The purpose of this study is to evaluate the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
360
Change From Baseline at Month 12 in Six-Minute Walk Test (6-MWT)
Distance in meters walked in 6 minutes, longer distances indicate greater functional capacity. Missing 6MWT values due to non-COVID-19 death or inability to walk due to ATTR disease progression were imputed using the worst 10th percentile change observed in the DB period. Missing 6-MWT values due to other reasons are multiply imputed to create 100 complete datasets. The change from baseline is averaged across the 100 complete datasets.
Time frame: Baseline, Month 12
Change From Baseline at Month 12 in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) Score
The KCCQ is a 23-item self-administered questionnaire quantifying 6 domains (symptoms, physical function, quality of life, social limitation, self-efficacy, and symptom stability) and 2 summary scores (clinical and overall summary \[OS\]). Scores are transformed to a range of 0-100, in which higher scores reflect better health status.
Time frame: Baseline, Month 12
Composite Endpoint of All-Cause Mortality, Frequency of Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) and Change From Baseline in 6-MWT Analyzed by Win Ratio
The composite endpoint was analyzed using the stratified win ratio method, stratified by baseline tafamidis use. This method combines all-cause mortality, frequency of CV events (CV hospitalizations and HF visits) and change from baseline in 6-MWT in a hierarchical fashion. This method makes within-stratum pairwise comparisons for all patisiran-placebo participant pairs in a sequential manner (first mortality, then CV events, then 6-MWT), with later steps evaluated only in the case of a tie on the prior step. Within each stratum, the win ratio is the total number of 'winners' divided by the total number of 'losers' in the active group. A win ratio \>1 represents a favorable outcome for patisiran.
Time frame: Up to Month 12
Composite Endpoint of All-Cause Mortality and Frequency of All-Cause Hospitalizations and Urgent HF Visits in Participants Not on Tafamidis at Baseline
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Clinical Trial Site
Los Angeles, California, United States
Clinical Trial Site
Chicago, Illinois, United States
Clinical Trial Site
Skokie, Illinois, United States
Clinical Trial Site
Iowa City, Iowa, United States
Clinical Trial Site
Kansas City, Kansas, United States
Clinical Trial Site
Baltimore, Maryland, United States
Clinical Trial Site
Boston, Massachusetts, United States
Clinical Trial Site
Burlington, Massachusetts, United States
Clinical Trial Site
Rochester, Minnesota, United States
Clinical Trial Site
St Louis, Missouri, United States
...and 80 more locations
The hazard rate of all-cause mortality and all-cause hospitalizations and urgent HF visits will be compared between treatment groups using an Andersen-Gill model. A hazard ratio \<1 represents a favorable outcome for patisiran.
Time frame: Up to Month 12
Composite Endpoint of All-cause Mortality and Frequency of All-cause Hospitalizations and Urgent HF Visits in All Participants
The hazard rate of all-cause mortality and all-cause hospitalizations and urgent HF visits was compared between treatment groups using a modified Andersen-Gill model. A hazard ratio \<1 represents a favorable outcome for patisiran.
Time frame: Up to Month 12