The purpose of this study was to evaluate the safety and efficacy of revusiran (ALN-TTRSC) in patients with transthyretin (TTR) mediated Familial Amyloidotic Cardiomyopathy. Dosing has been discontinued; patients are being followed-up for safety.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
206
Clinical Trial Site
Bakersfield, California, United States
Clinical Trial Site
Beverly Hills, California, United States
Clinical Trial Site
La Mesa, California, United States
Clinical Trial Site
Los Angeles, California, United States
Clinical Trial Site
San Francisco, California, United States
6 Minute Walk Distance (6-MWD)
The difference between revusiran and placebo group in change from baseline to 18 months in the total distance walked in 6 minutes
Time frame: 18 months
Serum TTR Levels
The difference between revusiran (ALN-TTRSC) and placebo group in the percent reduction in serum TTR levels over 18 months
Time frame: 18 months
Composite Cardiovascular (CV) Mortality and Cardiovascular (CV) Hospitalization
Number of cardiovascular-related deaths and cardiovascular-related hospitalizations in the placebo group compared to the revusiran (ALN-TTRSC) treatment group
Time frame: 18 months
New York Heart Association (NYHA) Class
The difference between revusiran (ALN-TTRSC) and placebo group in the change from baseline to 18 months in the NYHA class
Time frame: 18 months
Kansas City Cardiomyopathy Questionnaire (KCCQ)
The difference between revusiran (ALN-TTRSC) and placebo group in the change from Baseline to 18 months in the Kansas City Cardiomyopathy Questionnaire
Time frame: 18 months
Cardiovascular (CV) Mortality
Number of cardiovascular-related deaths in the placebo group compared to the revusiran (ALN-TTRSC) treatment group
Time frame: 18 months
Cardiovascular (CV) Hospitalization
Number of cardiovascular-related hospitalizations in the placebo group compared to the revusiran (ALN-TTRSC) treatment group
Time frame: 18 months
All-cause Mortality
Total number of deaths in the placebo group compared to the revusiran (ALN-TTRSC) treatment group
Time frame: 18 months
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Clinical Trial Site
Stanford, California, United States
Clinical Trial Site
Torrance, California, United States
Clinical Trial Site
Aurora, Colorado, United States
Clinical Trial Site
Washington D.C., District of Columbia, United States
Clinical Trial Site
Washington D.C., District of Columbia, United States
...and 51 more locations