This research study is looking at a condition called transthyretin amyloid cardiomyopathy, which is a disease where an abnormal protein called transthyretin builds up in the heart muscle and causes it to become stiff and not work properly. This buildup leads to heart failure, which means the heart cannot pump blood as well as it should, causing symptoms like shortness of breath, swelling in the legs, and tiredness. The disease can occur in two forms: one where the transthyretin protein is normal but still forms harmful deposits, and another where the protein has a genetic change that makes it more likely to form these deposits. The study will test a new medicine called coramitug, which is also known by its code name NNC6019-0001. This medicine is designed to target and remove the misfolded transthyretin protein that has built up in the heart. Participants in the study will receive either coramitug or placebo, both given in addition to their standard heart failure treatments.
The main goal of the study is to see if coramitug works better than placebo at reducing serious heart-related problems and deaths in people with this condition. During the study, participants will receive the study medicine through an intravenous infusion, which means it will be given directly into a vein. The treatment will continue for up to 192 weeks, which is about three and a half years. Throughout this time, doctors will monitor how well participants’ hearts are working, how far they can walk in six minutes, and how they feel in their daily lives using questionnaires about their symptoms and quality of life. The study will also track important events such as hospital visits for heart problems, worsening of heart failure requiring urgent medical care, and deaths related to heart and blood vessel problems.
To be eligible for this study, participants must have a confirmed diagnosis of transthyretin amyloid cardiomyopathy, which can be proven either through a heart tissue sample showing the abnormal protein deposits or through special imaging scans combined with other tests. They must have ongoing heart failure that requires treatment with water pills and must have either been hospitalized for heart failure at least once or have had clear signs of fluid buildup or heart pressure problems. Participants need to be on stable heart medications for at least four weeks before starting the study treatment, and they must have certain levels of a substance in their blood called NT-proBNP, which is a marker that shows how much strain the heart is under. They also need to be able to walk more than 50 meters during a walking test at the beginning of the study.
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