Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy
- Registration Number
- NCT02791230
- Lead Sponsor
- Pfizer
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 1733
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
-Liver and/or heart transplant, or implanted cardiac mechanical assist device
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Tafamidis Tafamidis Active treatment - 61 mg or if not available, tafamidis meglumine 80 mg
- Primary Outcome Measures
Name Time Method Time to All-Cause Mortality: Cohort A 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] 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).
Number of Participants With All-Cause Mortality Events: Cohort B B3461045: From first dose of treatment up to 28 days post last dose of study treatment (approximately up to 61 months) 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.
Number of Participants With Treatment-Emergent Adverse Events (AEs) B3461045: From first dose of treatment up to 28 days post last dose of study treatment (approximately up to 61 months) 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.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (80)
Boston Medical Center Investigational Pharmacy
🇺🇸Boston, Massachusetts, United States
Northwestern Medical Group
🇺🇸Chicago, Illinois, United States
Northwestern Memorial Hospital
🇺🇸Chicago, Illinois, United States
Advocate Christ Medical Center
🇺🇸Oak Lawn, Illinois, United States
University of Maryland, Baltimore
🇺🇸Baltimore, Maryland, United States
Johns Hopkins University
🇺🇸Baltimore, Maryland, United States
The Kirklin Clinic of UAB Hospital
🇺🇸Birmingham, Alabama, United States
Cardiovascular Clinical Trials Unit (CCTU)
🇺🇸Birmingham, Alabama, United States
University Hospital, University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
California Heart Center
🇺🇸Beverly Hills, California, United States
Cedars-Sinai Medical Care Foundation
🇺🇸Beverly Hills, California, United States
Altman Clinical Translational Research Institute
🇺🇸La Jolla, California, United States
University of California, San Diego
🇺🇸La Jolla, California, United States
Cedars-Sinai Medical Center
🇺🇸Los Angeles, California, United States
University of California, San Diego Medical Center - Hillcrest
🇺🇸San Diego, California, United States
Center Adv Lab Medicine
🇺🇸San Diego, California, United States
UCSF Ambulatory Care Center
🇺🇸San Francisco, California, United States
University of California
🇺🇸San Francisco, California, United States
UCSF Cardiovascular Care and Prevention Center
🇺🇸San Francisco, California, United States
Stanford University Hospital and Clinics
🇺🇸Stanford, California, United States
Sylvester at Deerfield Beach
🇺🇸Deerfield Beach, Florida, United States
University of Miami Hospital & Clinics/Sylvester Comprehensive Cancer Center
🇺🇸Miami, Florida, United States
Boston Medical Center
🇺🇸Boston, Massachusetts, United States
Boston University Medical Center
🇺🇸Boston, Massachusetts, United States
Michigan Medicine,University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Mayo Clinic Hospital-Rochester
🇺🇸Rochester, Minnesota, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Newark Beth Israel Medical Center
🇺🇸Newark, New Jersey, United States
NYU Langone Health
🇺🇸New York, New York, United States
Center for Advanced Cardiac Care
🇺🇸New York, New York, United States
Columbia University Irving Medical Center
🇺🇸New York, New York, United States
Duke University
🇺🇸Durham, North Carolina, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
OHSU Center for Health and Healing
🇺🇸Portland, Oregon, United States
OHSU Research Pharmacy Services
🇺🇸Portland, Oregon, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
Penn Presbyterian Medical Center
🇺🇸Philadelphia, Pennsylvania, United States
Vanderbilt Univ. Med. Ctr
🇺🇸Nashville, Tennessee, United States
Huntsman Cancer Hospital
🇺🇸Salt Lake City, Utah, United States
University of Utah Hospitals & Clinics
🇺🇸Salt Lake City, Utah, United States
University of Utah, Division of Cardiovascular Medicine
🇺🇸Salt Lake City, Utah, United States
Instituto Cardiovascular de Buenos Aires
🇦🇷Ciudad Autónoma de Buenos Aires, Argentina
Westmead Hospital
🇦🇺Westmead, New South Wales, Australia
Princess Alexandra Hospital
🇦🇺Woolloongabba, Queensland, Australia
Eastern Health (Box Hill Hospital)
🇦🇺Box Hill, Victoria, Australia
UZ Leuven
🇧🇪Leuven, Belgium
Hospital Universitário Clementino Fraga Filho (UFRJ)
🇧🇷Rio de Janeiro, RJ, Brazil
University of Calgary/Foothills Medical Centre
🇨🇦Calgary, Alberta, Canada
Vancouver General Hospital
🇨🇦Vancouver, British Columbia, Canada
Toronto General Hospital
🇨🇦Toronto, Ontario, Canada
Montreal Heart Institute
🇨🇦Montreal, Quebec, Canada
Fakultni nemocnice u sv. Anny v Brne
🇨🇿Brno, Czechia
Vseobecna fakultni nemocnice v Praze
🇨🇿Praha 2, Czechia
Institut klinicke a experimentalni mediciny
🇨🇿Praha 4, Czechia
Hopital de La Timone
🇫🇷Marseille, CAN, France
CHU Henri Mondor
🇫🇷Creteil, France
Hôpital De La Timone
🇫🇷Marseille, France
Hopital Bichat
🇫🇷Paris, France
CHU de Rennes - Hopital Pontchaillou
🇫🇷Rennes, France
CHU de Rennes - Hôpital Pontchaillou
🇫🇷Rennes, France
CHU de Toulouse - Hôpital Rangueil
🇫🇷Toulouse, France
Medical University of Heidelberg
🇩🇪Heidelberg, Germany
Universitätsklinikum Münster
🇩🇪Münster, Germany
Clinical Trial Pharmacy
🇭🇰Hong Kong, Hong Kong
Queen Mary Hospital
🇭🇰Hong Kong, Hong Kong
Azienda Ospedaliero-Universitaria Policlinico S. Orsola Malpighi
🇮🇹Bologna, Italy
Azienda Ospedaliero-Universitaria Careggi
🇮🇹Firenze, Italy
Fondazione IRCCS Policlinico San Matteo
🇮🇹Pavia, Italy
Kurume University Hospital
🇯🇵Kurume-shi, Fukuoka, Japan
Kumamoto University Hospital
🇯🇵Kumamoto-city, Kumamoto, Japan
Shinshu University Hospital
🇯🇵Nagano, Japan
University Medical Center Groningen
🇳🇱Groningen, Netherlands
Unidad de Insuficiencia Cardiaca Avanzada y Transplante Cardiaco
🇪🇸A Coruna, LA Coruna, Spain
Hospital Universitario Puerta de Hierro Majadahonda
🇪🇸Majadahonda, Madrid, Spain
Skellefteå Lasarett
🇸🇪Skellefteå, Sweden
Akademiska Sjukhuset
🇸🇪Uppsala, Sweden
Investigational Drug Services
🇨🇳Taipei, Taiwan
National Taiwan University Hospital
🇨🇳Taipei, Taiwan
Taipei Veterans General Hospital
🇨🇳Taipei, Taiwan
Barts Health NHS Trust, St Bartholomew's Hospital
🇬🇧London, United Kingdom