HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

To join this study, you must be between 18 and 85 years old and have a confirmed diagnosis of transthyretin (ATTR) amyloidosis that affects your heart. This means you would have been told by your doctor that you have this specific type of heart condition. You also need to have a history of heart failure, which might include having been admitted to hospital because of it, or your doctor has found signs of heart failure during an examination.

There are some reasons why you might not be able to join. For example, if you have other types of amyloidosis or certain other heart conditions that aren't ATTR. You also can't participate if your heart failure is very severe (known as NYHA Class IV or high-risk Class III) or if you have severe nerve damage from the amyloidosis. Additionally, if you have very poor kidney function or have already received other specific treatments for your TTR condition, you wouldn't be eligible.

Your study doctor will go through all the detailed requirements with you to make sure this study is the right fit for your health situation.

If you decide to join this study, you would receive the study medication – either vutrisiran or a placebo – as an injection under the skin once every three months. This would happen at regular visits to the study clinic. You would also have various medical tests and examinations at these visits, such as heart checks, blood tests, and physical examinations, to monitor your health and how you're responding to the treatment.

The study involves a number of visits over a period of time, and the research team will keep you informed about the schedule and what's involved at each appointment. You'll be closely monitored by healthcare professionals throughout the entire study duration, which could last for several years. All assessments are designed to keep you safe and gather important information about the new medicine.