Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy

NCT ID: NCT02791230

Last Updated: 2025-02-03

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

1733 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-06-13

Study Completion Date

2023-11-02

Brief Summary

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

Detailed Description

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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.

Conditions

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Transthyretin (TTR) Amyloid Cardiomyopathy

Study Design

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Allocation Method

NA

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Tafamidis

Active treatment - 61 mg or if not available, tafamidis meglumine 80 mg

Group Type EXPERIMENTAL

Tafamidis

Intervention Type DRUG

Soft gel capsules administered once a day for 60 months

Interventions

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Tafamidis

Soft gel capsules administered once a day for 60 months

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Pfizer

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

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The Kirklin Clinic of UAB Hospital

Birmingham, Alabama, United States

Site Status

Cardiovascular Clinical Trials Unit (CCTU)

Birmingham, Alabama, United States

Site Status

University Hospital, University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

California Heart Center

Beverly Hills, California, United States

Site Status

Cedars-Sinai Medical Care Foundation

Beverly Hills, California, United States

Site Status

Altman Clinical Translational Research Institute

La Jolla, California, United States

Site Status

University of California, San Diego

La Jolla, California, United States

Site Status

Cedars-Sinai Medical Center

Los Angeles, California, United States

Site Status

University of California, San Diego Medical Center - Hillcrest

San Diego, California, United States

Site Status

Center Adv Lab Medicine

San Diego, California, United States

Site Status

UCSF Ambulatory Care Center

San Francisco, California, United States

Site Status

University of California

San Francisco, California, United States

Site Status

UCSF Cardiovascular Care and Prevention Center

San Francisco, California, United States

Site Status

Stanford University Hospital and Clinics

Stanford, California, United States

Site Status

Sylvester at Deerfield Beach

Deerfield Beach, Florida, United States

Site Status

University of Miami Hospital & Clinics/Sylvester Comprehensive Cancer Center

Miami, Florida, United States

Site Status

Northwestern Medical Group

Chicago, Illinois, United States

Site Status

Northwestern Memorial Hospital

Chicago, Illinois, United States

Site Status

Advocate Christ Medical Center

Oak Lawn, Illinois, United States

Site Status

University of Maryland, Baltimore

Baltimore, Maryland, United States

Site Status

Johns Hopkins University

Baltimore, Maryland, United States

Site Status

Boston Medical Center Investigational Pharmacy

Boston, Massachusetts, United States

Site Status

Boston Medical Center

Boston, Massachusetts, United States

Site Status

Boston University Medical Center

Boston, Massachusetts, United States

Site Status

Michigan Medicine,University of Michigan

Ann Arbor, Michigan, United States

Site Status

Mayo Clinic Hospital-Rochester

Rochester, Minnesota, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

Newark Beth Israel Medical Center

Newark, New Jersey, United States

Site Status

NYU Langone Health

New York, New York, United States

Site Status

Center for Advanced Cardiac Care

New York, New York, United States

Site Status

Columbia University Irving Medical Center

New York, New York, United States

Site Status

Duke University

Durham, North Carolina, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

OHSU Center for Health and Healing

Portland, Oregon, United States

Site Status

OHSU Research Pharmacy Services

Portland, Oregon, United States

Site Status

Oregon Health & Science University

Portland, Oregon, United States

Site Status

Penn Presbyterian Medical Center

Philadelphia, Pennsylvania, United States

Site Status

Vanderbilt Univ. Med. Ctr

Nashville, Tennessee, United States

Site Status

Huntsman Cancer Hospital

Salt Lake City, Utah, United States

Site Status

University of Utah Hospitals & Clinics

Salt Lake City, Utah, United States

Site Status

University of Utah, Division of Cardiovascular Medicine

Salt Lake City, Utah, United States

Site Status

Instituto Cardiovascular de Buenos Aires

Buenos Aires, , Argentina

Site Status

Westmead Hospital

Westmead, New South Wales, Australia

Site Status

Princess Alexandra Hospital

Woolloongabba, Queensland, Australia

Site Status

Eastern Health (Box Hill Hospital)

Box Hill, Victoria, Australia

Site Status

UZ Leuven

Leuven, , Belgium

Site Status

Hospital Universitário Clementino Fraga Filho (UFRJ)

Rio de Janeiro, Rio de Janeiro, Brazil

Site Status

University of Calgary/Foothills Medical Centre

Calgary, Alberta, Canada

Site Status

Vancouver General Hospital

Vancouver, British Columbia, Canada

Site Status

Toronto General Hospital

Toronto, Ontario, Canada

Site Status

Montreal Heart Institute

Montreal, Quebec, Canada

Site Status

Fakultni nemocnice u sv. Anny v Brne

Brno, , Czechia

Site Status

Vseobecna fakultni nemocnice v Praze

Prague, , Czechia

Site Status

Institut klinicke a experimentalni mediciny

Prague, , Czechia

Site Status

Hopital de La Timone

Marseille, CAN, France

Site Status

CHU Henri Mondor

Créteil, , France

Site Status

Hôpital De La Timone

Marseille, , France

Site Status

Hopital Bichat

Paris, , France

Site Status

CHU de Rennes - Hopital Pontchaillou

Rennes, , France

Site Status

CHU de Rennes - Hôpital Pontchaillou

Rennes, , France

Site Status

CHU de Toulouse - Hôpital Rangueil

Toulouse, , France

Site Status

Medical University of Heidelberg

Heidelberg, , Germany

Site Status

Universitätsklinikum Münster

Münster, , Germany

Site Status

Clinical Trial Pharmacy

Hong Kong, , Hong Kong

Site Status

Queen Mary Hospital

Hong Kong, , Hong Kong

Site Status

Azienda Ospedaliero-Universitaria Policlinico S. Orsola Malpighi

Bologna, , Italy

Site Status

Azienda Ospedaliero-Universitaria Careggi

Florence, , Italy

Site Status

Fondazione IRCCS Policlinico San Matteo

Pavia, , Italy

Site Status

Kurume University Hospital

Kurume-shi, Fukuoka, Japan

Site Status

Kumamoto University Hospital

Kumamoto, Kumamoto, Japan

Site Status

Shinshu University Hospital

Nagano, , Japan

Site Status

University Medical Center Groningen

Groningen, , Netherlands

Site Status

Unidad de Insuficiencia Cardiaca Avanzada y Transplante Cardiaco

A Coruña, LA Coruna, Spain

Site Status

Hospital Universitario Puerta de Hierro Majadahonda

Majadahonda, Madrid, Spain

Site Status

Skellefteå Lasarett

Skellefteå, , Sweden

Site Status

Akademiska Sjukhuset

Uppsala, , Sweden

Site Status

Investigational Drug Services

Taipei, , Taiwan

Site Status

National Taiwan University Hospital

Taipei, , Taiwan

Site Status

Taipei Veterans General Hospital

Taipei, , Taiwan

Site Status

Barts Health NHS Trust, St Bartholomew's Hospital

London, , United Kingdom

Site Status

Countries

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United States Argentina Australia Belgium Brazil Canada Czechia France Germany Hong Kong Italy Japan Netherlands Spain Sweden Taiwan United Kingdom

References

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Crespo-Leiro MG, Hanna M, Damy T, Delgado D, Ebede B, Marino V, Wang R, Maurer MS, Garcia-Pavia P, Drachman BM. Early Access to Tafamidis for Patients With Transthyretin Amyloid Cardiomyopathy. JACC Adv. 2025 Sep 10;4(10 Pt 2):102122. doi: 10.1016/j.jacadv.2025.102122. Online ahead of print.

Reference Type DERIVED
PMID: 40934814 (View on PubMed)

Damy T, Wang R, Maurer MS, Gillmore JD, Fontana M. Long-term efficacy of tafamidis in patients with transthyretin amyloid cardiomyopathy by National Amyloidosis Centre stage. Eur J Heart Fail. 2025 Jun 9. doi: 10.1002/ejhf.3696. Online ahead of print.

Reference Type DERIVED
PMID: 40488446 (View on PubMed)

Drachman B, Damy T, Hanna M, Wang R, Angeli FS, Garcia-Pavia P. Long-term tafamidis efficacy in patients with transthyretin amyloid cardiomyopathy by baseline left ventricular ejection fraction. Eur J Heart Fail. 2024 Sep;26(9):2038-2046. doi: 10.1002/ejhf.3330. Epub 2024 Jun 26.

Reference Type DERIVED
PMID: 38932583 (View on PubMed)

Garcia-Pavia P, Sultan MB, Gundapaneni B, Sekijima Y, Perfetto F, Hanna M, Witteles R. Tafamidis Efficacy Among Octogenarian Patients in the Phase 3 ATTR-ACT and Ongoing Long-Term Extension Study. JACC Heart Fail. 2024 Jan;12(1):150-160. doi: 10.1016/j.jchf.2023.08.032. Epub 2023 Nov 8.

Reference Type DERIVED
PMID: 37943223 (View on PubMed)

Elliott P, Gundapaneni B, Sultan MB, Ines M, Garcia-Pavia P. Improved long-term survival with tafamidis treatment in patients with transthyretin amyloid cardiomyopathy and severe heart failure symptoms. Eur J Heart Fail. 2023 Nov;25(11):2060-2064. doi: 10.1002/ejhf.2974. Epub 2023 Jul 26.

Reference Type DERIVED
PMID: 37434378 (View on PubMed)

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 in people with transthyretin amyloid cardiomyopathy who took tafamidis: A Plain Language Summary. Future Cardiol. 2023 Jan;19(1):7-17. doi: 10.2217/fca-2022-0096. Epub 2023 Jan 30.

Reference Type DERIVED
PMID: 36715498 (View on PubMed)

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.

Reference Type DERIVED
PMID: 34923848 (View on PubMed)

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.

Reference Type DERIVED
PMID: 33070419 (View on PubMed)

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.

Reference Type DERIVED
PMID: 32524297 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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https://pmiform.com/clinical-trial-info-request?StudyID=B3461045

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

Other Identifiers

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2016-000868-42

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

B3461045

Identifier Type: -

Identifier Source: org_study_id

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