Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy

January 9, 2024 updated by: Pfizer

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

Intervention / Treatment

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)

1735

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

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
        • Gordon and Leslie Diamond Health Care Centre
      • Vancouver, British Columbia, Canada, V5Z 1M9
        • Vancouver General Hospital
    • Ontario
      • Toronto, Ontario, Canada, M5G 2C4
        • Toronto General Hospital
      • Toronto, Ontario, Canada, M5G 2C4
        • University Health Network-Toronto General Hospital
    • Quebec
      • Montreal, Quebec, Canada, H1T 1C8
        • Montreal Heart Institute
      • Montreal, Quebec, Canada, H1T 1C8
        • Montreal Heart Institute-Research Center
  • 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
      • Heidelberg, Germany, 69120
        • Medizinisches Universitatsklinikum 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 Ospedaliera 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
      • A Coruña, Spain, 15006
        • Complejo Hospitalario Universitario A Coruña
    • 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
      • Taipei, Taiwan, 112
        • Clinical Trial Pharmacy
  • 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
        • Altman Clinical Translational Research Institute
      • La Jolla, California, United States, 92037
        • UCSD Sulpizio Cardiovascular Center
      • 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
      • Stanford, California, United States, 94305
        • Stanford Hospital and Clinics Investigational Pharmacy
    • Florida
      • Deerfield Beach, Florida, United States, 33442
        • 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 University
      • 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 - Department of Pharmacy Services - Research Pharmacy
      • 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, 10034
        • Columbia University Medical Center- Clinical Cardiovascular Research Laboratory for the Elderly
    • 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

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Tafamidis
Active treatment - 61 mg or if not available, tafamidis megulmine 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
All-cause mortality and incidence of treatment emergent adverse events
Time Frame: Baseline to Month 60
Safety endpoints for analysis of the treatment groups
Baseline to Month 60

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Cardiovascular-related mortality
Time Frame: Baseline to Month 60
Cardiovascular-related mortality
Baseline to Month 60
All-cause hospitalization
Time Frame: Baseline to Month 60
Frequency of all-cause hospitalization
Baseline to Month 60
Cardiovascular-related hospitalization
Time Frame: Baseline to Month 60
Frequency of cardiovascular-related hospitalization
Baseline to Month 60
Kansas City Cardiomyopathy Questionnaire
Time Frame: Baseline to Month 60
Change from baseline at each visit in Kansas City Cardiomyopathy Questionnaire
Baseline to Month 60
New York Heart Association classification
Time Frame: Baseline to Month 60
New York Heart Association (NYHA) classification at each visit
Baseline to Month 60
Body Mass Index/modified Body Mass Index
Time Frame: Baseline to Month 60
Change from baseline in Body Mass Index/modified Body Mass Index at each visit
Baseline to Month 60
Cardiac biomarkers
Time Frame: Baseline to Month 60
Change from baseline in NT-proBNP concentration at each visit
Baseline to Month 60
Cardiac biomarkers
Time Frame: Baseline to Month 60
Change from baseline in Troponin I concentration at each visit
Baseline to Month 60

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Pfizer

Investigators

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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 13, 2016

Primary Completion (Actual)

October 26, 2023

Study Completion (Actual)

November 23, 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)

January 11, 2024

Last Update Submitted That Met QC Criteria

January 9, 2024

Last Verified

January 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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