The purpose of this study is to obtain safety, efficacy, and pharmacodynamic data on the use of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy who continued on extended use of vutrisiran, or switched from patisiran.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
700
Vutrisiran administered SC q3M
Frequency of Adverse Events (AEs)
An AE is any untoward medical occurrence in a patient or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment.
Time frame: Up to 36 Months
Change from Baseline in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS)
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 up to 36 Months
Change from Baseline in New York Heart Association (NYHA) Class
NYHA classification grades the severity of heart failure symptoms into the following stages: I (no symptoms; ordinary physical activity such as walking and climbing stairs does not cause fatigue or dyspnea), II (symptoms with ordinary physical activity; walking or climbing stairs rapidly, walking uphill, walking or stair climbing after meals, in cold weather, in wind or when under emotional stress causes undue fatigue or dyspnea), III (symptoms with less than ordinary physical activity; walking 1 to 2 blocks on the level and climbing more than 1 flight of stairs in normal conditions causes undue fatigue or dyspnea), IV (symptoms at rest; inability to carry on any physical activity without fatigue or dyspnea).
Time frame: Baseline up to 36 Months
Change From Baseline in the Cardiac Biomarker: N-terminal Prohormone of B-type Natriuretic Peptide (NT-proBNP)
NT-proBNP will be assessed through measurement of serum levels.
Time frame: Baseline up to 36 Months
Change From Baseline in the Cardiac Biomarker: Troponin I
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Clinical Trial Site
Phoenix, Arizona, United States
Clinical Trial Site
Beverly Hills, California, United States
Clinical Trial Site
La Jolla, California, United States
Clinical Trial Site
Stanford, California, United States
Clinical Trial Site
Washington D.C., District of Columbia, United States
Clinical Trial Site
Chicago, Illinois, United States
Clinical Trial Site
Glenview, Illinois, United States
Clinical Trial Site
Indianapolis, Indiana, United States
Clinical Trial Site
Kansas City, Kansas, United States
Clinical Trial Site
Baltimore, Maryland, United States
...and 62 more locations
Troponin I will be assessed through measurement of serum levels.
Time frame: Baseline up to 36 Months
All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits)
All deaths, hospitalizations and urgent HF visits will be recorded throughout the study as part of SAE and AE monitoring
Time frame: Up to 36 Months
Change from baseline in serum TTR level
Time frame: Baseline, Month 6 and Month 12