Back to resultsLong-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy
Primary Purpose
Transthyretin (TTR) Amyloid Cardiomyopathy
Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Tafamidis
Sponsored by
About this trial
This is an interventional treatment trial for Transthyretin (TTR) Amyloid Cardiomyopathy focused on measuring amyloidosis, amyloid, transthyretin, TTR, ATTR-CM
Eligibility Criteria
Inclusion Criteria:
Cohort A: Completion of 30 months of study treatment on Pfizer Protocol B3461028
Cohort B: Patients in specific countries diagnosed with ATTR-CM who did not previously participate in Pfizer Study B3461028
Exclusion Criteria:
-Liver and/or heart transplant, or implanted cardiac mechanical assist device
Sites / Locations
- The Kirklin Clinic of UAB Hospital
- Cardiovascular Clinical Trials Unit (CCTU)
- University Hospital, University of Alabama at Birmingham
- California Heart Center
- Cedars-Sinai Medical Care Foundation
- Altman Clinical Translational Research Institute
- UCSD Sulpizio Cardiovascular Center
- Cedars-Sinai Medical Center
- University of California, San Diego Medical Center - Hillcrest
- Center Adv Lab Medicine
- UCSF Ambulatory Care Center
- University of California
- UCSF Cardiovascular Care and Prevention Center
- Stanford Hospital and Clinics Investigational Pharmacy
- Stanford University Hospital and Clinics
- Sylvester at Deerfield Beach
- University of Miami Hospital & Clinics/Sylvester Comprehensive Cancer Center
- Northwestern Medical Group
- Northwestern Memorial Hospital
- Northwestern University
- Advocate Christ Medical Center
- University of Maryland, Baltimore
- Johns Hopkins University
- Boston Medical Center Investigational Pharmacy
- Boston Medical Center
- Boston University Medical Center
- Michigan Medicine - Department of Pharmacy Services - Research Pharmacy
- Michigan Medicine,University of Michigan
- Mayo Clinic Hospital-Rochester
- Mayo Clinic
- Newark Beth Israel Medical Center
- NYU Langone Health
- Columbia University Medical Center- Clinical Cardiovascular Research Laboratory for the Elderly
- Duke University
- Cleveland Clinic
- OHSU Center for Health and Healing
- OHSU Research Pharmacy Services
- Oregon Health & Science University
- Penn Presbyterian Medical Center
- Vanderbilt Univ. Med. Ctr
- Huntsman Cancer Hospital
- University of Utah Hospitals & Clinics
- University of Utah, Division of Cardiovascular Medicine
- Instituto Cardiovascular de Buenos Aires
- Westmead Hospital
- Princess Alexandra Hospital
- Eastern Health (Box Hill Hospital)
- UZ Leuven
- Hospital Universitário Clementino Fraga Filho (UFRJ)
- University of Calgary/Foothills Medical Centre
- Gordon and Leslie Diamond Health Care Centre
- Vancouver General Hospital
- Toronto General Hospital
- University health network-Toronto General Hospital
- Montreal Heart Institute-Research Center
- Montreal Heart Institute
- Fakultni nemocnice u sv. Anny v Brne
- Vseobecna fakultni nemocnice v Praze
- Institut klinicke a experimentalni mediciny
- Hopital de La Timone
- CHU Henri Mondor
- Hôpital De La Timone
- Hopital Bichat
- CHU de Rennes - Hopital Pontchaillou
- CHU de Rennes - Hôpital Pontchaillou
- CHU de Toulouse - Hôpital Rangueil
- Medical University of Heidelberg
- Medizinisches Universitatsklinikum Heidelberg
- Universitätsklinikum Münster
- Clinical Trial Pharmacy
- Queen Mary Hospital
- Azienda Ospedaliero-Universitaria Policlinico S. Orsola Malpighi
- Azienda Ospedaliera Universitaria Careggi
- Fondazione IRCCS Policlinico San Matteo
- Kurume University Hospital
- Kumamoto University Hospital
- Shinshu University Hospital
- University Medical Center Groningen
- Hospital Universitario Puerta de Hierro Majadahonda
- Complejo Hospitalario Universitario A Coruña
- Skellefteå Lasarett
- Akademiska Sjukhuset
- Investigational Drug Services
- National Taiwan University Hospital
- Clinical Trial Pharmacy
- Taipei Veterans General Hospital
- Barts Health NHS Trust, St Bartholomew's Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Tafamidis
Arm Description
Active treatment - 61 mg or if not available, tafamidis megulmine 80 mg
Outcomes
Primary Outcome Measures
All-cause mortality and incidence of treatment emergent adverse events
Safety endpoints for analysis of the treatment groups
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02791230
Brief Title
Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy
Official Title
A PHASE 3 MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE SAFETY OF DAILY ORAL DOSING OF TAFAMIDIS MEGLUMINE (PF-06291826-83) 20 MG OR 80 MG [OR TAFAMIDIS (PF-06291826-00) 61 MG] IN SUBJECTS DIAGNOSED WITH TRANSTHYRETIN CARDIOMYOPATHY (ATTR-CM)
Study Type
Interventional
2. Study Status
Record Verification Date
December 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
June 13, 2016 (Actual)
Primary Completion Date
February 16, 2027 (Anticipated)
Study Completion Date
February 16, 2027 (Anticipated)
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
Open label study to evaluate tafamidis for the treatment of transthyretin cardiomyopathy
Detailed Description
Global Phase 3, open label long term extension safety study designed to obtain additional safety data for tafamidis meglumine 20 mg and 80 mg (or tafamidis 61 mg where available), and to continue to provide enrolled subjects with tafamidis for up to 60 months, or until subject has access to tafamidis for ATTR CM via prescription, whichever occurs first.
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, ATTR-CM
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
1735 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Tafamidis
Arm Type
Experimental
Arm Description
Active treatment - 61 mg or if not available, tafamidis megulmine 80 mg
Intervention Type
Drug
Intervention Name(s)
Tafamidis
Intervention Description
Soft gel capsules administered once a day for 60 months
Primary Outcome Measure Information:
Title
All-cause mortality and incidence of treatment emergent adverse events
Description
Safety endpoints for analysis of the treatment groups
Time Frame
Baseline to Month 60
Other Pre-specified Outcome Measures:
Title
Cardiovascular-related mortality
Description
Cardiovascular-related mortality
Time Frame
Baseline to Month 60
Title
All-cause hospitalization
Description
Frequency of all-cause hospitalization
Time Frame
Baseline to Month 60
Title
Cardiovascular-related hospitalization
Description
Frequency of cardiovascular-related hospitalization
Time Frame
Baseline to Month 60
Title
Kansas City Cardiomyopathy Questionnaire
Description
Change from baseline at each visit in Kansas City Cardiomyopathy Questionnaire
Time Frame
Baseline to Month 60
Title
New York Heart Association classification
Description
New York Heart Association (NYHA) classification at each visit
Time Frame
Baseline to Month 60
Title
Body Mass Index/modified Body Mass Index
Description
Change from baseline in Body Mass Index/modified Body Mass Index at each visit
Time Frame
Baseline to Month 60
Title
Cardiac biomarkers
Description
Change from baseline in NT-proBNP concentration at each visit
Time Frame
Baseline to Month 60
Title
Cardiac biomarkers
Description
Change from baseline in Troponin I concentration at each visit
Time Frame
Baseline to Month 60
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Cohort A: Completion of 30 months of study treatment on Pfizer Protocol B3461028
Cohort B: Patients in specific countries diagnosed with ATTR-CM who did not previously participate in Pfizer Study B3461028
Exclusion Criteria:
-Liver and/or heart transplant, 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 of UAB Hospital
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Cardiovascular Clinical Trials Unit (CCTU)
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Facility Name
University Hospital, University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Facility Name
California Heart Center
City
Beverly Hills
State/Province
California
ZIP/Postal Code
90211
Country
United States
Facility Name
Cedars-Sinai Medical Care Foundation
City
Beverly Hills
State/Province
California
ZIP/Postal Code
90211
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
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Facility Name
University of California, San Diego Medical Center - Hillcrest
City
San Diego
State/Province
California
ZIP/Postal Code
92103
Country
United States
Facility Name
Center Adv Lab Medicine
City
San Diego
State/Province
California
ZIP/Postal Code
92121
Country
United States
Facility Name
UCSF Ambulatory Care Center
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
University of California
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
UCSF Cardiovascular Care and Prevention Center
City
San Francisco
State/Province
California
ZIP/Postal Code
94158
Country
United States
Facility Name
Stanford Hospital and Clinics Investigational Pharmacy
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Stanford University Hospital and Clinics
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Sylvester at Deerfield Beach
City
Deerfield Beach
State/Province
Florida
ZIP/Postal Code
33442
Country
United States
Facility Name
University of Miami Hospital & Clinics/Sylvester Comprehensive Cancer Center
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
Northwestern University
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Advocate Christ Medical Center
City
Oak Lawn
State/Province
Illinois
ZIP/Postal Code
60453
Country
United States
Facility Name
University of Maryland, Baltimore
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
Boston Medical Center Investigational Pharmacy
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
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States
Facility Name
Michigan Medicine - Department of Pharmacy Services - Research Pharmacy
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
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 Hospital-Rochester
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55902
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
NYU Langone Health
City
New York
State/Province
New York
ZIP/Postal Code
10016
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
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 & Science University
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 Univ. Med. Ctr
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Huntsman Cancer Hospital
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States
Facility Name
University of Utah Hospitals & Clinics
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
University of Utah, Division of Cardiovascular Medicine
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
Instituto Cardiovascular de Buenos Aires
City
Ciudad Autónoma de Buenos Aires
ZIP/Postal Code
C1428ART
Country
Argentina
Facility Name
Westmead Hospital
City
Westmead
State/Province
New South Wales
ZIP/Postal Code
2145
Country
Australia
Facility Name
Princess Alexandra Hospital
City
Woolloongabba
State/Province
Queensland
ZIP/Postal Code
4102
Country
Australia
Facility Name
Eastern Health (Box Hill Hospital)
City
Box Hill
State/Province
Victoria
ZIP/Postal Code
3128
Country
Australia
Facility Name
UZ Leuven
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
Hospital Universitário 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
Gordon and Leslie Diamond Health Care Centre
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V5Z 1M9
Country
Canada
Facility Name
Vancouver General Hospital
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V5Z 1M9
Country
Canada
Facility Name
Toronto General Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 2C4
Country
Canada
Facility Name
University health network-Toronto General Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 2C4
Country
Canada
Facility Name
Montreal Heart Institute-Research Center
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H1T 1C8
Country
Canada
Facility Name
Montreal Heart Institute
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H1T 1C8
Country
Canada
Facility Name
Fakultni 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
Institut klinicke a experimentalni mediciny
City
Praha 4
ZIP/Postal Code
140 21
Country
Czechia
Facility Name
Hopital de La Timone
City
Marseille
State/Province
CAN
ZIP/Postal Code
13005
Country
France
Facility Name
CHU Henri Mondor
City
Creteil
ZIP/Postal Code
94010
Country
France
Facility Name
Hôpital De La Timone
City
Marseille
ZIP/Postal Code
13385
Country
France
Facility Name
Hopital Bichat
City
Paris
ZIP/Postal Code
75018
Country
France
Facility Name
CHU de Rennes - Hopital Pontchaillou
City
Rennes
ZIP/Postal Code
35033
Country
France
Facility Name
CHU de Rennes - Hôpital Pontchaillou
City
Rennes
ZIP/Postal Code
35033
Country
France
Facility Name
CHU de Toulouse - Hôpital Rangueil
City
Toulouse
ZIP/Postal Code
31059
Country
France
Facility Name
Medical University of Heidelberg
City
Heidelberg
ZIP/Postal Code
69120
Country
Germany
Facility Name
Medizinisches Universitatsklinikum Heidelberg
City
Heidelberg
ZIP/Postal Code
69120
Country
Germany
Facility Name
Universitätsklinikum Münster
City
Münster
ZIP/Postal Code
48149
Country
Germany
Facility Name
Clinical Trial Pharmacy
City
Hong Kong
Country
Hong Kong
Facility Name
Queen Mary Hospital
City
Hong Kong
Country
Hong Kong
Facility Name
Azienda Ospedaliero-Universitaria Policlinico S. Orsola Malpighi
City
Bologna
ZIP/Postal Code
40138
Country
Italy
Facility Name
Azienda Ospedaliera Universitaria Careggi
City
Firenze
ZIP/Postal Code
50134
Country
Italy
Facility Name
Fondazione IRCCS Policlinico San 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
860-8556
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
Complejo Hospitalario Universitario A Coruña
City
A Coruña
ZIP/Postal Code
15006
Country
Spain
Facility Name
Skellefteå Lasarett
City
Skellefteå
ZIP/Postal Code
931 86
Country
Sweden
Facility Name
Akademiska Sjukhuset
City
Uppsala
ZIP/Postal Code
751 85
Country
Sweden
Facility Name
Investigational Drug Services
City
Taipei
ZIP/Postal Code
100
Country
Taiwan
Facility Name
National Taiwan University Hospital
City
Taipei
ZIP/Postal Code
100
Country
Taiwan
Facility Name
Clinical Trial Pharmacy
City
Taipei
ZIP/Postal Code
112
Country
Taiwan
Facility Name
Taipei Veterans General Hospital
City
Taipei
ZIP/Postal Code
112
Country
Taiwan
Facility Name
Barts Health NHS Trust, St Bartholomew's Hospital
City
London
ZIP/Postal Code
EC1A 7BE
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
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
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
Links:
URL
https://pmiform.com/clinical-trial-info-request?StudyID=B3461045
Description
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Learn more about this trial
Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy
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