TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

To join this study, you need to have been diagnosed with transthyretin amyloidosis that affects your heart, whether it's the type you're born with (hATTR) or the type that develops later in life (wATTR). You also need to have a history of heart failure, which might mean you've been in hospital for it or you're taking medication to help with symptoms like fluid build-up.

Your blood test results for a specific heart marker (called NT-proBNP) also need to be within a certain range. If you're already taking other approved medicines for your ATTR amyloidosis or for heart failure, that’s generally okay and you might still be able to take part.

However, you can't join if your heart failure is very severe (NYHA Class IV), or if it's quite severe (NYHA Class III) and your ATTR condition is also at an advanced stage (Stage 3). Also, if your nerves are significantly affected by the condition (PND Score IIIa, IIIb, or IV) or if your kidney function is very low, you won't be able to participate. Lastly, if you've previously received or are currently receiving other specific treatments that lower the transthyretin protein, you unfortunately cannot join this study.

If you decide to take part in this study, you will either receive the new medicine (nucresiran) or a placebo (a dummy treatment). Both will be given as injections. You will have regular visits to the clinic where doctors and nurses will monitor your health, perform physical exams, take blood samples, and conduct tests to check your heart and general well-being. They will also ask you to fill out questionnaires about how you are feeling and your quality of life. The total duration of your participation in the study will depend on how long the researchers need to collect enough information to understand the medicine's effects, but it typically involves several years of regular check-ups and follow-up after the main treatment period.