Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy (ATTR-ACT)
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|ClinicalTrials.gov Identifier: NCT01994889|
Recruitment Status : Completed
First Posted : November 26, 2013
Results First Posted : April 3, 2019
Last Update Posted : April 24, 2019
|Condition or disease||Intervention/treatment||Phase|
|Transthyretin (TTR) Amyloid Cardiomyopathy||Drug: Tafamidis Drug: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||441 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A MULTICENTER, INTERNATIONAL, PHASE 3, DOUBLE-BLIND, PLACEBO-CONTROLLED, RANDOMIZED STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF DAILY ORAL DOSING OF TAFAMIDIS MEGLUMINE (PF-06291826) 20 MG OR 80 MG IN COMPARISON TO PLACEBO IN SUBJECTS DIAGNOSED WITH TRANSTHYRETIN CARDIOMYOPATHY (TTR-CM)|
|Actual Study Start Date :||December 9, 2013|
|Actual Primary Completion Date :||February 7, 2018|
|Actual Study Completion Date :||February 7, 2018|
Experimental: Tafamidis - 20 mg
Active Treatment-Low dose
Tafamidis 20 mg in soft gel capsules administered once a day for 30 months
Experimental: Tafamidis - 80 mg
Active Treatment-High Dose
Tafamidis 80 mg in soft gel capsules administered once a day for 30 months
Placebo Comparator: Placebo
Placebo in soft gel capsules administered once a day for 30 months
- Hierarchical Combination of All-Cause Mortality and Frequency of Cardiovascular-Related Hospitalizations [ Time Frame: Baseline up to Month 30 ]All-cause mortality and frequency of cardiovascular hospitalization were analyzed using Finkelstein-Schoenfeld method. The method combines all-cause mortality and frequency of CV-related hospitalizations in a hierarchical fashion using all-cause mortality first. The method compares every participant with every other participant within strata, assigning a +1 to the "better" participant and a -1 to the "worse" participant and 0 if they are "tied". Participants who discontinued for transplantation (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. 'Win' represents a participant doing better based on hierarchical comparison. The reported unit is the total "wins" for each treatment group from performing such a hierarchical comparison across all 4 strata in the study.
- All-Cause Mortality [ Time Frame: Baseline up to Month 30 ]Number of deaths due to any cause was analyzed. Participants who discontinued for transplantation (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.
- Frequency of Cardiovascular-Related Hospitalizations [ Time Frame: Baseline to Month 30 ]CV related hospitalizations per year is calculated as participant's number of CV related hospitalizations upon duration on study in years.
- Change From Baseline in the Total Distance Walked During 6 Minute Walk Test (6MWT) at Month 30 [ Time Frame: Baseline, Month 30 ]6MWT is the total distance that a participant could walk in 6 minutes. Participants were asked to perform the test at a pace that was comfortable to them, with as many breaks as they needed.
- Change From Baseline in Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS) at Month 30 [ Time Frame: Baseline, Month 30 ]KCCQ is a 23-item participant-completed questionnaire that assesses health status and health-related quality of life in participants with heart failure. Eight domain scores were calculated for the KCCQ: Physical limitation, Social limitation, Quality of life, Self-efficacy, Symptom stability, Symptom frequency, Symptom burden, and Total symptoms (calculated as the mean of Symptom frequency and Symptom burden scores). Two summary scores were also calculated: Clinical Summary (calculated as mean of Physical limitation and Total symptom scores) and Overall Summary (calculated as mean of Physical limitation, Social limitation, Total symptoms, and Quality of life scores). Domain and summary scores were scaled to range from 0 (minimum) to 100 (maximum); higher scores represent better health status.
- Number of Participants With Cardiovascular-Related Mortality [ Time Frame: Baseline up to Month 30 ]Deaths adjudicated as CV-related and indeterminate are reported. Participants who discontinued for transplantation (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.
- Percentage of Participants With Stabilized Transthyretin (TTR) at Month 1 [ Time Frame: Month 1 ]TTR stabilization is a measure of the degree of stabilization afforded the TTR molecule by tafamidis.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01994889
|Study Director:||Pfizer CT.gov Call Center||Pfizer|