Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy

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)

Open label study to evaluate tafamidis for the treatment of transthyretin cardiomyopathy

Study Overview

• Status: Completed
• Conditions: Transthyretin (TTR) Amyloid Cardiomyopathy
• Intervention / Treatment: Drug: Tafamidis

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.

• Study Type: Interventional
• Enrollment (Actual): 1733
• Phase: Phase 3

Contacts and Locations

Study Locations

• Argentina
  • Ciudad Autónoma de Buenos Aires, Argentina, C1428ART
    • Instituto Cardiovascular de Buenos Aires
• Australia
  • New South Wales
    • Westmead, New South Wales, Australia, 2145
      • Westmead Hospital
  • Queensland
    • Woolloongabba, Queensland, Australia, 4102
      • Princess Alexandra Hospital
  • Victoria
    • Box Hill, Victoria, Australia, 3128
      • Eastern Health (Box Hill Hospital)
• Belgium
  • Leuven, Belgium, 3000
    • UZ Leuven
• Brazil
  • RJ
    • Rio de Janeiro, RJ, Brazil, 21941-913
      • Hospital Universitário Clementino Fraga Filho (UFRJ)
• Canada
  • Alberta
    • Calgary, Alberta, Canada, T2N 4Z6
      • University of Calgary/Foothills Medical Centre
  • British Columbia
    • Vancouver, British Columbia, Canada, V5Z 1M9
      • Vancouver General Hospital
  • Ontario
    • Toronto, Ontario, Canada, M5G 2C4
      • Toronto General Hospital
  • Quebec
    • Montreal, Quebec, Canada, H1T 1C8
      • Montreal Heart Institute
• Czechia
  • Brno, Czechia, 656 91
    • Fakultni nemocnice u sv. Anny v Brne
  • Praha 2, Czechia, 128 08
    • Vseobecna fakultni nemocnice v Praze
  • Praha 4, Czechia, 140 21
    • Institut klinicke a experimentalni mediciny
• France
  • Creteil, France, 94010
    • CHU Henri Mondor
  • Marseille, France, 13385
    • Hôpital de la Timone
  • Paris, France, 75018
    • Hôpital Bichat
  • Rennes, France, 35033
    • CHU de Rennes - Hôpital Pontchaillou
  • Toulouse, France, 31059
    • CHU de Toulouse - Hôpital Rangueil
  • CAN
    • Marseille, CAN, France, 13005
      • Hôpital de la Timone
• Germany
  • Heidelberg, Germany, 69120
    • Medical University of Heidelberg
  • Münster, Germany, 48149
    • Universitätsklinikum Münster
• Hong Kong
  • Hong Kong, Hong Kong
    • Queen Mary Hospital
  • Hong Kong, Hong Kong
    • Clinical trial Pharmacy
• Italy
  • Bologna, Italy, 40138
    • Azienda Ospedaliero-Universitaria Policlinico S. Orsola Malpighi
  • Firenze, Italy, 50134
    • Azienda Ospedaliero-Universitaria Careggi
  • Pavia, Italy, 27100
    • Fondazione IRCCS Policlinico San Matteo
• Japan
  • Nagano, Japan, 390-8621
    • Shinshu University Hospital
  • Fukuoka
    • Kurume-shi, Fukuoka, Japan, 830-0011
      • Kurume University Hospital
  • Kumamoto
    • Kumamoto-city, Kumamoto, Japan, 860-8556
      • Kumamoto University Hospital
• Netherlands
  • Groningen, Netherlands, 9713 GZ
    • University Medical Center Groningen
• Spain
  • LA Coruna
    • A Coruna, LA Coruna, Spain, 15006
      • Unidad de Insuficiencia Cardiaca Avanzada y Transplante Cardiaco
  • Madrid
    • Majadahonda, Madrid, Spain, 28222
      • Hospital Universitario Puerta de Hierro Majadahonda
• Sweden
  • Skellefteå, Sweden, 931 86
    • Skellefteå lasarett
  • Uppsala, Sweden, 751 85
    • Akademiska sjukhuset
• Taiwan
  • Taipei, Taiwan, 100
    • National Taiwan University Hospital
  • Taipei, Taiwan, 112
    • Taipei Veterans General Hospital
  • Taipei, Taiwan, 100
    • Investigational Drug Services
• United Kingdom
  • London, United Kingdom, EC1A 7BE
    • Barts Health NHS Trust, St Bartholomew's Hospital
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • The Kirklin Clinic of UAB Hospital
    • Birmingham, Alabama, United States, 35294
      • Cardiovascular Clinical Trials Unit (CCTU)
    • Birmingham, Alabama, United States, 35294
      • University Hospital, University of Alabama at Birmingham
  • California
    • Beverly Hills, California, United States, 90211
      • California Heart Center
    • Beverly Hills, California, United States, 90211
      • Cedars-Sinai Medical Care Foundation
    • La Jolla, California, United States, 92037
      • University of California, San Diego
    • La Jolla, California, United States, 92037
      • Altman Clinical Translational Research Institute
    • Los Angeles, California, United States, 90048
      • Cedars-Sinai Medical Center
    • San Diego, California, United States, 92103
      • University of California, San Diego Medical Center - Hillcrest
    • San Diego, California, United States, 92121
      • Center Adv Lab Medicine
    • San Francisco, California, United States, 94143
      • University of California
    • San Francisco, California, United States, 94143
      • UCSF Ambulatory Care Center
    • San Francisco, California, United States, 94158
      • UCSF Cardiovascular Care and Prevention Center
    • Stanford, California, United States, 94305
      • Stanford University Hospital and Clinics
  • Florida
    • Deerfield Beach, Florida, United States, 33136
      • Sylvester at Deerfield Beach
    • Miami, Florida, United States, 33136
      • University of Miami Hospital & Clinics/Sylvester Comprehensive Cancer Center
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Northwestern Memorial Hospital
    • Chicago, Illinois, United States, 60611
      • Northwestern Medical Group
    • Oak Lawn, Illinois, United States, 60453
      • Advocate Christ Medical Center
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • Johns Hopkins University
    • Baltimore, Maryland, United States, 21201
      • University of Maryland, Baltimore
  • Massachusetts
    • Boston, Massachusetts, United States, 02118
      • Boston Medical Center
    • Boston, Massachusetts, United States, 02118
      • Boston University Medical Center
    • Boston, Massachusetts, United States, 02118
      • Boston Medical Center Investigational Pharmacy
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • Michigan Medicine,University of Michigan
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic
    • Rochester, Minnesota, United States, 55902
      • Mayo Clinic Hospital-Rochester
  • New Jersey
    • Newark, New Jersey, United States, 07112
      • Newark Beth Israel Medical Center
  • New York
    • New York, New York, United States, 10016
      • NYU Langone Health
    • New York, New York, United States, 10032
      • Columbia University Irving Medical Center
    • New York, New York, United States, 10032
      • Center for Advanced Cardiac Care
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health & Science University
    • Portland, Oregon, United States, 97239
      • OHSU Center for Health and Healing
    • Portland, Oregon, United States, 97239
      • OHSU Research Pharmacy Services
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Penn Presbyterian Medical Center
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt Univ. Med. Ctr
  • Utah
    • Salt Lake City, Utah, United States, 84112
      • Huntsman Cancer Hospital
    • Salt Lake City, Utah, United States, 84132
      • University of Utah Hospitals & Clinics
    • Salt Lake City, Utah, United States, 84132
      • University of Utah, Division of Cardiovascular Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Tafamidis; Active treatment - 61 mg or if not available, tafamidis meglumine 80 mg	Drug: Tafamidis; Soft gel capsules administered once a day for 60 months

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to All-Cause Mortality: Cohort A	Time to all-cause mortality was calculated from first dose of randomized treatment in parent study (B3461028) to all-cause mortality events. All-cause mortality events included deaths, heart transplants and cardiac mechanical assist devices implantation treated as death. Treated participants from the parent study who discontinued prior to the start of this study were also included in this analysis as planned. Data from participants who dropped out for a liver-only transplantation were handled in the same manner as the data from all other censored participants. Censored participants were participants who completed study or discontinued from the study (including discontinued by sponsor or participants withdrew, or discontinued due to Adverse event (AE), or alive at the time of analysis. Kaplan Meier method was used for analysis. Therefore, this analysis was based on the pooled dose groups, as per the statistical analysis plan (SAP).	From first dose of randomized treatment in parent study (B3461028) up to 28 days post last dose of study treatment in current extension study (B3461045), [approximately up to 91 months]
Number of Participants With All-Cause Mortality Events: Cohort B	All-cause mortality included all participants who had discontinue for transplantation (i.e. 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. Data from participants who dropped out for a liver-only transplantation were handled in the same manner as the data from all other censored participants. Censored participants were participants who completed study or discontinued from the study (including discontinued by sponsor or participants withdrew, or discontinued due to AE), or alive at the time of analysis. Kaplan Meier method was used for analysis.	B3461045: From first dose of treatment up to 28 days post last dose of study treatment (approximately up to 61 months)
Number of Participants With Treatment-Emergent Adverse Events (AEs)	An AE was any untoward medical occurrence in a participant who received investigational product or medical device; the event need not necessarily have a causal relationship with the treatment or usage. A treatment emergent AE was defined as an event that emerged during the treatment period that was absent before treatment or worsened during the treatment period relative to the pretreatment state. AEs included both SAEs and all Non-SAEs. A serious adverse event (SAE) was any untoward medical occurrence at any dose that: resulted in death; was life-threatening (immediate risk of death); required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/ incapacity; resulted in congenital anomaly/birth defect; considered an important medical event.	B3461045: From first dose of treatment up to 28 days post last dose of study treatment (approximately up to 61 months)

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to Cardiovascular-Related Mortality Events: Cohort A	Time to cardiovascular-related mortality was calculated from first dose of randomized treatment in B3461028 . Participants who discontinued for transplantation (that is heart transplantation and combined heart and liver transplantation) or for implantation of cardiac mechanical assist device were treated as a death. Treated participants from the parent study who discontinued prior to the start of this study were also included in this analysis as planned. Kaplan-Meier method was used. Therefore, this analysis was based on the pooled dose groups, as per the SAP.	From first dose of randomized treatment in parent study (B3461028) up to 28 days post last dose of study treatment in current extension study (B3461045), [approximately up to 91 months]
Number of Participants With Cardiovascular Related Mortality Events: Cohort B	Deaths adjudicated as cardiovascular-related and indeterminate were reported. Cardiovascular-related mortality events included deaths, heart transplants and cardiac mechanical assist devices implantation treated as death. Cardiovascular relatedness was based on clinical judgement.	B3461045: From first dose of treatment up to 28 days post last dose of study treatment (approximately up to 61 months)
Mean Annualized Rate of All Cause Hospitalizations	Mean annual rate of all cause hospitalization was calculated for each participant as the participant's number of all cause hospitalizations divided by this participant's duration on study in years. Hospitalization was defined as any initial admission (even less than 24 hours) in a hospital or equivalent healthcare facility or any prolongation of an existing admission. Only all cause hospitalizations where the participant was admitted to a hospital during the current extension study were included in this analysis. Any hospitalizations prior to randomization date were not included.	B3461045: From first dose of treatment up to 28 days post last dose of study treatment (approximately up to 61 months)
Mean Annualized Rate of Cardiovascular (CV)-Related Hospitalizations	Mean annual rate of CV related hospitalization was calculated for each participant as the participant's number of CV related hospitalizations divided by this participant's duration on study in years. CV-related hospitalizations included hospitalizations due to heart failure, arrhythmia, myocardial infarction, stroke, and other cardiovascular-related events. Hospitalization was defined as any initial admission (even less than 24 hours) in a hospital or equivalent healthcare facility or any prolongation of an existing admission. Admission also included transfer within the hospital to an acute/intensive care unit (example: from the psychiatric wing to a medical floor, medical floor to a coronary care unit, or neurological floor to a tuberculosis unit).	B3461045: From first dose of treatment up to 28 days post last dose of study treatment (approximately up to 61 months)
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Clinical Summary Scores at Months 12, 30, 42, 60 and 90: Cohort A	KCCQ: 23-item, self-administered questionnaire that assesses health status and health-related quality of life in participants with heart failure. KCCQ quantifies 8 domains: physical limitations, symptom stability, symptom frequency, symptom burden, total symptom (mean of symptom frequency and symptom burden), self-efficacy, quality of life and social limitations). Scores were generated for each domain and scaled from 0 (worst) to 100 (best possible status). KCCQ-clinical summary score was calculated as mean of the following domains- physical limitation and total symptoms and transformed to a single score which ranged from 0 (worst) -100 (best possible status), higher scores = better health status. Data for change from baseline was reported as least square (LS) mean.	B3461028: Baseline (Day 1, pre-dose), Months 12, 30; B3461045: Months 42, 60 and 90 (time points were relative to Day 1 from B3461028; 42, 60 and 90 Months were 12, 30 and 60 Months of B3461045)
Change From Baseline in KCCQ Overall Score at Months 12, 30, 42, 60, 90: Cohort A	KCCQ: 23-item, self-administered questionnaire that assesses health status and health-related quality of life in participants with heart failure. KCCQ quantifies 8 domains: physical limitations, symptom stability, symptom frequency, symptom burden, total symptom (mean of symptom frequency and symptom burden), self-efficacy, quality of life and social limitations). Scores were generated for each domain and scaled from 0 (worst) to 100 (best possible status). KCCQ-overall score was calculated as mean of the following domains- physical limitation, total symptoms (average of symptom frequency & symptom burden), quality of life, social limitation and transformed to a single score which ranged from 0 (worst) -100 (best possible status), higher scores = better health status. Data for change from baseline was reported as LS mean.	B3461028: Baseline (Day 1, pre-dose), Months 12, 30; B3461045: Months 42, 60 and 90 (time points were relative to Day 1 from B3461028; 42, 60 and 90 Months were 12, 30 and 60 Months of B3461045)
Change From Baseline in KCCQ Total Symptom Score at Months 12, 30, 42, 60 and 90: Cohort A	KCCQ: 23-item, self-administered questionnaire that assesses health status and health-related quality of life in participants with heart failure. KCCQ quantifies 8 domains: physical limitations, symptom stability, symptom frequency, symptom burden, total symptom (mean of symptom frequency and symptom burden), self-efficacy, quality of life and social limitations). Scores were generated for each domain and scaled from 0 (worst) to 100 (best possible status). KCCQ Total Symptoms score was calculated as mean of the following domains- symptom frequency and symptom burden and transformed to a single score which ranged from 0 (worst) -100 (best possible status), higher scores = better health status. Data for change from baseline was reported as LS mean.	B3461028: Baseline (Day 1, pre-dose), Months 12, 30; B3461045: Months 42, 60 and 90 (time points were relative to Day 1 from B3461028; 42, 60 and 90 Months were 12, 30 and 60 Months of B3461045)
Change From Baseline in KCCQ Clinical Summary Scores at Months 12, 30 and 54: Cohort B	KCCQ: 23-item, self-administered questionnaire that assesses health status and health-related quality of life in participants with heart failure. KCCQ quantifies 8 domains: physical limitations, symptom stability, symptom frequency, symptom burden, total symptom (mean of symptom frequency and symptom burden), self-efficacy, quality of life and social limitations). Scores were generated for each domain and scaled from 0 (worst) to 100 (best possible status). KCCQ-clinical summary score was calculated as mean of the following domains- physical limitation and total symptoms and transformed to a single score which ranged from 0 (worst) -100 (best possible status), higher scores = better health status. Data for change from baseline was reported as mean.	B3461045: Baseline (Day 1, pre-dose), Months 12, 30 and 54
Change From Baseline in KCCQ Overall Score at Months 12, 30 and 54: Cohort B	KCCQ: 23-item, self-administered questionnaire that assesses health status and health-related quality of life in participants with heart failure. KCCQ quantifies 8 domains: physical limitations, symptom stability, symptom frequency, symptom burden, total symptom (mean of symptom frequency and symptom burden), self-efficacy, quality of life and social limitations). Scores were generated for each domain and scaled from 0 (worst) to 100 (best possible status). KCCQ-overall score was calculated as mean of the following domains- physical limitation, total symptoms (average of symptom frequency & symptom burden), quality of life, social limitation and transformed to a single score which ranged from 0 (worst) -100 (best possible status), higher scores = better health status. Data for change from baseline was reported as mean.	B3461045: Baseline (Day 1, pre-dose), Months 12, 30 and 54
Change From Baseline in KCCQ Total Symptom Score at Months 12, 30 and 54: Cohort B	KCCQ: 23-item, self-administered questionnaire that assesses health status and health-related quality of life in participants with heart failure. KCCQ quantifies 8 domains: physical limitations, symptom stability, symptom frequency, symptom burden, total symptom (mean of symptom frequency and symptom burden), self-efficacy, quality of life and social limitations). Scores were generated for each domain and scaled from 0 (worst) to 100 (best possible status). KCCQ Total Symptoms score was calculated as mean of the following domains- symptom frequency and symptom burden and transformed to a single score which ranged from 0 (worst) -100 (best possible status), higher scores = better health status. Data for change from baseline was reported as mean.	B3461045: Baseline (Day 1, pre-dose), Months 12, 30 and 54
Number of Participants With Shift From Baseline in New York Heart Association (NYHA) Classification at Month 6, 12, 18, 24, 30, 36, 42, 48, 54 and 60	NYHA classification: Class I: Participants with cardiovascular disease (CVD) but without resulting limitation of physical activity. Ordinary physical activity does not cause undue fatigue, palpitation, dyspnea, or anginal pain; Class II: Participants with CVD resulting in slight limitation of physical activity and were comfortable at rest. Ordinary physical activity results in fatigue, palpitation, dyspnea, or angina pain; Class III: participants with CVD resulting in marked limitation of physical activity and were comfortable at rest. Less than ordinary physical activity causes fatigue, palpitation, dyspnea, or anginal pain; Class IV: participants with CVD resulting in inability to carry on any physical activity without discomfort. Symptoms of cardiac insufficiency or of the anginal syndrome may be present even at rest. If any physical activity was undertaken, discomfort was increased.	B3461045: Baseline (Day 1, pre-dose), Months 6, 12, 18, 24, 30, 36, 42, 48, 54 and 60
Change From Baseline in Body Mass Index (BMI) at Months 6, 12, 18, 24, 30, 36, 42, 48, 54 and 60	BMI was calculated as: body weight in kilogram (kg) divided by (/) square of body height measurement in meters square (m^2).	B3461045: Baseline (Day 1, pre-dose), Months 6, 12, 18, 24, 30, 36, 42, 48, 54 and 60
Change From Baseline in Modified Body Mass Index (mBMI) at Months 6, 12, 18, 24, 30, 36, 42, 48, 54 and 60	Modified (m)BMI of participants was calculated to determine if there was any gastrointestinal involvement in participants. The mBMI was calculated by multiplying BMI by serum albumin concentration gram/liter (g/L).	B3461045: Baseline (Day 1, pre-dose), Months 6, 12, 18, 24, 30, 36, 42, 48, 54 and 60
Change From Baseline in Troponin I Concentration at Months 6, 12, 18, 24, 30, 36, 42, 48, 54 and 60	Troponin I is the cardiac markers. Higher level of the marker was indicative of heart damage. It was measure in nanogram per milliliter (ng/mL). Safety analysis population included all participants (Cohorts A and B) who were enrolled in this study and taken at least 1 dose of study medication.	B3461045: Baseline (Day 1, pre-dose), Months 6, 12, 18, 24, 30, 36, 42, 48, 54 and 60
Change From Baseline in N-Terminal Prohormone Brain Natriuretic Peptide (NT-proBNP) Concentration at Months 6, 12, 18, 24, 30, 36, 42, 48, 54 and 60	NT-proBNP was a cardiac marker which had the prognostic value for participants with heart failure or left ventricular dysfunction. It was measure in picomole per liter (pmole/L). Higher level of the marker was indicative of heart damage. Safety analysis population included all participants (Cohorts A and B) who were enrolled in this study and taken at least 1 dose of study medication.	B3461045: Baseline (Day 1, pre-dose), Months 6, 12, 18, 24, 30, 36, 42, 48, 54 and 60

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

General Publications

• 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.
• 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.
• 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.

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): June 13, 2016
• Primary Completion (Actual): October 26, 2023
• Study Completion (Actual): November 2, 2023

Study Registration Dates

• First Submitted: June 1, 2016
• First Submitted That Met QC Criteria: June 1, 2016
• First Posted (Estimated): June 6, 2016

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 28, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Keywords: amyloid; amyloidosis; transthyretin; TTR; ATTR-CM
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Heart Diseases; Cardiomyopathies
• Other Study ID Numbers: B3461045; 2016-000868-42 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD 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.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No