This study is testing a potential new medicine, NNC6019-0001, for people who have a heart disease due to TTR amyloidosis.The study will look at if this medicine can reduce the symptoms of a heart disease due to TTR amyloidosis, such as heart failure. Participants will either get NNC6019-0001 (apotential new medicine) or placebo (a medicine which has no effect on the body). Which treatment participants get is decided by chance. The chance of getting NNC6019-0001 is two times higher than getting placebo. NNC6019-0001 is not yet approved in any country or region in the world. It is a new medicine that doctors cannot prescribe yet. Participants will get an infusion of the study medicine 13 times, once every 4 weeks. The study will last for about 64 weeks after the first dose of medicine. Participants cannot participate in this study if they have a heart disease other than a heart disease due to TTR amyloidosis.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
105
Participants will receive i.v infusionof NNC6019-0001.
Participants will receive i.v. infusion of placebo (NNC6019-0001).
Mayo Clinic Arizona
Phoenix, Arizona, United States
Cedars-Sinai Medical Center_Los Angeles
Beverly Hills, California, United States
Stanford Hlth Cre-Boswell Clin
Stanford, California, United States
Mayo Clinic Jacksonville
Jacksonville, Florida, United States
NW Univ-Bluhm Cardiovasc Inst
Evanston, Illinois, United States
Change in 6-minute walk test (6-MWT)
Measured in Meters
Time frame: From baseline (week 0) to visit 15 (week 52)
Change in N-terminal-pro brain natriuretic peptide (NT-proBNP)
Measured in Percentage
Time frame: From baseline (week 0) to visit 15 (week 52)
Change in myocardial extracellular volume (ECV)
Measured in Percentage (%)-points
Time frame: From baseline (week 0) to visit 15 (week 52)
Change in Kansas City Cardiomyopathy Questionnaire (KCCQ) Clinical Summary Score (CSS)
The KCCQ is a disease-specific health status instrument composed of 23 items that quantify the domains of physical limitation, symptoms, self-efficacy, social limitation, and health-related quality of life limitation from heart failure. The overall summary score and all domains have been independently demonstrated to be valid, reliable, and responsive to clinical change. CSS scores range from 0 to 100 and lower scores represent more severe symptoms and/or limitations and scores of 100 indicate no symptoms, no limitations, and excellent quality of life.
Time frame: From baseline (week 0) to visit 15 (week 52)
Change in neuropathy impairment score (NIS)
NIS is a clinical assessment that tests muscle strength, reflex activity, and sensation of toes and fingers, and can be used to assess neurologic function in hereditary transthyretin amyloid (hATTR). The total NIS score is graded on a scale of 0-244, with a higher score indicating greater impairment.
Time frame: From baseline (week 0) to visit 15 (week 52)
Change in troponin I
Measured in nanogram per milliliter (ng/mL)
Time frame: From baseline (week 0) to visit 15 (week 52)
Change in global longitudinal strain (GLS) on echocardiography
Measured in Percentage (%)-points
Time frame: From baseline (week 0) to visit 15 (week 52)
Number of treatment emergent adverse events
Measured as Events
Time frame: From baseline (week 0) to visit 16 (week 64)
Time to occurrence of all-cause mortality
Measured in Weeks
Time frame: From baseline (week 0) to visit 16 (week 64)
Number of cardiovascular (CV) events comprising hospitalisation due to CV events or urgent heart failure visits
Measured as Events
Time frame: From baseline (week 0) to visit 16 (week 64)
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Univ of MD Schl of Med
Baltimore, Maryland, United States
University of Maryland School of Medicine
Baltimore, Maryland, United States
Mayo Clinic Rochester
Rochester, Minnesota, United States
Oregon Hlth Sci Univ-Portland
Portland, Oregon, United States
University of Calgary_Cardiology
Calgary, Alberta, Canada
...and 25 more locations