. 2022 Mar;18(3):165-172. doi: 10.2217/fca-2021-0095. Epub 2021 Nov 15.
Results Reference
derived
PubMed Identifier
33895806
Citation
Rozenbaum MH, Garcia A, Grima D, Tran D, Bhambri R, Stewart M, Li B, Heeg B, Postma M, Masri A. Health impact of tafamidis in transthyretin amyloid cardiomyopathy patients: an analysis from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) and the open-label long-term extension studies. Eur Heart J Qual Care Clin Outcomes. 2022 Aug 17;8(5):529-538. doi: 10.1093/ehjqcco/qcab031.
Results Reference
derived
PubMed Identifier
33770392
Citation
Vong C, Boucher M, Riley S, Harnisch LO. Modeling of Survival and Frequency of Cardiovascular-Related Hospitalization in Patients with Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis. Am J Cardiovasc Drugs. 2021 Sep;21(5):535-543. doi: 10.1007/s40256-021-00464-y. Epub 2021 Mar 26.
Results Reference
derived
PubMed Identifier
33667442
Citation
Miller AB, Januzzi JL, O'Neill BJ, Gundapaneni B, Patterson TA, Sultan MB, Lopez-Sendon J. Causes of Cardiovascular Hospitalization and Death in Patients With Transthyretin Amyloid Cardiomyopathy (from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial [ATTR-ACT]). Am J Cardiol. 2021 Jun 1;148:146-150. doi: 10.1016/j.amjcard.2021.02.035. Epub 2021 Mar 3.
Results Reference
derived
PubMed Identifier
33309574
Citation
Rapezzi C, Elliott P, Damy T, Nativi-Nicolau J, Berk JL, Velazquez EJ, Boman K, Gundapaneni B, Patterson TA, Schwartz JH, Sultan MB, Maurer MS. Efficacy of Tafamidis in Patients With Hereditary and Wild-Type Transthyretin Amyloid Cardiomyopathy: Further Analyses From ATTR-ACT. JACC Heart Fail. 2021 Feb;9(2):115-123. doi: 10.1016/j.jchf.2020.09.011. Epub 2020 Dec 9.
Results Reference
derived
PubMed Identifier
33220323
Citation
Hanna M, Damy T, Grogan M, Stewart M, Gundapaneni B, Patterson TA, Schwartz JH, Sultan MB, Maurer MS. Impact of Tafamidis on Health-Related Quality of Life in Patients With Transthyretin Amyloid Cardiomyopathy (from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial). Am J Cardiol. 2021 Feb 15;141:98-105. doi: 10.1016/j.amjcard.2020.10.066. Epub 2020 Nov 19.
Results Reference
derived
PubMed Identifier
33070419
Citation
Damy T, Garcia-Pavia P, Hanna M, Judge DP, Merlini G, Gundapaneni B, Patterson TA, Riley S, Schwartz JH, Sultan MB, Witteles R. Efficacy and safety of tafamidis doses in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) and long-term extension study. Eur J Heart Fail. 2021 Feb;23(2):277-285. doi: 10.1002/ejhf.2027. Epub 2020 Nov 12.
Results Reference
derived
PubMed Identifier
32524297
Citation
Li B, Alvir J, Stewart M. Extrapolation of Survival Benefits in Patients with Transthyretin Amyloid Cardiomyopathy Receiving Tafamidis: Analysis of the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial. Cardiol Ther. 2020 Dec;9(2):535-540. doi: 10.1007/s40119-020-00179-2. Epub 2020 Jun 10.
Results Reference
derived
PubMed Identifier
30145929
Citation
Maurer MS, Schwartz JH, Gundapaneni B, Elliott PM, Merlini G, Waddington-Cruz M, Kristen AV, Grogan M, Witteles R, Damy T, Drachman BM, Shah SJ, Hanna M, Judge DP, Barsdorf AI, Huber P, Patterson TA, Riley S, Schumacher J, Stewart M, Sultan MB, Rapezzi C; ATTR-ACT Study Investigators. Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy. N Engl J Med. 2018 Sep 13;379(11):1007-1016. doi: 10.1056/NEJMoa1805689. Epub 2018 Aug 27.
Results Reference
derived
PubMed Identifier
28611125
Citation
Maurer MS, Elliott P, Merlini G, Shah SJ, Cruz MW, Flynn A, Gundapaneni B, Hahn C, Riley S, Schwartz J, Sultan MB, Rapezzi C; ATTR-ACT Study Investigators. Design and Rationale of the Phase 3 ATTR-ACT Clinical Trial (Tafamidis in Transthyretin Cardiomyopathy Clinical Trial). Circ Heart Fail. 2017 Jun;10(6):e003815. doi: 10.1161/CIRCHEARTFAILURE.116.003815.
Results Reference
derived
Links:
URL
https://trialinfoemail.pfizer.com/pages/landing.aspx?StudyID=B3461028&StudyName=Safety%20and%20Efficacy%20of%20Tafamidis%20in%20Patients%20with%20Transthyretin%20Cardiomyopathy
Description
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Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy

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