MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

To join this study, you would need to have been diagnosed with transthyretin amyloidosis affecting your heart, and have a history of heart failure. Your heart failure symptoms should be stable and well-managed in the month before starting the study, and a specific blood test for heart failure (NT-proBNP) must fall within a certain range.

There are also some reasons why you might not be able to join. For example, if your heart failure is very severe (classed as New York Heart Association Class IV), or if you are confined to a wheelchair or bed due to nerve damage. You also can't participate if you have certain infections like hepatitis B, hepatitis C, or HIV, or a recent history of cancer.

Additionally, if you've recently used certain other medications for amyloidosis within the last year, or if you have uncontrolled high blood pressure, severe kidney problems, or certain liver diseases, you would not be able to take part. You also need to be willing and able to take vitamin A supplements throughout the study.

If you decide to participate in this study, you will receive either the study drug NTLA-2001 or a placebo (a dummy drug) as a single infusion. This will be given in hospital. For the duration of the study, you will have regular check-ups, which include blood tests, heart assessments, and discussions about your health and any side effects you might experience. You will also need to take vitamin A supplements throughout the study. The total duration of your participation and the exact schedule of visits will be explained in detail by the study team, but it will involve ongoing monitoring after your single treatment.