A study of NTLA-2001 for transthyretin amyloidosis with heart problems
Part of Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This study tests a new treatment, NTLA-2001, for people with a type of heart disease called transthyretin amyloidosis with cardiomyopathy. It aims to see if the medicine can help improve heart function and quality of life.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of transthyretin amyloidosis that affects your heart.
- You must have a history of heart failure that is well-controlled and stable for at least 28 days before the study starts.
- Your NT-proBNP level (a heart stress marker) must be between 600 and 10,000 pg/mL.
- You cannot have severe heart failure (class IV) or be wheelchair- or bed-bound due to nerve damage.
- You must not have used certain RNA-targeting medications (like patisiran, inotersen, or eplontersen) in the last year, and never have used vutrisiran.
- Your kidney function must be good (eGFR 30 or higher), and you must be willing to take vitamin A supplements during the study.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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This study is testing a new gene-silencing treatment called NTLA-2001 for people with hereditary ATTR amyloidosis that causes nerve damage. It works by stopping the body from making a faulty protein that builds up and harms nerves.
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